Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Recruiting
Phase 3
Interventional Study
Myotonic Dystrophy Type 1 (DM1)
DM1
Myotonic Dystrophy
Steinert Disease
Steinert
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 16 and older
- Sex
- Any
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 150 (estimated)
- Sponsor
- Dyne Therapeutics · Industry
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About This Trial
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).
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\>) 100. Historical results from clinical testing are acceptable.
* Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the…
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