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Also known as Zeleciment Rostudirsen (DYNE-251)
Zeleciment Rostudirsen · 1 trial · 13 indications
| Arm | Type | Description |
|---|---|---|
| Placebo-Controlled Period: Zeleciment Rostudirsen (DYNE-251) | EXPERIMENTAL | Participants will be randomized to receive zeleciment rostudirsen, once every 4 weeks (Q4W) for up to 72 weeks. |
| Placebo-Controlled Period: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo, Q4W for up to 72 weeks. |
| Open-Label Long-Term Extension Period: Zeleciment Rostudirsen (DYNE-251) | EXPERIMENTAL | All participants who complete the Placebo-Controlled Period of the study will receive zeleciment rostudirsen administered Q4W for up to 96 weeks. |
| Name | Type | Description |
|---|---|---|
| Zeleciment Rostudirsen (DYNE-251) | DRUG | Administered by IV infusion |
| Placebo | DRUG | Administered by IV infusion |
Inclusion Criteria: * Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping . * Rise From Floor (RFF) time must be \< 10 seconds for both screening assessments . * Receiving a stable daily or weekend do...
Zeleciment Rostudirsen is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy (DMD). It is currently in Phase 3 clinical development and is being studied in ambulatory male participants with DMD, a rare genetic neuromuscular disease. The drug is administered intravenously every 4 weeks.
Zeleciment Rostudirsen targets TfR1, also known as the transferrin receptor 1. This molecular target is involved in cellular iron uptake and is highly expressed on muscle cells, which may allow for targeted delivery of the therapy to affected tissues in Duchenne Muscular Dystrophy.
Zeleciment Rostudirsen is being developed by Dyne Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol DYN. The company is conducting a Phase 3 clinical trial of the drug in patients with Duchenne Muscular Dystrophy.
Zeleciment Rostudirsen is currently in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 3 trial is recruiting participants to evaluate the efficacy, safety, and tolerability of the drug in ambulatory patients with Duchenne Muscular Dystrophy.
Zeleciment Rostudirsen is being studied in the FORZETTO trial, registered as NCT07608432. This is a Phase 3, randomized, double-blind, placebo-controlled study with an enrollment target of 90 participants. The trial is recruiting male patients aged 4 years and older with Duchenne Muscular Dystrophy in the United States.
Yes, Zeleciment Rostudirsen is also known as DYNE-251. Both names refer to the same investigational drug being developed by Dyne Therapeutics for Duchenne Muscular Dystrophy. The clinical trial NCT07608432 uses the name DYNE-251 in its title, while Zeleciment Rostudirsen is the assigned nonproprietary name.