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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03985878
• Other study ID #: 4658-102-OLE
• Secondary ID: 2019-000337-39
• Status: Terminated
• Phase: Phase 2
• Start date: June 26, 2019
• Est. completion date: August 31, 2022

Study information

• Verified date: July 2023
• Source: Sarepta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: August 31, 2022
• Est. primary completion date: August 31, 2022
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years to 5 Years

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Eteplirsen
Eteplirsen IV infusion once weekly.

Locations

Country	Name	City	State
Belgium	UZ-Gent	Gent
France	Hopital Trousseau, Bâtiment lemariey	Paris
Italy	Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile	Rome
United Kingdom	UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre	London	England

Sponsors (1)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.

Countries where clinical trial is conducted

Belgium,  France,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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