Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age16+
SponsorDyne Therapeutics
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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 (<) 35 kilograms per meter square (kg/m²).
Disqualifiers
A known diagnosis of congenital DM1.
History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator.
Use of glucagon-like peptide 1 (GLP-1) agonist/incretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.
Trial design
Design model
Parallel
Treatments tested in this trial
zeleciment basivarsen (DYNE-101)
DrugAdministered by IV infusion
Placebo
DrugAdministered by IV infusion
Treatment groups
150 Participants
are divided into 3 treatment groupsGroup A: Placebo-Controlled Period: DYNE-101Experimental treatment 1 intervention
Group B: Placebo-Controlled Period: PlaceboPlacebo comparator 1 intervention
Group C: Long-Term Extension Period: DYNE-101Experimental treatment 2 interventions
Trial outcomes
Primary outcomes
1
5 Times Sit-To-Stand (5×STS) Time
Time frame
Baseline, Week 49Secondary outcomes
1
Video Hand Opening Time (vHOT) [Middle Finger]
Time frame
Baseline, Week 492
Quantitative Muscle Testing (QMT) Total
Time frame
Baseline, Week 493
Clinician Global Impression of Change (CGI-C)
Time frame
Week 494
10-Meter Walk/Run Test (10-MWRT) Velocity (m/s)
Time frame
Baseline, Week 49Other outcomes
1
Fatigue and Daytime Sleepiness Scale (FDSS)
Time frame
Up to Week 732
Cognitive function as measured by individual and composite Cogstate cognition scores
Time frame
Up to Week 733
Digital functional assessments as measured by the ActiGraph LEAP® wearable device
Time frame
Up to Week 73Sponsors and contacts
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Source ClinicalTrials.gov