Trial Outcomes & Findings for Troriluzole in Adult Participants With Spinocerebellar Ataxia (NCT NCT03701399)
NCT ID: NCT03701399
Last Updated: 2025-11-04
Results Overview
The f-SARA was a 4-item performance based scale: 1-gait, 2-stance, 3-sitting, 4-speech disturbance (0:normal (no impairment), 1: mildly impaired function, but no assistance required, 2: moderately impaired function, but needs assistance for certain parts of task, 3: severely impaired function to degree that assistance is needed for all parts of the task, 4: unable to perform function). Total score was derived as sum of individual items; ranged from 0 to 16. Higher score indicated worst outcome. A negative change in score showed improvement.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE3
Target enrollment
299 participants
Primary outcome timeframe
Randomization Baseline; Week 48
Results posted on
2025-11-04
Participant Flow
The study was conducted at 23 sites in 2 countries (21 sites in the US and 2 sites in China).
A total of 299 participants were enrolled, of which 218 participants were randomized in a 1:1: ratio to receive troriluzole or placebo. Study consisted of double-blind randomization phase (up to Week 48) and open-label extension phase (up to Week 192). The study is ongoing, and results are reported only for randomization phase (up to Week 48).
Participant milestones
| Measure |
Troriluzole
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
Open-label extension (OLE) phase (OLE Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 200 mg capsules orally once daily for 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.
|
Placebo
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
OLE phase (Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 140 mg capsules orally once daily for the first 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.
|
|---|---|---|
|
Overall Study
STARTED
|
109
|
109
|
|
Overall Study
Treated
|
108
|
109
|
|
Overall Study
COMPLETED
|
96
|
96
|
|
Overall Study
NOT COMPLETED
|
13
|
13
|
Reasons for withdrawal
| Measure |
Troriluzole
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
Open-label extension (OLE) phase (OLE Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 200 mg capsules orally once daily for 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.
|
Placebo
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
OLE phase (Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 140 mg capsules orally once daily for the first 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.
|
|---|---|---|
|
Overall Study
Adverse Event
|
3
|
5
|
|
Overall Study
Withdrawal by Subject
|
6
|
3
|
|
Overall Study
Investigators decision
|
1
|
2
|
|
Overall Study
Lost to Follow-up
|
1
|
2
|
|
Overall Study
Death
|
1
|
1
|
|
Overall Study
Randomized but not treated
|
1
|
0
|
Baseline Characteristics
Troriluzole in Adult Participants With Spinocerebellar Ataxia
Baseline characteristics by cohort
| Measure |
Troriluzole
n=108 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
Open-label extension (OLE) phase (OLE Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 200 mg capsules orally once daily for 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.
|
Placebo
n=109 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
OLE phase (Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 140 mg capsules orally once daily for the first 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.
|
Total
n=217 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
47.7 years
STANDARD_DEVIATION 13.16 • n=15 Participants
|
47.6 years
STANDARD_DEVIATION 12.48 • n=161 Participants
|
47.6 years
STANDARD_DEVIATION 12.79 • n=100 Participants
|
|
Sex: Female, Male
Female
|
50 Participants
n=15 Participants
|
56 Participants
n=161 Participants
|
106 Participants
n=100 Participants
|
|
Sex: Female, Male
Male
|
58 Participants
n=15 Participants
|
53 Participants
n=161 Participants
|
111 Participants
n=100 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
13 Participants
n=15 Participants
|
11 Participants
n=161 Participants
|
24 Participants
n=100 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
95 Participants
n=15 Participants
|
98 Participants
n=161 Participants
|
193 Participants
n=100 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
1 Participants
n=100 Participants
|
|
Race (NIH/OMB)
Asian
|
19 Participants
n=15 Participants
|
21 Participants
n=161 Participants
|
40 Participants
n=100 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
|
Race (NIH/OMB)
Black or African American
|
12 Participants
n=15 Participants
|
14 Participants
n=161 Participants
|
26 Participants
n=100 Participants
|
|
Race (NIH/OMB)
White
|
74 Participants
n=15 Participants
|
74 Participants
n=161 Participants
|
148 Participants
n=100 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
2 Participants
n=100 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=15 Participants
|
0 Participants
n=161 Participants
|
0 Participants
n=100 Participants
|
|
Number of Participants with Spinocerebellar ataxia (SCA) Genotype
SCA1
|
15 participants
n=15 Participants
|
11 participants
n=161 Participants
|
26 participants
n=100 Participants
|
|
Number of Participants with Spinocerebellar ataxia (SCA) Genotype
SCA2
|
32 participants
n=15 Participants
|
35 participants
n=161 Participants
|
67 participants
n=100 Participants
|
|
Number of Participants with Spinocerebellar ataxia (SCA) Genotype
SCA3
|
44 participants
n=15 Participants
|
45 participants
n=161 Participants
|
89 participants
n=100 Participants
|
|
Number of Participants with Spinocerebellar ataxia (SCA) Genotype
SCA6
|
6 participants
n=15 Participants
|
6 participants
n=161 Participants
|
12 participants
n=100 Participants
|
|
Number of Participants with Spinocerebellar ataxia (SCA) Genotype
SCA7
|
5 participants
n=15 Participants
|
5 participants
n=161 Participants
|
10 participants
n=100 Participants
|
|
Number of Participants with Spinocerebellar ataxia (SCA) Genotype
SCA8
|
3 participants
n=15 Participants
|
2 participants
n=161 Participants
|
5 participants
n=100 Participants
|
|
Number of Participants with Spinocerebellar ataxia (SCA) Genotype
SCA10
|
3 participants
n=15 Participants
|
5 participants
n=161 Participants
|
8 participants
n=100 Participants
|
PRIMARY outcome
Timeframe: Randomization Baseline; Week 48Population: The modified intent-to-treat (mITT) analysis set in randomization phase included randomized participants who received at least one dose of double-blind study medication (troriluzole or placebo) during the randomization phase and provided a non-missing baseline measurement and at least one non-missing post-baseline efficacy measurement during the randomization phase. Participants with available data were included. Pre-specified randomization strata by SCA genotype.
The f-SARA was a 4-item performance based scale: 1-gait, 2-stance, 3-sitting, 4-speech disturbance (0:normal (no impairment), 1: mildly impaired function, but no assistance required, 2: moderately impaired function, but needs assistance for certain parts of task, 3: severely impaired function to degree that assistance is needed for all parts of the task, 4: unable to perform function). Total score was derived as sum of individual items; ranged from 0 to 16. Higher score indicated worst outcome. A negative change in score showed improvement.
Outcome measures
| Measure |
Troriluzole
n=94 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=95 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Change From Baseline in the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) Total Score at Week 48 in SCA Participants
All SCA
|
0.20 score on a scale
Standard Error 0.19
|
0.27 score on a scale
Standard Error 0.18
|
|
Randomization Phase: Change From Baseline in the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) Total Score at Week 48 in SCA Participants
SCA3
|
-0.03 score on a scale
Standard Error 0.20
|
0.53 score on a scale
Standard Error 0.19
|
SECONDARY outcome
Timeframe: Randomization Baseline, Week 48Population: Participants in mITT analysis set with available data were analyzed.
PIFAS was a 17-item instrument designed to assess the level of functional disability. The scale was rater administered and the items were selected to encompass domains of relevance to SCA (i.e., mobility, speech/swallowing, and fatigue), and to capture function and activities of daily living within these domains. Statements were rated on a 5-point Likert scale ranging from "0" reflecting "not at all" to "4" reflecting "very much." The total score was derived as the sum of the individual items, ranged between 0 to 68. Higher score indicated a worst outcome. A negative change in score showed improvement.
Outcome measures
| Measure |
Troriluzole
n=94 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=90 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Change From Baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) Total Score at Week 48 in SCA Participants
SCA3
|
0.56 score on a scale
Standard Error 1.65
|
2.96 score on a scale
Standard Error 1.67
|
|
Randomization Phase: Change From Baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) Total Score at Week 48 in SCA Participants
All SCA
|
-0.93 score on a scale
Standard Error 1.49
|
1.03 score on a scale
Standard Error 1.43
|
SECONDARY outcome
Timeframe: Randomization Baseline, Week 48Population: Participants in mITT analysis set with available data were analyzed.
FARS was a scale consisting of combined timed measures of performance, FUNC (0-6, higher score indicated worst outcome), ADL with paramount neurologic examination (0-36, higher score indicated worst outcome), FARSn (0-125, higher score indicated worst outcome). Total score was sum of these 3 subscales: 0-167, higher score indicated worst outcome. For this outcome measure, only FARS-ADL sub-scale was evaluated. It was multicomponent scale designed to assess neurological domains affected in Friedreich's Ataxia, another hereditary cerebellar ataxia disorder. This subscale had been validated in Friedreich's ataxia, was a measure of functional disability, and correlated with SARA total scores. It assessed 9 areas of ADL with response categories rated on 5-point scale with "0" reflecting "normal" and "4" reflecting inability to perform specific function. Total score was derived as sum of individual items, ranging 0-36, higher score indicated worst outcome. FARS-ADL was rater administered.
Outcome measures
| Measure |
Troriluzole
n=94 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=95 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Total Score at Week 48 in SCA Participants
All SCA
|
0.453 score on a scale
Standard Error 0.502
|
-0.017 score on a scale
Standard Error 0.480
|
|
Randomization Phase: Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Total Score at Week 48 in SCA Participants
SCA3
|
0.918 score on a scale
Standard Error 0.523
|
1.130 score on a scale
Standard Error 0.506
|
SECONDARY outcome
Timeframe: Randomization Baseline, Week 48Population: Participants in mITT analysis set with available data were analyzed.
FARS was a scale consisting of combined timed measures of performance, FUNC (0-6, higher score indicated worst outcome), ADL with paramount neurologic examination (0-36, higher score indicated worst outcome), FARSn (0-125, higher score indicated worst outcome). The total score was a sum of these 3 subscales; 0-167, higher score indicated worst outcome. For this outcome measure, only the FARS-FUNC sub-scale was evaluated. FARS-FUNC was a subscale of the FARS designed to provide functional staging for ataxia in which clinicians were asked to assess function on a 6-stage staging system, with "0" reflecting "normal" and "6" reflecting a stage in participant was "total disabled".
Outcome measures
| Measure |
Troriluzole
n=93 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=94 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Change From Baseline in Functional Staging for Ataxia Scale From the Friedreich's Ataxia Rating Scale (FARS-FUNC) Total Score at Week 48 in SCA Participants
All SCA
|
0.266 score on a scale
Standard Error 0.075
|
0.226 score on a scale
Standard Error 0.072
|
|
Randomization Phase: Change From Baseline in Functional Staging for Ataxia Scale From the Friedreich's Ataxia Rating Scale (FARS-FUNC) Total Score at Week 48 in SCA Participants
SCA3
|
0.216 score on a scale
Standard Error 0.079
|
0.328 score on a scale
Standard Error 0.077
|
SECONDARY outcome
Timeframe: From first dose of study drug to Week 48 plus 30 days (maximum duration: 52 weeks)Population: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo).
An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a participant that did not necessarily had a causal relationship with this treatment. An SAE was defined as any AE that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect in the offspring of the participant or was considered another important medical event. Treatment-emergent AEs (TEAEs) in the randomization phase included any AE with an onset date on or after the first day of double-blind study drug and up to the first day of open-label study drug in the extension phase (for participants entering the extension phase) or last day of the randomization phase study drug + 30 days.
Outcome measures
| Measure |
Troriluzole
n=108 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=109 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (TESAEs); and AEs Leading to Treatment Discontinuation
TEAEs
|
87 participants
|
92 participants
|
|
Randomization Phase: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (TESAEs); and AEs Leading to Treatment Discontinuation
TESAEs
|
6 participants
|
8 participants
|
|
Randomization Phase: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (TESAEs); and AEs Leading to Treatment Discontinuation
AEs Leading to Treatment Discontinuation
|
5 participants
|
5 participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Randomization Baseline; Week 48Population: Participants in mITT analysis set with available data were analyzed.
The f-SARA was a 4-item performance based scale: 1-gait, 2-stance, 3-sitting, 4-speech disturbance (0:normal (no impairment), 1:mildly impaired function, but no assistance required, 2:moderately impaired function, but needs assistance for certain parts of task, 3: severely impaired function to degree that assistance is needed for all parts of the task, 4:unable to perform function). Total score was derived as sum of individual items; ranged from 0 to 16. Higher score indicated worst outcome. A negative change in score shows improvement.
Outcome measures
| Measure |
Troriluzole
n=38 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=40 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Change From Baseline in the f-SARA Total Score at Week 48 in SCA3 Participants
|
-0.03 score on a scale
Standard Error 0.20
|
0.53 score on a scale
Standard Error 0.19
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Randomization Baseline, Week 48Population: Participants in mITT analysis set with available data were analyzed.
PIFAS was a 17-item instrument designed to assess the level of functional disability. The scale is rater administered and the items were selected to encompass domains of relevance to SCA (i.e., mobility, speech/swallowing, and fatigue), and to capture function and activities of daily living within these domains. Statements are rated on a 5-point Likert scale ranging from "0" reflecting "not at all" to "4" reflecting "very much." The total score is derived as the sum of the individual items, ranged between 0 to 68. Higher score indicated a worst outcome. A negative change in score showed improvement.
Outcome measures
| Measure |
Troriluzole
n=38 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=37 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Change From Baseline in PIFAS Total Score at Week 48 in SCA3 Participants
|
0.56 score on a scale
Standard Error 1.65
|
2.96 score on a scale
Standard Error 1.67
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Randomization Baseline, Week 48Population: Participants in mITT analysis set with available data were analyzed.
FARS was a scale consisting of combined timed measures of performance, FUNC (0-6, higher score indicated worst outcome), ADL with paramount neurologic examination (0-36, higher score indicated worst outcome), FARSn (0-125, higher score indicated worst outcome). Total score was sum of these 3 subscales: 0-167, higher score indicated worst outcome. For this outcome measure, only FARS-ADL sub-scale was evaluated. It was multicomponent scale designed to assess neurological domains affected in Friedreich's Ataxia, another hereditary cerebellar ataxia disorder. This subscale had been validated in Friedreich's ataxia, was a measure of functional disability, and correlated with SARA total scores. It assessed 9 areas of ADL with response categories rated on 5-point scale with "0" reflecting "normal" and "4" reflecting inability to perform specific function. Total score was derived as sum of individual items, ranging 0-36, higher score indicated worst outcome. FARS-ADL was rater administered.
Outcome measures
| Measure |
Troriluzole
n=37 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=39 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Change From Baseline in FARS-ADL Total Score at Week 48 in SCA3 Participants
|
0.918 score on a scale
Standard Error 0.523
|
1.130 score on a scale
Standard Error 0.506
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Randomization Baseline, Week 48Population: Participants in mITT analysis set with available data were analyzed.
FARS was a scale consisting of combined timed measures of performance, FUNC (0-6, higher score indicated worst outcome), ADL with paramount neurologic examination (0-36, higher score indicated worst outcome), FARSn (0-125, higher score indicated worst outcome). The total score was a sum of these 3 subscales; 0-167, higher score indicated worst outcome. For this outcome measure, only the FARS-FUNC sub-scale was evaluated. FARS-FUNC was a subscale of the FARS designed to provide functional staging for ataxia in which clinicians were asked to assess function on a 6-stage staging system, with "0" reflecting "normal" and "6" reflecting a stage in participant was "total disabled".
Outcome measures
| Measure |
Troriluzole
n=37 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=39 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Randomization Phase: Change From Baseline in FARS-FUNC Total Score at Week 48 in SCA3 Participants
|
0.216 score on a scale
Standard Error 0.079
|
0.328 score on a scale
Standard Error 0.077
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Randomization Baseline; Week 48Population: Participants in mITT analysis set with available data were analyzed.
The CGI-I was a 7-point scale that required the clinician to assess how much the participant's illness had improved or worsened relative to the baseline visit and it was rated on a 7 point scale: 1= "Very much improved", 2= "Much improved", 3= "Minimally improved", 4= "No change", 5= "Minimally worse", 6= "Much worse" and 7= "Very much worse". Higher scores indicated greater impairment.
Outcome measures
| Measure |
Troriluzole
n=35 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=38 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Change From Baseline in Clinical Global Impression - Global Improvement Scale (CGI-I) in SCA3 Participants
|
4.14 score on a scale
Standard Error 0.17
|
4.68 score on a scale
Standard Error 0.17
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Randomization Baseline, Week 48Population: All randomized participants who received at least one dose of study medication during the randomization phase.
An AE of Fall was recorded if there was a worsening of frequency of falls or if a fall was associated with an injury.
Outcome measures
| Measure |
Troriluzole
n=108 Participants
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48 week duration of the double-blind randomization phase.
|
Placebo
n=109 Participants
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Falls Events in Troriluzole Compared to Placebo for All SCA Participants
|
20 Number of events
|
42 Number of events
|
Adverse Events
Troriluzole
Serious events: 6 serious events
Other events: 54 other events
Deaths: 1 deaths
Placebo
Serious events: 8 serious events
Other events: 58 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Troriluzole
n=108 participants at risk
Randomization phase (Randomization through Week 48) Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48-week duration of the double-blind randomization phase.
|
Placebo
n=109 participants at risk
Randomization phase (Randomization through Week 48) Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Fall
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Injury, poisoning and procedural complications
Pulmonary contusion
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Investigations
Human chorionic gonadotropin positive
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Investigations
Liver function test increased
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Infections and infestations
COVID-19
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
1.8%
2/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Infections and infestations
Abscess
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Infections and infestations
Pyelonephritis
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
General disorders
Pneumatosis
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
General disorders
Asthenia
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Hepatobiliary disorders
Cholecystitis
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Vascular disorders
Haematocoele
|
0.93%
1/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.00%
0/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Psychiatric disorders
Suicide attempt
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
1.8%
2/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Psychiatric disorders
Major depression
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Renal and urinary disorders
Ureterolithiasis
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Nervous system disorders
Syncope
|
0.00%
0/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
Other adverse events
| Measure |
Troriluzole
n=108 participants at risk
Randomization phase (Randomization through Week 48) Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48-week duration of the double-blind randomization phase.
|
Placebo
n=109 participants at risk
Randomization phase (Randomization through Week 48) Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
|
|---|---|---|
|
Nervous system disorders
Headache
|
12.0%
13/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
7.3%
8/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Nervous system disorders
Dizziness
|
8.3%
9/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
10.1%
11/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Injury, poisoning and procedural complications
Fall
|
12.0%
13/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
22.0%
24/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Injury, poisoning and procedural complications
Contusion
|
6.5%
7/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
6.4%
7/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Infections and infestations
Covid-19
|
5.6%
6/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
2.8%
3/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
4.6%
5/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
6.4%
7/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Infections and infestations
Urinary Tract Infection
|
4.6%
5/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
6.4%
7/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
General disorders
Fatigue
|
9.3%
10/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
8.3%
9/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
5.6%
6/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
8.3%
9/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
3.7%
4/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
6.4%
7/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
|
Gastrointestinal disorders
Nausea
|
6.5%
7/108 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
0.92%
1/109 • For Adverse events (AEs): From first dose of study drug up to Week 48 plus 30 days (maximum duration: 52 weeks) For All-cause mortality: From randomization up to Week 48 plus 30 days (maximum duration: 52 weeks)
For AEs: The treated analysis set (TAS) included all randomized/enrolled participants who received at least one dose of study medication (blinded or open label troriluzole, or blinded placebo) For All-cause mortality: All randomized participants.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER