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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-06-26

Study Completion Date

2022-08-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Eteplirsen

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

Group Type EXPERIMENTAL

Eteplirsen

Intervention Type DRUG

Eteplirsen IV infusion once weekly.

Interventions

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Eteplirsen

Eteplirsen IV infusion once weekly.

Intervention Type DRUG

Other Intervention Names

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AVI-4658 EXONDYS 51 EXONDYS

Eligibility Criteria

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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.

Minimum Eligible Age

2 Years

Maximum Eligible Age

5 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No