Phase 1 study of WVE-210201 in patients with DMD
Research type
Research Study
Full title
A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy
IRAS ID
239586
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
Wave Life Sciences Ltd.
Eudract number
2017-002686-21
Duration of Study in the UK
0 years, 8 months, 1 days
Research summary
This is a Phase 1, double-blind, placebo-controlled, single ascending dose study to evaluate the safety and tolerability 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 linked to 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. This is the first clinical study conducted with WVE-210201; however, non-clinical studies have not identified any potential risks that would prevent the initiation of the proposed study design.
Approximately 32 males ≥5 and ≤18 years of age with a genetically confirmed diagnosis of DMD who have a gene mutation (deletion) amenable to exon 51 skipping will be enrolled. Four dose levels are planned to be administered in this study. Each cohort will have 8 patients, 6 receiving WVE-210201 and 2 receiving placebo.
The study will include a Screening Visit, a Dosing Visit, and Follow-up for approximately 12 weeks. Safety assessments will be performed weekly until Day 29 and monthly afterwards for a total of approximately 12 weeks, and will include laboratory tests (haematology, chemistry, coagulation, urinalysis) and electrocardiograms.REC name
London - Chelsea Research Ethics Committee
REC reference
18/LO/0126
Date of REC Opinion
11 Apr 2018
REC opinion
Further Information Favourable Opinion