Eteplirsen in Patients with Duchenne Muscular Dystrophy – OLE
Research type
Research Study
Full title
An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
IRAS ID
264146
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc.
Eudract number
2019-000337-39
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
6 years, 0 months, 1 days
Research summary
This is a phase 2 open label extension study to assess the safety, tolerability and efficacy of a study medicine eteplirsen in young boys with Duchenne Muscular Dystrophy (DMD) who have completed study 4658-102.
DMD is caused by a mutation (a change) in the gene that makes dystrophin (a protein). Dystrophin is important for protecting muscles from stress and damage during activity. If a person has DMD, their body is not able to make enough working dystrophin to protect their muscles.
Symptoms of DMD may be present during the first year of life, but diagnosis is usually made between the ages of 3 and 5 years. The risk of having a child with DMD is about 1 in every 5000 male births. There is no cure for DMD, but there are medicines and other therapies that can ease the symptoms.
Eteplirsen is an investigational product being developed by Sarepta Therapeutics, Inc. which is designed to “skip” the missing part of the gene that makes the dystrophin protein. “Skipping” over the missing area may allow the body to produce a shortened, but still working, form of the dystrophin protein.
Approximately 15 male DMD participants will be included. Participation will last up to 288 weeks: up to 284 weeks Treatment Period and about 4 weeks Safety Follow-up Period.
We are doing this study as an extension so we can understand if eteplirsen is safe to take over the long-term. This study will test the effects of eteplirsen on muscle function in young boys with DMD who have deletions that may be treated by skipping exon 51. We will also be looking for any changes from baseline in the blood and urine tests, vital signs, electrocardiogram findings, physical exam findings, weight, height and an assessment called North Star Ambulatory Assessment.
REC name
London - Hampstead Research Ethics Committee
REC reference
19/LO/0919
Date of REC Opinion
28 Jun 2019
REC opinion
Further Information Favourable Opinion