= Fully recruited
= Not yet recruiting
= Enrolling by invitation
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, aged 4 to 16 years, Amenable to Exon 51 Skipping.
DYNE-251 is Dyne’s product candidate being developed for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create a truncated, functional dystrophin protein, with the goal of stopping or reversing disease progression. In preclinical studies with Dyne’s FORCE™ platform, robust and durable exon skipping and dystrophin expression were observed in the mdx mouse model in skeletal and cardiac muscle as well as reduced muscle damage and increased muscle function. In non-human primates, DYNE-251 demonstrated a favorable safety profile and achieved impressive exon skipping, especially in the heart and diaphragm, muscles in people living with DMD that weaken over time leading to mortality.
The DELIVER study is testing an investigational drug called DYNE-251 in males 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 administered DYNE-251 intravenously once every 4 weeks for up to 3 years, including multiple scheduled visits to the study site. Approximately 46 participants will be entered into the study globally. There are inclusion and exclusion 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, or study staff will know which study treatment will be received for the first 24 weeks. 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.
The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks)