DM1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Steinert, Steinert Disease
Conditions
Keywords
DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy, HARMONIA, Dyne Therapeutics, Dyne, DYNE-101, zeleciment basivarsen, z-basivarsen
Brief summary
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
DRUGzeleciment basivarsen (DYNE-101)
Administered by IV infusion
DRUGPlacebo
Administered by IV infusion
Sponsors
Dyne Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 5 Times Sit-To-Stand (5×STS) Time | Baseline, Week 49 |
Secondary
| Measure | 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) | 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 |
Countries
Australia, Belgium, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom, United States
Contacts
CONTACTDyne Clinical Trials
Outcome results
None listed