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A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1

Status
Not yet recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2025-522957-20-00
Enrollment
51
Registered
2026-04-28
Start date
Unknown
Completion date
Unknown
Last updated
2026-04-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myotonic Dystrophy Type 1

Brief summary

5×STS time, change from baseline at Week 49

Detailed description

Change from baseline at Week 49 in: vHOT; middle finger QMT total CGI-C 10-MWRT velocity (m/s) PGI-C DM1-ACTIVC total score MDHI total PGI-S CGI-S 9-HPT MDHI subscale, Maximum observed drug concentration (Cmax) Time to maximum concentration (tmax) Area under the concentration-time curve (AUC) from hour 0 to the last measurable concentration (AUCtlast) AUC extrapolated to infinity (AUC∞) Apparent terminal elimination rate constant (λZ) Apparent terminal elimination half-life (t½) Plasma clearance (CL) Volume of distribution at the terminal phase (Vz), if appropriate Volume of distribution at steady state (Vss), if appropriate, Incidence of anti-drug antibodies (ADAs), * Treatment-emergent adverse events (TEAEs) including: - Treatment-emergent serious adverse events (SAEs) - TEAEs considered related to study drug - TEAEs leading to discontinuation from study drug and discontinuation from the study * Other clinically significant safety and tolerability observations including: - Vital sign findings - 12-lead electrocardiogram (ECG) findings -Clinical laboratory values *C-SSRS results

Interventions

DRUGSODIUM CHLORIDE

Sponsors

Dyne Therapeutics Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 64 Years

Design outcomes

Primary

MeasureTime frame
5×STS time, change from baseline at Week 49

Secondary

MeasureTime frame
Change from baseline at Week 49 in: vHOT; middle finger QMT total CGI-C 10-MWRT velocity (m/s) PGI-C DM1-ACTIVC total score MDHI total PGI-S CGI-S 9-HPT MDHI subscale, Maximum observed drug concentration (Cmax) Time to maximum concentration (tmax) Area under the concentration-time curve (AUC) from hour 0 to the last measurable concentration (AUCtlast) AUC extrapolated to infinity (AUC∞) Apparent terminal elimination rate constant (λZ) Apparent terminal elimination half-life (t½) Plasma clearance (CL) Volume of distribution at the terminal phase (Vz), if appropriate Volume of distribution at steady state (Vss), if appropriate, Incidence of anti-drug antibodies (ADAs), * Treatment-emergent adverse events (TEAEs) including: - Treatment-emergent serious adverse events (SAEs) - TEAEs considered related to study drug - TEAEs leading to discontinuation from study drug and discontinuation from the study * Other clinically significant safety and tolerability observations including: - Vital si

Outcome results

None listed

Source: EU CTIS · Data processed: Apr 30, 2026