Open Label Extension study of WVE-210201 in patients with DMD
Research type
Research Study
Full title
A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001
IRAS ID
245332
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
Wave Life Sciences Ltd.
Eudract number
2018-000975-34
Duration of Study in the UK
0 years, 10 months, 15 days
Research summary
This open-label extension study is being conducted to evaluate the safety, tolerability and preliminary 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.
The only approved therapy indicated for the treatment of DMD in the EU is Ataluren, which targets ribosomal read through of mRNA in patients with a nonsense mutation. Ataluren is indicated for the treatment of ambulatory patients with a nonsense mutation in the Dystrophin gene. 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. To date, a single Phase 1 clinical study of WVE-210201 (WVE-DMDX51-001) has been initiated. Final data analysis from this study is pending, however there have been no safety signals to date that would preclude conduct of the open-label extension study.
Up to 40 male patients who have completed the WVE-DMDX51-001 study will be enrolled in North America and the EU. All patients will receive WVE-210201. The study will include a Screening period (up to 4 weeks), Treatment period (13 weeks), and Follow-up period (12 weeks). Safety, pharmacokinetic, pharmacodynamic, and efficacy assessments will include laboratory tests (haematology, chemistry, coagulation, immunogenicity, urinalysis, WVE-210201 concentration), muscle biopsy, motor function tests, echocardiograms, and electrocardiograms.
REC name
London - Chelsea Research Ethics Committee
REC reference
18/LO/1238
Date of REC Opinion
19 Sep 2018
REC opinion
Favourable Opinion