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Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

NCT02420379 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 33

Last updated 2021-01-25

Study results available
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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.

Conditions

  • Duchenne Muscular Dystrophy (DMD)

Interventions

DRUG

eteplirsen

Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks.

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
6 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-06-30
Primary Completion
2018-12-17
Completion
2018-12-17
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT02420379 on ClinicalTrials.gov