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

Sarepta Therapeutics

Status and phase: Terminated; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Eteplirsen

Study type: Interventional

Funder types: Industry

Identifiers

NCT03985878

2019-000337-39 (EudraCT Number)

4658-102-OLE

Details and patient eligibility

About

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.

Enrollment: 15 patients

Sex: Male

Ages: 2 to 5 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

Eteplirsen

Experimental group

Description:

Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks.

Treatment:

Drug: Eteplirsen