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

Phase 2TerminatedNCT03985878

Sarepta Therapeutics, Inc.15 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

EteplirsenDRUG

Eteplirsen IV infusion once weekly.

Eligibility

Sex: MALEMin age: 2 YearsMax age: 5 Years

Medical Language ↔ Plain English

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.

Locations (4)

UZ-Gent

Ghent, Belgium

Hopital Trousseau, Bâtiment lemariey

Paris, France

Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile

Rome, Italy

UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre

London, England, United Kingdom

Outcomes

Primary Outcomes

Number of Participants Experiencing Adverse Events (AEs)

A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.

Time frame: Up to 162 weeks

Number of Participants Experiencing Death Due to Adverse Events

A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.

Time frame: Up to 162 weeks

Number of Participants Experiencing Adverse Events of Special Interest (AESIs)

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.

Time frame: Up to 162 weeks

Data from ClinicalTrials.gov

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