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

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Dyne Therapeutics
Study ID
NCT07486934
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • DM1
  • Myotonic Dystrophy
  • Myotonic Dystrophy Type 1 (DM1)
  • Steinert
  • Steinert Disease

Eligibility Criteria

Sex
ALL
Age
16 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • zeleciment basivarsen (DYNE-101) — DRUG
    Administered by IV infusion
  • Placebo — DRUG
    Administered by IV infusion

Study Details

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).

Key Dates

Start date
Apr 30, 2026
Status verified
Apr 2026
Primary completion
Jul 31, 2028
Completion
Jan 31, 2029

Study Design

Enrollment
150 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Placebo-Controlled Period: DYNE-101
    Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks.
  • Placebo Comparator: Placebo-Controlled Period: Placebo
    Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks.
  • Experimental: Long-Term Extension Period: DYNE-101
    Participants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks. Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.

Primary Outcome Measure

5 Times Sit-To-Stand (5×STS) Time [ Time Frame: Baseline, Week 49 ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Rare Disease Research, LLCAtlantaGeorgia30329
Roy Blunt NextGen Precision Health InstituteColumbiaMissouri65211
Heather McHatton
[email protected]
573-882-7619
Rare Disease Research, LLCHillsboroughNorth Carolina27278
Hannah Nation
[email protected]
984-314-2252
The University of Texas Health Science Center at San AntonioSan AntonioTexas78229
Marlon Humbert Tamayo Muradas
[email protected]
210-450-7370

Find similar trials in Atlanta, GA

By research site
Rare Disease Research, LLC· Atlanta, GARoy Blunt NextGen Precision Health Institute· Columbia, MORare Disease Research, LLC· Hillsborough, NCThe University of Texas Health Science Center at San Antonio· San Antonio, TX

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