Phase 2
N=33
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Bottom Line
View on ClinicalTrials.gov: NCT02420379 ↗Enrolled (actual)
33
Serious AEs
12.1%
Results posted
Jul 2020
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation — 26; 5; 4; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- eteplirsen (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- Male
- Sponsor
- Sarepta Therapeutics, Inc.
- Primary completion
- Dec 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation |
26; 5; 4; 0; 0; 0 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs |
1; 0; 1; 0; 1; 0 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs |
11; 1; 1; 0; 1; 0 | — |
| PRIMARY Number of Participants With at Least One Abnormal Physical Examination Finding |
23; 4 | — |
| PRIMARY Number of Participants With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs |
1; 0 | — |
| PRIMARY Number of Participants With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs |
1; 0 | — |
| SECONDARY Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96 |
0.102; 0.321 | — |
| SECONDARY Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96 |
0.004; 0.015 | — |
Summary
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Eligibility Criteria
Inclusion Criteria
- Male 4-6 years of age.
- Diagnosis of DMD, genotypically confirmed.
- Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks.
- Intact right and left biceps muscles or two alternative upper arm muscle groups.
- Parent that is willing to provide consent and comply with study procedures.
Exclusion Criteria
- Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
- Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months.
- Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities.
- Presence of other clinically significant illness.
Data sourced from ClinicalTrials.gov (NCT02420379). 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.