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

ArmDescriptionAssigned 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.
  • Drug: zeleciment basivarsen (DYNE-101)
    Administered by IV infusion
Placebo Comparator
Placebo-Controlled Period: Placebo 		Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks.
  • Drug: Placebo
    Administered by IV infusion
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.
  • Drug: zeleciment basivarsen (DYNE-101)
    Administered by IV infusion
  • Drug: Placebo
    Administered by IV infusion

Recruiting Locations

The University of Texas Health Science Center at San Antonio
San Antonio, Texas 78229
Contact:
Marlon Humbert Tamayo Muradas
210-450-7370
tamayomurada@uthscsa.edu

More Details

Status
Recruiting
Sponsor
Dyne Therapeutics

Study Contact

Dyne Clinical Trials
+1-781-317-1919
clinicaltrials@dyne-tx.com

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.