Trial Outcomes & Findings for Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (NCT NCT03907072)
NCT ID: NCT03907072
Last Updated: 2021-05-20
Results Overview
US/other regions (as applicable)
Recruitment status
TERMINATED
Study phase
PHASE2/PHASE3
Target enrollment
6 participants
Primary outcome timeframe
Day 1 to Week 12, Week 22, or Week 46
Results posted on
2021-05-20
Participant Flow
Participant milestones
| Measure |
WVE-210201 (3 mg/kg)
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
WVE-210201 (4.5 mg/kg)
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
Placebo
Placebo: Buffered saline solution
|
|---|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
2
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
2
|
Reasons for withdrawal
| Measure |
WVE-210201 (3 mg/kg)
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
WVE-210201 (4.5 mg/kg)
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
Placebo
Placebo: Buffered saline solution
|
|---|---|---|---|
|
Overall Study
Study Terminated by Sponsor
|
2
|
2
|
2
|
Baseline Characteristics
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
WVE-210201 (3 mg/kg)
n=2 Participants
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
WVE-210201 (4.5 mg/kg)
n=2 Participants
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
Placebo
n=2 Participants
Placebo: Buffered saline solution
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
6 Participants
n=31 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Age, Continuous
|
6.5 Years
n=39 Participants
|
9 Years
n=41 Participants
|
7 Years
n=35 Participants
|
7.5 Years
n=31 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
6 Participants
n=31 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
5 Participants
n=31 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Region of Enrollment
Sweden
|
0 participants
n=39 Participants
|
1 participants
n=41 Participants
|
0 participants
n=35 Participants
|
1 participants
n=31 Participants
|
|
Region of Enrollment
Belgium
|
1 participants
n=39 Participants
|
0 participants
n=41 Participants
|
0 participants
n=35 Participants
|
1 participants
n=31 Participants
|
|
Region of Enrollment
Italy
|
0 participants
n=39 Participants
|
0 participants
n=41 Participants
|
2 participants
n=35 Participants
|
2 participants
n=31 Participants
|
|
Region of Enrollment
France
|
1 participants
n=39 Participants
|
1 participants
n=41 Participants
|
0 participants
n=35 Participants
|
2 participants
n=31 Participants
|
PRIMARY outcome
Timeframe: Day 1 to Week 12, Week 22, or Week 46Population: No statistical analysis has been performed due to early study termination
US/other regions (as applicable)
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Day 1 through Week 48Population: No statistical analysis has been performed due to early study termination.
European Union (EU)/other regions (as applicable)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through Week 48Population: No statistical analysis has been performed due to early study termination.
US/other regions (as applicable)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 to Week 12, Week 22, or Week 46Population: No statistical analysis has been performed due to early study termination.
European Union (EU)/other regions (as applicable)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through Week 48Population: No statistical analysis has been performed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through Week 48Population: No statistical analysis has been performed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through Week 48Population: No statistical analysis has been performed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through Week 48Population: No statistical analysis has been performed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through Week 48Population: No statistical analysis has been performed due to early study termination.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through Week 96Population: No statistical analysis has been performed due to early study termination.
Long-term evaluation, open label from Week 48 through Week 96
Outcome measures
Outcome data not reported
Adverse Events
WVE-210201 (3 mg/kg)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
WVE-210201 (4.5 mg/kg)
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
WVE-210201 (3 mg/kg)
n=2 participants at risk
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
WVE-210201 (4.5 mg/kg)
n=2 participants at risk
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
Placebo
n=2 participants at risk
Placebo: Buffered saline solution
|
|---|---|---|---|
|
Cardiac disorders
Cardiac Disorder
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
Other adverse events
| Measure |
WVE-210201 (3 mg/kg)
n=2 participants at risk
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
WVE-210201 (4.5 mg/kg)
n=2 participants at risk
WVE-210201 (suvodirsen): WVE-210201 is a stereopure antisense oligonucleotide (ASO)
|
Placebo
n=2 participants at risk
Placebo: Buffered saline solution
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Bone contusion
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
|
Investigations
C-reactive protein increased
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
|
Nervous system disorders
Headache
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 4 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
|
General disorders
Pyrexia
|
50.0%
1/2 • Number of events 3 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
100.0%
2/2 • Number of events 4 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 4 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
|
Infections and infestations
Nasopharyngitis
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
|
Infections and infestations
Oral herpes
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
50.0%
1/2 • Number of events 1 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
0.00%
0/2 • Study enrollment through early study termination by Sponsor (maximum of 11 weeks).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place