A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)

July 25, 2023 updated by: Sarepta Therapeutics, Inc.

An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102

The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have successfully completed the 96-week eteplirsen Study 4658-102.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Gent, Belgium, 9000
        • UZ-Gent
  • France
      • Paris, France, 75021
        • Hopital Trousseau, Bâtiment lemariey
  • Italy
      • Rome, Italy, 00168
        • Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile
  • United Kingdom
    • England
      • London, England, United Kingdom, WC1N 1EH
        • UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 5 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Participant successfully completes 96 weeks of treatment in Study 4658-102.

Exclusion Criteria:

  • Participant has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the participant, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.

Other inclusion/exclusion criteria apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Eteplirsen
Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks.
Drug: Eteplirsen
Eteplirsen IV infusion once weekly.
Other Names:
  • AVI-4658
  • EXONDYS 51
  • EXONDYS

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants Experiencing Adverse Events (AEs)
Time Frame: Up to 162 weeks
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Up to 162 weeks
Number of Participants Experiencing Death Due to Adverse Events
Time Frame: Up to 162 weeks
A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.
Up to 162 weeks
Number of Participants Experiencing Adverse Events of Special Interest (AESIs)
Time Frame: Up to 162 weeks
AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Up to 162 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Investigators

  • Study Director: Medical Director, Sarepta Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 26, 2019

Primary Completion (Actual)

August 31, 2022

Study Completion (Actual)

August 31, 2022

Study Registration Dates

First Submitted

June 11, 2019

First Submitted That Met QC Criteria

June 11, 2019

First Posted (Actual)

June 14, 2019

Study Record Updates

Last Update Posted (Actual)

August 18, 2023

Last Update Submitted That Met QC Criteria

July 25, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 4658-102-OLE
  • 2019-000337-39 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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