Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Purpose
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).
Conditions
- Myotonic Dystrophy Type 1 (DM1)
- DM1
- Myotonic Dystrophy
- Steinert Disease
- Steinert
Eligibility
- Eligible Ages
- Over 16 Years
- Eligible Sex
- All
- Accepts 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 exclusion criteria may apply.
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Placebo-Controlled Period: DYNE-101 |
Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks. |
|
|
Placebo Comparator Placebo-Controlled Period: Placebo |
Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks. |
|
|
Experimental Long-Term Extension Period: DYNE-101 |
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. |
|
Recruiting Locations
Rare Disease Research, LLC
Atlanta, Georgia 30329
Atlanta, Georgia 30329
Rare Disease Research, LLC
Hillsborough, North Carolina 27278
Hillsborough, North Carolina 27278
The University of Texas Health Science Center at San Antonio
San Antonio, Texas 78229
San Antonio, Texas 78229
More Details
- Status
- Recruiting
- Sponsor
- Dyne Therapeutics
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.