Phase 2
Completed N=33
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Source: ClinicalTrials.gov NCT02420379 ↗
Enrolled (actual)
33
Serious AEs
12.1%
Results posted
Jul 2020
Primary outcomePrimary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation — 26; 5; 4; 0 Participants
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.
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 | — |
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. Informational only — not medical advice.