Phase 2 Study for Duchenne muscular dystrophy
Research type
Research Study
Full title
A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
IRAS ID
265653
Contact name
Giovanni Baranello
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc.
Eudract number
2019-000601-77
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
134648, IND
Duration of Study in the UK
1 years, 6 months, 2 days
Research summary
The Sponsor is studying an investigational drug, SRP-5051, for the treatment of Duchenne muscular dystrophy (DMD).
DMD is a genetic condition caused by a mistake in the genetic code (DNA). The mistake occurs in the dytrophin gene. This gene provides instructions for making the dystrophin protein which is needed for muscle function. The lack of this protein in people with DMD results in fragile muscles which become easily damaged. This degeneration process leads eventually to premature death.
SRP-5051 targets a nucleic acid present in all living cells called RNA. RNA stores the information needed by cells to create proteins such as dystrophin. The drug targets the RNA in order that the dystrophin protein is produced.
The purpose of this study is to find the highest dose of SRP-5051 that does not cause unreasonable side effects. Side effects are unwanted or unexpected reactions to the study drug.
The study is split in to two parts, Part A (dose determination) and Part B (dose expansion).
The study will include up to 24 male DMD participants aged 7-21 years.
The duration of each patient's participation in the study is expected to be
approximately 60 weeks, inclusive of Screening, Part A, Part B, and the Safety Follow-up Period.Participants will undergo multiple visits to the study site including some overnight stays. Some visits will include drug administration, muscle biopsies, blood samples, physical examinations, tests to assess how well the heart and lungs work and assessments to evaluate quality of life and physical ability.
REC name
London - Hampstead Research Ethics Committee
REC reference
19/LO/1456
Date of REC Opinion
4 Nov 2019
REC opinion
Further Information Favourable Opinion