Recruiting NCT07486934
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: zeleciment basivarsen (DYNE-101), Placebo.
- Who it may be relevant to
- Registry conditions: Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy, Steinert Disease. Basic parameters: from 16 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Belgium, Denmark, France, Germany +4
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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).
Detailed description
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.
Interventions
- Drug zeleciment basivarsen (DYNE-101)
Administered by IV infusion - Drug Placebo
Administered by IV infusion
Primary outcome measures
- 5 Times Sit-To-Stand (5×STS) Time [Time frame: Baseline, Week 49]
Secondary outcome measures (12)
- 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]
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 (<) 35 kilograms per meter square (kg/m\^2).
Exclusion criteria
- 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.
Note: Other inclusion and exclusion criteria may apply.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 8 centers
- UCSD - Altman Clinical and Translational Research Institute — La Jolla
- Rare Disease Research, LLC — Atlanta
- IU Health Neuroscience Center — Indianapolis
- Roy Blunt NextGen Precision Health Institute — Columbia
- Rare Disease Research, LLC — Hillsborough
- The University of Texas Health Science Center at San Antonio — San Antonio
- University of Utah Clinical Neurosciences Center — Salt Lake City
- Virginia Commonwealth University - Biotech One — Richmond
France · 4 centers
- CHU Marseille - Hôpital de la Timone (APHM) — Marseille
- CHU de Nantes - Hôtel-Dieu — Nantes
- Pitié-Salpêtrière Hospital — Paris
- CHU de Toulouse - Hôpital Pierre-Paul Riquet — Toulouse
Germany · 3 centers
- LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-In — Munich
- Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuro — Bonn
- Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit — Berlin
Italy · 3 centers
- Centro Clinico Nemo Milano, Fondazione Serena ETS — Milan
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and P — Roma
- Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia — Roma
Japan · 2 centers
- National Hospital Organization Osaka Toneyama Medical Center — Toyonaka-Shi
- Yamaguchi University Hospital — Ube-Shi
United Kingdom · 2 centers
- University College London Hospitals NHS Foundation Trust National Hospital for Neurology a — London
- Northern Care Alliance NHS Foundation Trust — Salford
Belgium · 1 center
- UZ Leuven — Leuven
Denmark · 1 center
- Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077) — Copenhagen
Spain · 1 center
- Hospital Universitario Infanta Sofía — San Sebastián de los Reyes
Identifiers
NCT: NCT07486934 · DYNE101-DM1-301 · 2025-522957-20-00