Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2025-522957-20-00 | EU Trial (CTIS) Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo-Controlled Period: DYNE-101 | Experimental | Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks. |
|
| Placebo-Controlled Period: Placebo | Placebo Comparator | Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks. |
|
| Long-Term Extension Period: DYNE-101 | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| zeleciment basivarsen (DYNE-101) | Drug | Administered by IV infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| 5 Times Sit-To-Stand (5×STS) Time | Baseline, Week 49 |
| Measure | Description | Time Frame |
|---|---|---|
| Video Hand Opening Time (vHOT) [Middle Finger] | Baseline, Week 49 | |
| Quantitative Muscle Testing (QMT) Total | Baseline, Week 49 | |
| Clinician Global Impression of Change (CGI-C) |
| Measure | Description | Time Frame |
|---|---|---|
| Fatigue and Daytime Sleepiness Scale (FDSS) | Up to Week 73 | |
| Cognitive function as measured by individual and composite Cogstate cognition scores | Up to Week 73 | |
| Digital functional assessments as measured by the ActiGraph LEAP® wearable device |
Inclusion Criteria:
Exclusion Criteria:
Note: Other inclusion and exclusion criteria may apply.
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Dyne Clinical Trials | Contact | +1-781-317-1919 | clinicaltrials@dyne-tx.com |
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSD - Altman Clinical and Translational Research Institute | Recruiting | La Jolla | California | 92037 | United States |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug | Administered by IV infusion |
|
| Week 49 |
| 10-Meter Walk/Run Test (10-MWRT) Velocity (m/s) | Baseline, Week 49 |
| Patient Global Impression of Change (PGI-C) | Week 49 |
| DM1-ACTIV^C Total Score | Baseline, Week 49 |
| Myotonic Dystrophy Health Index (MDHI) Total | Baseline, Week 49 |
| Patient Global Impression of Severity (PGI-S) | Baseline, Week 49 |
| Clinician Global Impression of Severity (CGI-S) | Baseline, Week 49 |
| 9-Hole Peg Test (9-HPT) Time | Baseline, Week 49 |
| Myotonic Dystrophy Health Index (MDHI) Subscale Scores | Baseline, Week 49 |
| Maximum Observed Plasma Drug Concentration (Cmax) of DYNE-101 | Pre-dose, and at multiple time points up to Week 73 |
| Time to Maximum Concentration (tmax) of DYNE-101 | 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 | Pre-dose, and at multiple time points up to Week 73 |
| Area Under the Concentration-Time Curve Extrapolated to Infinity (AUC0-∞) of DYNE-101 | Pre-dose, and at multiple time points up to Week 73 |
| Apparent Terminal Elimination Rate Constant (λZ) of DYNE-101 | Pre-dose, and at multiple time points up to Week 73 |
| Apparent Terminal Elimination Half-Life (t½) of DYNE-101 | Pre-dose, and at multiple time points up to Week 73 |
| Plasma clearance (CL) of DYNE-101 | Pre-dose, and at multiple time points up to Week 73 |
| Volume of Distribution at the Terminal Phase (Vz), if Appropriate of DYNE-101 | Pre-dose, and at multiple time points up to Week 73 |
| Volume of Distribution at Steady State (Vss), if Appropriate of DYNE-101 | Pre-dose, and at multiple time points up to Week 73 |
| Number of Participants With Antidrug Antibodies (ADAs) | Up to Week 73 |
| Up to Week 73 |
| Rare Disease Research, LLC | Recruiting | Atlanta | Georgia | 30329 | United States |
|
| IU Health Neuroscience Center | Recruiting | Indianapolis | Indiana | 46202 | United States |
|
| Roy Blunt NextGen Precision Health Institute | Recruiting | Columbia | Missouri | 65211 | United States |
|
| Rare Disease Research, LLC | Recruiting | Hillsborough | North Carolina | 27278 | United States |
|
| The University of Texas Health Science Center at San Antonio | Recruiting | San Antonio | Texas | 78229 | United States |
|
| UZ Leuven | Recruiting | Leuven | 3000 | Belgium |
|
| Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077) | Recruiting | Copenhagen | 2100 | Denmark |
|
| CHU de Nantes - Hôtel-Dieu | Recruiting | Nantes | 44000 | France |
|
| Pitié-Salpêtrière Hospital | Recruiting | Paris | 75013 | France |
|
| CHU de Toulouse - Hôpital Pierre-Paul Riquet | Recruiting | Toulouse | 31300 | France |
|
| LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt | Recruiting | Munich | Bavaria | 80336 | Germany |
|
| Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit | Recruiting | Berlin | State of Berlin | 13125 | Germany |
|
| National Hospital Organization Osaka Toneyama Medical Center | Recruiting | Toyonaka-Shi | Osaka | 560-8552 | Japan |
|
| Yamaguchi University Hospital | Recruiting | Ube-Shi | Yamaguchi | 755-8505 | Japan |
|
| Hospital Universitario Infanta Sofía | Recruiting | San Sebastián de los Reyes | Madrid | 28702 | Spain |
|
| University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery | Recruiting | London | England | WC1N 3BG | United Kingdom |
|
| ID | Term |
|---|---|
| D009223 | Myotonic Dystrophy |
| D009222 | Myotonia |
| D020967 | Myotonic Disorders |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020879 | Neuromuscular Manifestations |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided