Clinical trials

3

Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).

Participants needed: 150
Trial details
Phase: Phase 3Age: 16+Biological sex: AllType: InterventionalSponsor: Dyne TherapeuticsUpdated: Jun 30, 2026Locations: 14
Eligibility criteria

Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size... [+2]

A known diagnosis of congenital DM1. [+2]

Status: Recruiting

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.

Participants needed: 90
Trial details
Phase: Phase 3Age: 4-18Biological sex: MaleType: InterventionalSponsor: Dyne TherapeuticsUpdated: May 27, 2026Locations: 1
Eligibility criteria

Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystr... [+2]

Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within... [+6]

Status: Recruiting

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.

Participants needed: 116
Trial details
Phase: Phase 1, Phase 2Age: 18-65Biological sex: AllType: InterventionalSponsor: Dyne TherapeuticsUpdated: May 12, 2026Locations: 20
Eligibility criteria

Diagnosis of DM1 with trinucleotide repeat size >100. [+4]

History of major surgical procedure within 12 weeks prior to the start of invest... [+9]