Trial Outcomes & Findings for A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) (NCT NCT03783923)
NCT ID: NCT03783923
Last Updated: 2022-06-27
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
11 participants
Primary outcome timeframe
Baseline, Week 26
Results posted on
2022-06-27
Participant Flow
Participants enrolled under protocol v3.0 were 1:1 randomized to get placebo or deflazacort. After protocol v4.0, the study became an open-label study and all participants received deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety and efficacy summaries.
Participant milestones
| Measure |
Deflazacort
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.
|
Placebo
Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.
|
|---|---|---|
|
Placebo-Controlled Period (26 Weeks)
STARTED
|
5
|
6
|
|
Placebo-Controlled Period (26 Weeks)
Safety Population
|
5
|
2
|
|
Placebo-Controlled Period (26 Weeks)
COMPLETED
|
1
|
2
|
|
Placebo-Controlled Period (26 Weeks)
NOT COMPLETED
|
4
|
4
|
|
Open-Label Extension (26 Weeks)
STARTED
|
1
|
2
|
|
Open-Label Extension (26 Weeks)
COMPLETED
|
0
|
0
|
|
Open-Label Extension (26 Weeks)
NOT COMPLETED
|
1
|
2
|
Reasons for withdrawal
| Measure |
Deflazacort
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.
|
Placebo
Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.
|
|---|---|---|
|
Placebo-Controlled Period (26 Weeks)
Study Terminated
|
3
|
2
|
|
Placebo-Controlled Period (26 Weeks)
Adverse Event
|
1
|
0
|
|
Placebo-Controlled Period (26 Weeks)
Withdrawal by Subject
|
0
|
2
|
|
Open-Label Extension (26 Weeks)
Withdrawal by Subject
|
1
|
1
|
|
Open-Label Extension (26 Weeks)
Study Terminated
|
0
|
1
|
Baseline Characteristics
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Baseline characteristics by cohort
| Measure |
Deflazacort
n=5 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
Placebo
n=6 Participants
Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.
|
Total
n=11 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 26Population: Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.
Outcome measures
| Measure |
Deflazacort
n=7 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort
Baseline
|
5.476 seconds
Standard Deviation 2.0178
|
|
Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort
Change at Week 26
|
-0.200 seconds
|
SECONDARY outcome
Timeframe: Baseline, Week 26Population: Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 26Population: Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.
Outcome measures
| Measure |
Deflazacort
n=7 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort
Baseline
|
135.4 meters
Standard Deviation 26.75
|
|
Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort
Change at Week 26
|
2.0 meters
|
SECONDARY outcome
Timeframe: Baseline, Week 26Population: Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.
Outcome measures
| Measure |
Deflazacort
n=7 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort
Baseline
|
11.93 seconds
Standard Deviation 4.743
|
|
Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort
Change at Week 26
|
9.70 seconds
|
SECONDARY outcome
Timeframe: Baseline, Week 26Population: Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.
Outcome measures
| Measure |
Deflazacort
n=7 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort
Change at Week 26
|
0.10 seconds
|
|
Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort
Baseline
|
3.66 seconds
Standard Deviation 1.707
|
SECONDARY outcome
Timeframe: Baseline, Week 26Population: Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.
Outcome measures
| Measure |
Deflazacort
n=7 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort
Baseline
|
8.53 seconds
Standard Deviation 1.897
|
|
Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort
Change at Week 26
|
-0.40 seconds
|
SECONDARY outcome
Timeframe: Baseline, Week 26Population: Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 26Population: Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 26Population: Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline up to Week 52Population: Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo during the study were not included for safety analysis.
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.
Outcome measures
| Measure |
Deflazacort
n=7 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Number of Participants With Adverse Events (AEs)
|
5 Participants
|
SECONDARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13Population: The pharmacokinetic (PK) population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Deflazacort
n=4 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Baseline
|
401.1 nanograms (ng)*hour (hr)/milliliter (mL)
Standard Deviation 139.62
|
|
Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Week 13
|
365.6 nanograms (ng)*hour (hr)/milliliter (mL)
Standard Deviation NA
Due to concentration below the limit of quantitation (BLQ) of 0.50 ng/mL, data not calculated.
|
|
Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 1
|
418.2 nanograms (ng)*hour (hr)/milliliter (mL)
Standard Deviation 58.569
|
|
Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 13
|
515.3 nanograms (ng)*hour (hr)/milliliter (mL)
Standard Deviation NA
Due to concentration BLQ of 0.50 ng/mL, data not calculated.
|
SECONDARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13Population: The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Deflazacort
n=4 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Baseline
|
423.6 ng*hr/mL
Standard Deviation 150.16
|
|
Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 1
|
520.3 ng*hr/mL
Standard Deviation 31.508
|
SECONDARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13Population: The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Deflazacort
n=4 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Baseline
|
184.8 ng/mL
Standard Deviation 49.054
|
|
Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Week 13
|
171.0 ng/mL
Standard Deviation NA
Due to concentration BLQ of 0.50 ng/mL, data not calculated.
|
|
Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 1
|
135.8 ng/mL
Standard Deviation 29.205
|
|
Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 13
|
162.0 ng/mL
Standard Deviation NA
Due to concentration BLQ of 0.50 ng/mL, data not calculated.
|
SECONDARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13Population: The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Deflazacort
n=4 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Baseline
|
0.992 hr
Interval 0.5 to 1.0
|
|
Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Week 13
|
0.525 hr
Interval 0.5 to 0.55
|
|
Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 1
|
1.000 hr
Interval 1.0 to 1.9
|
|
Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 13
|
1.550 hr
Interval 1.1 to 2.0
|
SECONDARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13Population: The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.
Outcome measures
| Measure |
Deflazacort
n=4 Participants
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 13
|
1.95 hr
|
|
Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Baseline
|
1.174 hr
Standard Deviation 0.0849
|
|
Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
21-desacetyl deflazacort: Week 13
|
1.235 hr
Standard Deviation NA
Due to concentration BLQ of 0.50 ng/mL, data not calculated.
|
|
Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
6β-hydroxy-21-desacetyl deflazacort: Week 1
|
2.358 hr
Standard Deviation 0.5825
|
Adverse Events
Deflazacort
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Deflazacort
n=7 participants at risk
Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
|
|---|---|
|
Endocrine disorders
Cushingoid
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Gastrointestinal disorders
Dyspepsia
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
General disorders
Fatigue
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Infections and infestations
Influenza
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Infections and infestations
Upper respiratory tract infection
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Injury, poisoning and procedural complications
Contusion
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Investigations
Weight increased
|
28.6%
2/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Metabolism and nutrition disorders
Increased appetite
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Musculoskeletal and connective tissue disorders
Muscle tightness
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Nervous system disorders
Headache
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Nervous system disorders
Hypoaesthesia
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Nervous system disorders
Tension headache
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Nervous system disorders
Visual field defect
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Psychiatric disorders
Apathy
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Psychiatric disorders
Depressed mood
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Psychiatric disorders
Sleep disorder
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Renal and urinary disorders
Pollakiuria
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Reproductive system and breast disorders
Amenorrhoea
|
20.0%
1/5 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Reproductive system and breast disorders
Menstruation irregular
|
20.0%
1/5 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Skin and subcutaneous tissue disorders
Acne
|
28.6%
2/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Skin and subcutaneous tissue disorders
Abnormal hair growth
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Skin and subcutaneous tissue disorders
Hirsutism
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
|
Injury, poisoning and procedural complications
Fall
|
14.3%
1/7 • Baseline up to Week 52
Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
|
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