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 Overview
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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.

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

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: Eteplirsen


Participation Criteria
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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. Other inclusion/exclusion criteria apply.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 5 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Eteplirsen<br>Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks. | Drug: Eteplirsen<br>* Eteplirsen IV infusion once weekly.<br>* Other names: EXONDYS;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | Up to 162 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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DMD, Duchenne, Eteplirsen, Dystrophy, Dystrophin, Exon Skipping, Exon 51, Ambulatory, Pediatric