A Phase 3 interventional study of zeleciment basivarsen (DYNE-101) and Placebo in Myotonic Dystrophy Type 1 (DM1), DM1 and Myotonic Dystrophy, sponsored by Dyne Therapeutics. Recruiting at 44 sites in 12 countries. Open to participants aged 16 Years and older. Per ClinicalTrials.gov, last updated 2026-11-06.
Sponsored by Dyne Therapeutics · Phase 3, Interventional, and Treatment
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).
The study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks).
An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.
126 studies on the registry are indexed under Myotonic Dystrophy; 50 are open to participants now.
This study's planned enrollment of 150 is above the median of 40 across 67 interventional studies indexed under Myotonic Dystrophy.
Browse Myotonic Dystrophy studies →Dyne Therapeutics is the lead sponsor of 4 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Note: Other inclusion and exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks.
Drug: zeleciment basivarsen (DYNE-101)
Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks.
Drug: Placebo
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.
Drug: zeleciment basivarsen (DYNE-101) · Drug: Placebo
Administered by IV infusion
Administered by IV infusion
5 Times Sit-To-Stand (5×STS) Time
Time frame: Baseline, Week 49
Video Hand Opening Time (vHOT) [Middle Finger]
Time frame: Baseline, Week 49
Quantitative Muscle Testing (QMT) Total
Time frame: Baseline, Week 49
Clinician Global Impression of Change (CGI-C)
Time frame: Week 49
10-Meter Walk/Run Test (10-MWRT) Velocity (m/s)
Time frame: Baseline, Week 49
Patient Global Impression of Change (PGI-C)
Time frame: Week 49
DM1-ACTIV^C Total Score
Time frame: Baseline, Week 49
Myotonic Dystrophy Health Index (MDHI) Total
Time frame: Baseline, Week 49
Patient Global Impression of Severity (PGI-S)
Time frame: Baseline, Week 49
Clinician Global Impression of Severity (CGI-S)
Time frame: Baseline, Week 49
9-Hole Peg Test (9-HPT) Time
Time frame: Baseline, Week 49
Myotonic Dystrophy Health Index (MDHI) Subscale Scores
Time frame: Baseline, Week 49
Maximum Observed Plasma Drug Concentration (Cmax) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Time to Maximum Concentration (tmax) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Area Under the Concentration-Time Curve (AUC) from Hour 0 to the Last Measurable Concentration (AUC0-tlast) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Area Under the Concentration-Time Curve Extrapolated to Infinity (AUC0-∞) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Apparent Terminal Elimination Rate Constant (λZ) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Apparent Terminal Elimination Half-Life (t½) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Plasma clearance (CL) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Volume of Distribution at the Terminal Phase (Vz), if Appropriate of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Volume of Distribution at Steady State (Vss), if Appropriate of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Number of Participants With Antidrug Antibodies (ADAs)
Time frame: Up to Week 73
Fatigue and Daytime Sleepiness Scale (FDSS)
Time frame: Up to Week 73
Cognitive function as measured by individual and composite Cogstate cognition scores
Time frame: Up to Week 73
Digital functional assessments as measured by the ActiGraph LEAP® wearable device
Time frame: Up to Week 73
Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Dyne Therapeutics