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Recruiting
NCT07486934
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Conditions: Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy, Steinert Disease, Steinert
Sex: All
Ages: 16 Years – N/A
Healthy volunteers: No
Phase: PHASE3
Enrollment: 150
Sponsor: Dyne Therapeutics
Location: UCSD - Altman Clinical and Translational Research Institute La Jolla California
Summary
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).
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 ankle are allowed).
* Body mass index (BMI) less than (\
Source: ClinicalTrials.gov (NCT07486934). StuddyBuddy aggregates publicly available trial information.