Trial Outcomes & Findings for A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia (NCT NCT04577352)
NCT ID: NCT04577352
Last Updated: 2026-04-13
Results Overview
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. Least square (LS) mean and standard error (SE) was calculated using mixed-model repeated measures (MMRM).
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
146 participants
Primary outcome timeframe
Baseline, Week 72
Results posted on
2026-04-13
Participant Flow
The study included a double-blind, placebo-controlled phase and an open-label extension phase.
Participant milestones
| Measure |
Vatiquinone
Participants received vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Double-blind Phase (72 Weeks)
STARTED
|
73
|
73
|
|
Double-blind Phase (72 Weeks)
Received at Least 1 Dose of Study Drug
|
73
|
73
|
|
Double-blind Phase (72 Weeks)
COMPLETED
|
63
|
65
|
|
Double-blind Phase (72 Weeks)
NOT COMPLETED
|
10
|
8
|
|
Open-label Phase (24 Weeks)
STARTED
|
63
|
65
|
|
Open-label Phase (24 Weeks)
Received at Least 1 Dose of Study Drug
|
63
|
65
|
|
Open-label Phase (24 Weeks)
COMPLETED
|
63
|
62
|
|
Open-label Phase (24 Weeks)
NOT COMPLETED
|
0
|
3
|
Reasons for withdrawal
| Measure |
Vatiquinone
Participants received vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Double-blind Phase (72 Weeks)
Adverse Event
|
6
|
3
|
|
Double-blind Phase (72 Weeks)
Withdrawal by Subject
|
3
|
2
|
|
Double-blind Phase (72 Weeks)
Death
|
0
|
1
|
|
Double-blind Phase (72 Weeks)
Protocol Violation
|
0
|
1
|
|
Double-blind Phase (72 Weeks)
Other than specified
|
1
|
1
|
|
Open-label Phase (24 Weeks)
Adverse Event
|
0
|
2
|
|
Open-label Phase (24 Weeks)
Other than specified
|
0
|
1
|
Baseline Characteristics
The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
Baseline characteristics by cohort
| Measure |
Vatiquinone
n=73 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=73 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
Total
n=146 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
18.9 years
STANDARD_DEVIATION 12.24 • n=73 Participants
|
18.2 years
STANDARD_DEVIATION 11.27 • n=73 Participants
|
18.6 years
STANDARD_DEVIATION 11.73 • n=146 Participants
|
|
Sex: Female, Male
Female
|
45 Participants
n=73 Participants
|
42 Participants
n=73 Participants
|
87 Participants
n=146 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=73 Participants
|
31 Participants
n=73 Participants
|
59 Participants
n=146 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
10 Participants
n=73 Participants
|
9 Participants
n=73 Participants
|
19 Participants
n=146 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
63 Participants
n=73 Participants
|
63 Participants
n=73 Participants
|
126 Participants
n=146 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=73 Participants
|
1 Participants
n=73 Participants
|
1 Participants
n=146 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=73 Participants
|
0 Participants
n=73 Participants
|
0 Participants
n=146 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=73 Participants
|
0 Participants
n=73 Participants
|
0 Participants
n=146 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=73 Participants
|
0 Participants
n=73 Participants
|
0 Participants
n=146 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=73 Participants
|
0 Participants
n=73 Participants
|
0 Participants
n=146 Participants
|
|
Race (NIH/OMB)
White
|
68 Participants
n=73 Participants
|
69 Participants
n=73 Participants
|
137 Participants
n=146 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=73 Participants
|
0 Participants
n=73 Participants
|
0 Participants
n=146 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
5 Participants
n=73 Participants
|
4 Participants
n=73 Participants
|
9 Participants
n=146 Participants
|
|
Modified Friedreich's Ataxia Rating Scale (mFARS) Score
|
43.30 units on a scale
STANDARD_DEVIATION 10.754 • n=73 Participants • The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
|
42.47 units on a scale
STANDARD_DEVIATION 11.637 • n=70 Participants • The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
|
42.89 units on a scale
STANDARD_DEVIATION 11.163 • n=143 Participants • The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
|
PRIMARY outcome
Timeframe: Baseline, Week 72Population: The mITT analysis set included all randomized participants, between age of 7 and 21 years who received at least 1 dose of study drug (vatiquinone or placebo), had baseline and at least 1 postbaseline measurement of primary endpoint.
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. Least square (LS) mean and standard error (SE) was calculated using mixed-model repeated measures (MMRM).
Outcome measures
| Measure |
Vatiquinone
n=61 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=62 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set
|
1.218 units on a scale
Standard Error 0.9652 • Interval 0.9652 to
|
2.828 units on a scale
Standard Error 0.9994 • Interval 0.9994 to
|
PRIMARY outcome
Timeframe: Baseline, Week 72Population: The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Outcome measures
| Measure |
Vatiquinone
n=70 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=73 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis Set
|
0.898 units on a scale
Standard Error 0.8500 • Interval 0.85 to
|
2.555 units on a scale
Standard Error 0.8770 • Interval 0.877 to
|
SECONDARY outcome
Timeframe: Baseline, Week 72Population: The mITT analysis set included all randomized participants, between age of 7 and 21 years, inclusive, at Screening, had received at least 1 dose of study drug (vatiquinone or placebo), had baseline and at least 1 post-baseline measurement of the primary endpoint.
The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Outcome measures
| Measure |
Vatiquinone
n=61 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=62 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72 - mITT Analysis Set
|
0.759 units on a scale
Standard Error 0.5992 • Interval 0.5992 to
|
1.677 units on a scale
Standard Error 0.6202 • Interval 0.6202 to
|
SECONDARY outcome
Timeframe: Baseline, Week 72Population: The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Outcome measures
| Measure |
Vatiquinone
n=70 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=73 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in FARS-ADL Score at Week 72 - ITT Analysis Set
|
0.707 units on a scale
Standard Error 0.5219 • Interval 0.5219 to
|
1.692 units on a scale
Standard Error 0.5402 • Interval 0.5402 to
|
SECONDARY outcome
Timeframe: Baseline, Week 72Population: The mITT analysis set included all randomized participants, between age of 7 and 21 years, inclusive, at Screening, had received at least 1 dose of study drug (vatiquinone or placebo), had baseline and at least 1 post-baseline measurement of the primary endpoint.
The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. The mean change from baseline in the distance the participant walked is reported. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Outcome measures
| Measure |
Vatiquinone
n=61 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=62 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72 - mITT Analysis Set
|
-4.324 meters
Standard Error 2.5882 • Interval 2.5882 to
|
-9.290 meters
Standard Error 2.6843 • Interval 2.6843 to
|
SECONDARY outcome
Timeframe: Baseline, Week 72Population: The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. The mean change from baseline in the distance the participant walked is reported. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Outcome measures
| Measure |
Vatiquinone
n=70 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=73 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in 1MWT at Week 72 - ITT Analysis Set
|
-4.623 meters
Standard Error 2.2113 • Interval 2.2113 to
|
-9.462 meters
Standard Error 2.2687 • Interval 2.2687 to
|
SECONDARY outcome
Timeframe: Week 1-24, Week 25-48, and Week 49-72Population: The mITT analysis set included all randomized participants, between age of 7 and 21 years, inclusive, at Screening, had received at least 1 dose of study drug (vatiquinone or placebo), had baseline and at least 1 post-baseline measurement of the primary endpoint. 'Overall number of participants analyzed' = participants evaluable for this endpoint. 'Number analyzed' = participants evaluable at specified timepoint.
Each participant was required to maintain a fall log, which included the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the analysis.
Outcome measures
| Measure |
Vatiquinone
n=60 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=62 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Number of Falls Per 28 Days Over Every 24-Week Period - mITT Analysis Set
Week 25-48
|
4.098 falls
Standard Deviation 11.5289
|
3.900 falls
Standard Deviation 9.0212
|
|
Number of Falls Per 28 Days Over Every 24-Week Period - mITT Analysis Set
Week 49-72
|
3.732 falls
Standard Deviation 10.0790
|
4.462 falls
Standard Deviation 12.1785
|
|
Number of Falls Per 28 Days Over Every 24-Week Period - mITT Analysis Set
Week 1-24
|
5.029 falls
Standard Deviation 14.4686
|
3.999 falls
Standard Deviation 6.9569
|
SECONDARY outcome
Timeframe: Week 1-24, Week 25-48, and Week 49-72Population: The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint. 'Overall number of participants analyzed' = participants evaluable for this endpoint. 'Number analyzed' = participants evaluable at specified timepoint.
Each participant was required to maintain a fall log, which included the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the analysis.
Outcome measures
| Measure |
Vatiquinone
n=69 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=73 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Number of Falls Per 28 Days Over Every 24-Week Period - ITT Analysis Set
Week 1-24
|
4.464 falls
Standard Deviation 13.5618
|
3.535 falls
Standard Deviation 6.5231
|
|
Number of Falls Per 28 Days Over Every 24-Week Period - ITT Analysis Set
Week 25-48
|
3.920 falls
Standard Deviation 10.9850
|
3.518 falls
Standard Deviation 8.4701
|
|
Number of Falls Per 28 Days Over Every 24-Week Period - ITT Analysis Set
Week 49-72
|
3.407 falls
Standard Deviation 9.6043
|
3.974 falls
Standard Deviation 11.4690
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 72Population: The mITT analysis set included all randomized participants, between age of 7 and 21 years who received at least 1 dose of study drug (vatiquinone or placebo), had baseline and at least 1 postbaseline measurement of primary endpoint.
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The Upright Stability subscale of the mFARS includes following items: 1. Sitting posture (score: 0 to 4); 2a. Stance with feet apart and eyes open (score: 0 to 4); 2b. Stance with feet apart and eyes closed (score: 0 to 4); 3a. Stance with feet together and eyes open (score: 0 to 4); 3b. Stance with feet together and eyes closed (score: 0 to 4); 4. Tandem stance (score: 0 to 4); 5. Stance on dominant foot (score: 0 to 4); 6. Tandem walk (score: 0 to 3); 7. Gait (score: 0 to 5). The upright stability subscale total score ranges from 0 (normal) to 36 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Outcome measures
| Measure |
Vatiquinone
n=61 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=62 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - mITT Analysis Set
|
1.734 units on a scale
Standard Error 0.4911 • Interval 0.4911 to
|
2.991 units on a scale
Standard Error 0.5094 • Interval 0.5094 to
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 72Population: The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The Upright Stability subscale of the mFARS includes following items: 1. Sitting posture (score: 0 to 4); 2a. Stance with feet apart and eyes open (score: 0 to 4); 2b. Stance with feet apart and eyes closed (score: 0 to 4); 3a. Stance with feet together and eyes open (score: 0 to 4); 3b. Stance with feet together and eyes closed (score: 0 to 4); 4. Tandem stance (score: 0 to 4); 5. Stance on dominant foot (score: 0 to 4); 6. Tandem walk (score: 0 to 3); 7. Gait (score: 0 to 5). The upright stability subscale total score ranges from 0 (normal) to 36 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Outcome measures
| Measure |
Vatiquinone
n=70 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=73 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - ITT Analysis Set
|
1.379 units on a scale
Standard Error 0.4321 • Interval 0.4321 to
|
2.489 units on a scale
Standard Error 0.4468 • Interval 0.4468 to
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 72Population: The mITT analysis set included all randomized participants, between age of 7 and 21 years who received at least 1 dose of study drug (vatiquinone or placebo), had baseline and at least 1 postbaseline measurement of primary endpoint.
The MFIS is a 21-item, reliable, validated instrument that has been utilized in many neurological disorders. It is a modified form of the Fatigue Impact Scale, a component of the Multiple Sclerosis Quality of Life Inventory. Each item was scored on a scale of 0 (never) to 4 (almost always). The total score was the sum of all item's score and ranged from 0 (no fatigue impact) to 84 (almost always impacted by fatigue). Higher scores indicated greater impact of fatigue on participant function.
Outcome measures
| Measure |
Vatiquinone
n=61 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=62 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in the Modified Fatigue Impact Scale (MFIS) Score at Week 72 - mITT Analysis Set
|
-0.756 units on a scale
Standard Error 2.2197 • Interval 2.2197 to
|
4.291 units on a scale
Standard Error 2.3344 • Interval 2.3344 to
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 72Population: The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
The MFIS is a 21-item, reliable, validated instrument that has been utilized in many neurological disorders. It is a modified form of the Fatigue Impact Scale, a component of the Multiple Sclerosis Quality of Life Inventory. Each item was scored on a scale of 0 (never) to 4 (almost always). The total score was the sum of all item's score and ranged from 0 (no fatigue impact) to 84 (almost always impacted by fatigue). Higher scores indicated greater impact of fatigue on participant function.
Outcome measures
| Measure |
Vatiquinone
n=70 Participants
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
|
Placebo
n=73 Participants
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|
|
Change From Baseline in the MFIS Score at Week 72 - ITT Analysis Set
|
-1.200 units on a scale
Standard Error 1.9897 • Interval 1.9897 to
|
4.354 units on a scale
Standard Error 2.0758 • Interval 2.0758 to
|
Adverse Events
Double-blind Phase: Vatiquinone
Serious events: 8 serious events
Other events: 71 other events
Deaths: 1 deaths
Double-blind Phase: Placebo
Serious events: 8 serious events
Other events: 73 other events
Deaths: 1 deaths
On-Vatiquinone Period: Vatiquinone/Vatiquinone
Serious events: 10 serious events
Other events: 71 other events
Deaths: 1 deaths
On-Vatiquinone Period: Placebo/Vatiquinone
Serious events: 2 serious events
Other events: 53 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Double-blind Phase: Vatiquinone
n=73 participants at risk
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase.
|
Double-blind Phase: Placebo
n=73 participants at risk
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase.
|
On-Vatiquinone Period: Vatiquinone/Vatiquinone
n=73 participants at risk
Participants who received vatiquinone for 72 weeks in the double-blind phase continued to receive vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
On-Vatiquinone Period: Placebo/Vatiquinone
n=65 participants at risk
Participants who received placebo for 72 weeks in the double-blind phase, received vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|---|---|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Cardiac disorders
Cardiac failure
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Cardiac disorders
Myocarditis
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Nervous system disorders
Seizure
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Psychiatric disorders
Anxiety
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Psychiatric disorders
Completed suicide
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Immune system disorders
Multisystem inflammatory syndrome in children
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Appendicitis perforated
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Gastroenteritis
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Gastroenteritis viral
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Injury, poisoning and procedural complications
Intentional product misuse
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Psychiatric disorders
Depression
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Psychiatric disorders
Suicide attempt
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Metabolism and nutrition disorders
Type 2 diabetes mellitus
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Impaired gastric emptying
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
COVID-19
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Nervous system disorders
Presyncope
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
Other adverse events
| Measure |
Double-blind Phase: Vatiquinone
n=73 participants at risk
Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase.
|
Double-blind Phase: Placebo
n=73 participants at risk
Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase.
|
On-Vatiquinone Period: Vatiquinone/Vatiquinone
n=73 participants at risk
Participants who received vatiquinone for 72 weeks in the double-blind phase continued to receive vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
On-Vatiquinone Period: Placebo/Vatiquinone
n=65 participants at risk
Participants who received placebo for 72 weeks in the double-blind phase, received vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
|
|---|---|---|---|---|
|
Infections and infestations
Gastroenteritis
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Gastroenteritis viral
|
6.8%
5/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Influenza
|
11.0%
8/73 • Number of events 9 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
15.1%
11/73 • Number of events 13 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
12.3%
9/73 • Number of events 10 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
3.1%
2/65 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Nasopharyngitis
|
16.4%
12/73 • Number of events 19 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
23.3%
17/73 • Number of events 34 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
16.4%
12/73 • Number of events 20 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
9.2%
6/65 • Number of events 9 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Sinusitis
|
5.5%
4/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Cardiac disorders
Tachycardia
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Abdominal pain
|
13.7%
10/73 • Number of events 15 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
8.2%
6/73 • Number of events 7 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
13.7%
10/73 • Number of events 15 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
9.6%
7/73 • Number of events 9 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
13.7%
10/73 • Number of events 17 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
11.0%
8/73 • Number of events 11 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
12.3%
8/65 • Number of events 14 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Diarrhoea
|
26.0%
19/73 • Number of events 28 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
13.7%
10/73 • Number of events 12 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
28.8%
21/73 • Number of events 31 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
7.7%
5/65 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Dyspepsia
|
5.5%
4/73 • Number of events 8 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 10 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Nausea
|
17.8%
13/73 • Number of events 16 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
13.7%
10/73 • Number of events 16 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
20.5%
15/73 • Number of events 19 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
7.7%
5/65 • Number of events 8 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Odynophagia
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Gastrointestinal disorders
Vomiting
|
19.2%
14/73 • Number of events 17 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
12.3%
9/73 • Number of events 13 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
23.3%
17/73 • Number of events 21 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
10.8%
7/65 • Number of events 10 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
General disorders
Fatigue
|
12.3%
9/73 • Number of events 12 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
17.8%
13/73 • Number of events 21 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
15.1%
11/73 • Number of events 14 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.6%
3/65 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
General disorders
Pyrexia
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
12.3%
9/73 • Number of events 14 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
3.1%
2/65 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
COVID-19
|
43.8%
32/73 • Number of events 36 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
32.9%
24/73 • Number of events 26 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
49.3%
36/73 • Number of events 41 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.2%
4/65 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Upper respiratory tract infection
|
12.3%
9/73 • Number of events 17 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
11.0%
8/73 • Number of events 10 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
16.4%
12/73 • Number of events 24 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
12.3%
8/65 • Number of events 9 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Urinary tract infection
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
2.7%
2/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
8.2%
6/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Viral infection
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
3.1%
2/65 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Viral rhinitis
|
11.0%
8/73 • Number of events 18 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.1%
3/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
15.1%
11/73 • Number of events 24 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Injury, poisoning and procedural complications
Contusion
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
8.2%
6/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Investigations
Weight increased
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
8.2%
6/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
6.8%
5/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Nervous system disorders
Headache
|
30.1%
22/73 • Number of events 37 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
35.6%
26/73 • Number of events 53 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
34.2%
25/73 • Number of events 43 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
10.8%
7/65 • Number of events 9 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.2%
6/73 • Number of events 7 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
9.6%
7/73 • Number of events 9 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Nervous system disorders
Syncope
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.6%
7/73 • Number of events 8 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
8.2%
6/73 • Number of events 8 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
12.3%
9/73 • Number of events 14 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
8.2%
6/73 • Number of events 7 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
3.1%
2/65 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
12.3%
9/73 • Number of events 11 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
9.6%
7/73 • Number of events 10 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
12.3%
9/73 • Number of events 11 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Psychiatric disorders
Anxiety
|
6.8%
5/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.1%
3/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
8.2%
6/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Nervous system disorders
Balance disorder
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Nervous system disorders
Dizziness
|
15.1%
11/73 • Number of events 15 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
11.0%
8/73 • Number of events 8 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
16.4%
12/73 • Number of events 18 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
10.8%
7/65 • Number of events 8 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Injury, poisoning and procedural complications
Fall
|
79.5%
58/73 • Number of events 363 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
83.6%
61/73 • Number of events 441 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
80.8%
59/73 • Number of events 468 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
52.3%
34/65 • Number of events 89 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Investigations
International normalised ratio increased
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/73 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
5.5%
4/73 • Number of events 9 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.1%
3/73 • Number of events 8 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 9 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.8%
5/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
8.2%
6/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
8.2%
6/73 • Number of events 8 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.1%
3/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
11.0%
8/73 • Number of events 10 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
11.0%
8/73 • Number of events 11 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
11.0%
8/73 • Number of events 11 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
11.0%
8/73 • Number of events 11 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.6%
3/65 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
2.7%
2/73 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.5%
1/65 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
4.1%
3/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
5.5%
4/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
3.1%
2/65 • Number of events 2 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
4.1%
3/73 • Number of events 3 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
1.4%
1/73 • Number of events 1 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 5 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.5%
4/73 • Number of events 4 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 6 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
6.8%
5/73 • Number of events 7 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
0.00%
0/65 • Baseline up to Week 100
Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER