Phase 2
Completed N=15
Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
Source: ClinicalTrials.gov NCT03218995 ↗Enrolled (actual)
15
Serious AEs
6.7%
Results posted
Dec 2021
Primary outcomePrimary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug — 9; 6; 0; 1 Participants
Summary
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug |
9; 6; 0; 1; 0; 0 | — |
| PRIMARY Number of Participants With at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality |
9; 6 | — |
| PRIMARY Number of Participants With at Least 1 Markedly Abnormal Vital Sign |
9; 6 | — |
| PRIMARY Abnormal Changes From Baseline or Worsening of Physical Examination Findings |
— | — |
| PRIMARY Number of Participants With at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO) |
4; 5 | — |
| SECONDARY Maximum Plasma Concentration (Cmax) of Eteplirsen |
9.67; 4.22; 46.5; 17.2; 63.3; 85.0 | — |
| SECONDARY Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen |
0.58; 0.58; 0.58; 0.72; 0.78; 0.73 | — |
| SECONDARY Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma |
13.8; 6.13; 56.1; 27.8; 92.1; 81.4 | — |
| SECONDARY Amount of Drug Eliminated in Urine |
7720; 1430; 56000; 28700; 102000; 65600 | — |
Eligibility Criteria
Inclusion Criteria
- Male between 6 months to 48 months of age (inclusive)
- Diagnosis of DMD with a deletion mutation amenable to exon 51 skipping
- Parent(s) or legal guardian(s) who is willing to provide written informed consent
Exclusion Criteria
- Received treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing
- Received previous or current treatment with any experimental treatment
- Clinically significant illness other than DMD
- Clinically significant laboratory abnormality
- Any other condition that could interfere with the participation in the study.
Data sourced from ClinicalTrials.gov (NCT03218995). 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.