Phase 1/2 study of DYNE-251 in Duchenne Muscular Dystrophy
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
IRAS ID
1005310
Contact name
Caitlin Abell
Contact email
Sponsor organisation
Dyne Therapeutics, Inc
Eudract number
2021-005478-24
Research summary
The DELIVER study is testing an investigational drug called DYNE-251 in boys and male adolescents with Duchenne muscular dystrophy (DMD) with mutations amenable to exon 51 skipping. The DELIVER study is designed to understand if DYNE-251 is safe, what the best dose of DYNE-251 is, and if it can increase muscle dystrophin and improve muscle function. Participants will be in the study for up to 35 months, including multiple scheduled visits to the study site. Approximately 46 participants will be entered into the study globally. There are inclusion and exclusions criteria, and therefore not every patient is eligible.
In the DELIVER study, a computer will assign a participant to receive either DYNE-251 or placebo, at random, for the first 24 weeks. Neither the participant nor parent or caregiver will know which study treatment will be received for the first 24 weeks. The study doctor and nurse also will not know.
The placebo is a water and sugar solution that looks like DYNE-251 but contains no active treatment. A placebo is used in a clinical trial to better understand whether an investigational therapy is working and safe. In this study, comparing DYNE-251 to placebo will help doctors determine if any effects seen in your body are due to DYNE-251 or not. All participants on placebo will receive DYNE-251 after 24 weeks.REC name
South Central - Berkshire Research Ethics Committee
REC reference
22/SC/0372
Date of REC Opinion
8 Dec 2022
REC opinion
Further Information Favourable Opinion