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Purpose

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.

Condition

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Diagnosis of DM1 with trinucleotide repeat size >100. - Age of onset of DM1 muscle symptoms ≥12 years. - Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator. - Hand grip strength and ankle dorsiflexion strength. - Able to complete 10-MWRT, stair ascend/descend (MAD cohorts only), and 5×STS at screening without the use of assistive devices such as canes, walkers, or orthoses.

Exclusion Criteria

  • History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical procedure (eg, implantation of cardiac defibrillator) during the study. - History of anaphylaxis. - Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments. - Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to performing screening assessments. - Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or a conduction defect, which is clinically significant in the opinion of the Investigator. - Percent predicted forced vital capacity (FVC) <50%. - History of tibialis anterior biopsy within 3 months of Day 1 or planning to undergo tibialis anterior biopsies during study period for reasons unrelated to the study. - Participant has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) or who, in the opinion of the Investigator, is at significant risk to commit suicide. - Use of glucagon-like peptide 1 (GLP-1) agonist medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments. - Significant weight loss during study participation may impact weight-based dosing, performance on muscle function assessments, and pharmacodynamic (PD) biomarkers. Note: Other inclusion and exclusion criteria may apply.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
MAD Cohort: Placebo-Controlled Period: DYNE-101 		Participants will be randomized to receive ascending doses of DYNE-101, once every 4 weeks (Q4W) or once every 8 weeks (Q8W) for up to 24 weeks.
  • Drug: DYNE-101
    Administered by IV infusion
Placebo Comparator
MAD Cohort: Placebo-Controlled Period: Placebo 		Participants will be randomized to receive DYNE-101 matching placebo, Q4W or Q8W for up to 24 weeks.
  • Drug: Placebo
    Administered by IV infusion
Experimental
MAD Cohort: Treatment Period: DYNE-101 		Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q4W or Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q4W or Q8W for up to 24 weeks.
  • Drug: DYNE-101
    Administered by IV infusion
  • Drug: Placebo
    Administered by IV infusion
Experimental
MAD Cohort: Long-Term Extension Period: DYNE-101 		Participants will receive DYNE-101, Q4W or Q8W for up to 168 weeks.
  • Drug: DYNE-101
    Administered by IV infusion
Experimental
Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101 		Participants will receive DYNE-101, Q8W for up to 24 weeks.
  • Drug: DYNE-101
    Administered by IV infusion
Experimental
Dose Expansion Cohort: Placebo-Controlled Period: Placebo 		Participants will receive DYNE-101 matching placebo, Q8W for up to 24 weeks.
  • Drug: Placebo
    Administered by IV infusion
Experimental
Dose Expansion Cohort: Treatment Period: DYNE-101 		Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.
  • Drug: DYNE-101
    Administered by IV infusion
  • Drug: Placebo
    Administered by IV infusion
Experimental
Dose Expansion Cohort: Long-Term Extension Period: DYNE-101 		Participants will receive DYNE-101, Q8W for up to 168 weeks.
  • Drug: DYNE-101
    Administered by IV infusion

Recruiting Locations

Washington University in St. Louis and nearby locations

Washington University in St. Louis
St Louis, Missouri 63110
Contact:
Natalie Goedeker
ngoedeker@wustl.edu

More Details

NCT ID
NCT05481879
Status
Recruiting
Sponsor
Dyne Therapeutics

Study Contact

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.