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Study of Eteplirsen in DMD Patients

NCT02255552 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 109

Last updated 2021-01-25

Study results available
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Summary

The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).

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, followed by a safety extension (not to exceed 48 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
7 Years
Max Age
16 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-11-17
Primary Completion
2019-06-14
Completion
2019-06-14

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 NCT02255552 on ClinicalTrials.gov