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Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping

NCT03218995 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2021-12-09

Study results available
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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.

Conditions

Interventions

DRUG

Eteplirsen

Infusion for intravenous use.

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
48 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-08-16
Primary Completion
2021-03-10
Completion
2021-03-10
FDA Drug
Yes

Countries

  • Belgium
  • France
  • Italy
  • United Kingdom

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03218995 on ClinicalTrials.gov