Open-label and Double-Blind study of Eteplirsen in DMD patients
Research type
Research Study
Full title
A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
IRAS ID
264562
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc
Eudract number
2018-001762-42
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
077,429, IND NUMBER
Duration of Study in the UK
5 years, 5 months, 10 days
Research summary
The purpose of this phase 3b research study is to find a higher dose (100 mg/kg or 200 mg/kg) of the study medicine eteplirsen that can safely treat Duchenne Muscular Dystrophy (DMD) patients more effectively than the 30 mg/kg dose.
DMD is caused by a mutation (a change) in the gene that makes dystrophin (a protein in the muscle). Dystrophin is important for protecting muscles from damage during activity. If a person has DMD, their body is not able to make enough working dystrophin to protect their muscles.
Eteplirsen is an investigational product being developed by Sarepta Therapeutics, Inc. 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.
The study has 2 parts:
• Open-label Dose Escalation Part (UK not involved): To assess safety at 2 different doses: 100 mg/kg and 200 mg/kg once weekly.
• Double-Blind Part: Split into 2 parts:
-Dose Finding (to identify which of the 100 mg/kg or 200 mg/kg once a week higher doses affects levels of dystrophin as compared with the 30 mg/kg dose).
-Dose Comparison (to compare the high dose selected from the Dose Finding part of the study with 30 mg/kg).
Eteplirsen will be administered by intravenous (IV) infusion.Up to 8 participants in the Open-label Dose Escalation part and up to 144 participants in the Double-blind part will be included. Approximately 50 sites globally will participate. The Double-Blind part will last for up to 156 weeks: Screening and Baseline Period (up to 8 weeks), Treatment Period (144 weeks) and Safety Follow-up Period (4 weeks).
This study is designed to help evaluate whether administration of eteplirsen may increase production of this shorter form of dystrophin and improve muscle function.
REC name
London - Riverside Research Ethics Committee
REC reference
19/LO/1258
Date of REC Opinion
17 Sep 2019
REC opinion
Further Information Favourable Opinion