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

NCT03985878 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: 4658-102-OLE2019-000337-39
• Study Type: interventional
• Target: 15
• Locations: 4 countries
• Sites: 4

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; Duchenne; Eteplirsen; Dystrophy; Dystrophin; Exon Skipping; Exon 51; Ambulatory; Pediatric

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (1)

Eteplirsen

EXPERIMENTAL

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

Drug: Eteplirsen

Interventions

Eteplirsen DRUG

Eteplirsen IV infusion once weekly.

Also known as: AVI-4658, EXONDYS 51, EXONDYS

Eteplirsen

Eligibility Criteria

• Age: 2 Years - 5 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant successfully completes 96 weeks of treatment in Study 4658-102.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (4)

UZ-Gent

Ghent, 9000, Belgium

Location

Hopital Trousseau, Bâtiment lemariey

Paris, 75021, France

Location

Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile

Rome, 00168, Italy

Location

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

London, England, WC1N 1EH, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

eteplirsen

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.

Results Point of Contact

• Title: Medical Director
• Organization: Sarepta Therapeutics, Inc.

SareptAlly@sarepta.com

1-888-SAREPTA (1-888-727-3782)

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 11, 2019
• First Posted: June 14, 2019
• Study Start: June 26, 2019
• Primary Completion: August 31, 2022
• Study Completion: August 31, 2022
• Last Updated: August 18, 2023
• Results First Posted: August 18, 2023

Record last verified: 2023-07

Locations

BE (1); IT (1); GB (1); FR (1)