Phase 3
Completed N=109
Study of Eteplirsen in DMD Patients
Duchenne Muscular Dystrophy (DMD)
Source: ClinicalTrials.gov NCT02255552 ↗
Enrolled (actual)
109
Serious AEs
11.9%
Results posted
Jul 2020
Primary outcomePrimary: Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96 — -117.91; -133.56 meters
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96 |
-117.91; -133.56 | — |
| SECONDARY Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96 |
0.516 | — |
| SECONDARY Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96 |
33; 3 | — |
| SECONDARY Number of Participants Who Lost Ambulation (LOA) by Week 96 |
12; 1 | — |
| SECONDARY Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96 |
-3.413; -2.461 | — |
| SECONDARY Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96 |
-7.23; -8.44 | — |
| SECONDARY Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 96 |
0.030 | — |
Eligibility Criteria
Inclusion Criteria
- Male 7-16 years old
- Diagnosed with DMD, genotypically confirmed
- Stable dose of corticosteroids for at least 24 weeks
- Have intact right and left alternative upper muscle groups
- Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)
- Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%
Exclusion Criteria
- Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months
- Participation in any other DMD interventional clinical study within 12 weeks
- Major surgery within 3 months
- Presence of other clinically significant illness
- Major change in the physical therapy regime within 3 months
Other inclusion/exclusion criteria apply.
Data sourced from ClinicalTrials.gov (NCT02255552). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.