Phase 2/3 Study of WVE-210201 in Ambulatory Patients with DMD
Research type
Research Study
Full title
A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy
IRAS ID
261190
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
Wave Life Sciences UK Limited
Eudract number
2018-004009-22
Duration of Study in the UK
1 years, 6 months, 1 days
Research summary
This double-blinded Phase 2/3 study is being conducted to evaluate the safety and efficacy of weekly doses of WVE-210201 in male paediatric patients with Duchenne muscular dystrophy (DMD). DMD is a fatal, progressive, genetic neuromuscular disease that affects approximately one in 3,500 to 5,000 males born worldwide, and is caused by mutations in the gene Dystrophin.
There is one approved therapy for the treatment of DMD in the EU (Ataluren), however, Ataluren is indicated for only a small fraction of patients with DMD (13%) who have a nonsense mutation in the gene for Dystrophin. Furthermore, the clinical benefit of Ataluren has not been established and additional evidence is required to support its continued approval in the EU.
WVE-210201 is an antisense oligonucleotide, a type of nucleic acid molecule, that is intended for the treatment of DMD in patients with a confirmed mutation in the Dystrophin gene that would benefit from exon 51 skipping, i.e. ‘skipping over’ mutated DNA to produce a shortened, but functional version of the protein created by the Dystrophin gene. A Phase 1 study of WVE-210201 in ambulatory and non-ambulatory males, and an associated OLE study, are ongoing. Final data analysis from these studies is pending, however there have been no safety signals to date that would preclude conduct of this study.
Up to 150 male patients will be enrolled globally, and patients will be randomly assigned treatment with intravenous WVE-210201 or a placebo. The study will include a Screening period (up to 6 weeks), Treatment period (48 weeks), and Follow-up period (2 weeks, if not enrolling in the planned OLE study). Safety and efficacy assessments will include laboratory tests (haematology, chemistry, coagulation, immunogenicity, urinalysis, WVE-210201 concentration), muscle biopsy, vital signs, physical examinations, functional assessments, questionnaires, echocardiograms, and electrocardiograms.
REC name
London - Chelsea Research Ethics Committee
REC reference
19/LO/0642
Date of REC Opinion
27 Jun 2019
REC opinion
Further Information Favourable Opinion