Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
NCT ID: NCT05481879
Last Updated: 2025-11-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
116 participants
INTERVENTIONAL
2022-09-05
2029-07-31
Brief Summary
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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.
Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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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.
DYNE-101
Administered by IV infusion
MAD Cohort: Placebo-Controlled Period: Placebo
Participants will be randomized to receive DYNE-101 matching placebo, Q4W or Q8W for up to 24 weeks.
Placebo
Administered by IV infusion
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.
DYNE-101
Administered by IV infusion
Placebo
Administered by IV infusion
MAD Cohort: Long-Term Extension Period: DYNE-101
Participants will receive DYNE-101, Q4W or Q8W for up to 168 weeks.
DYNE-101
Administered by IV infusion
Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101
Participants will receive DYNE-101, Q8W for up to 24 weeks.
DYNE-101
Administered by IV infusion
Dose Expansion Cohort: Placebo-Controlled Period: Placebo
Participants will receive DYNE-101 matching placebo, Q8W for up to 24 weeks.
Placebo
Administered by IV infusion
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.
DYNE-101
Administered by IV infusion
Placebo
Administered by IV infusion
Dose Expansion Cohort: Long-Term Extension Period: DYNE-101
Participants will receive DYNE-101, Q8W for up to 168 weeks.
DYNE-101
Administered by IV infusion
Interventions
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DYNE-101
Administered by IV infusion
Placebo
Administered by IV infusion
Eligibility Criteria
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Inclusion Criteria
* 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 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.
18 Years
65 Years
ALL
No
Sponsors
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Dyne Therapeutics
INDUSTRY
Responsible Party
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Locations
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Indiana University School of Medicine
Indianapolis, Indiana, United States
Neurology Rare Disease Center
Denton, Texas, United States
St. Vincent's Hospital
Fitzroy, Victoria, Australia
CHU de Nantes
Nantes, , France
Institut de Myologie
Paris, , France
Ludwig Maximilians University, Munich - Friedrich Baur Institut
Munich, , Germany
Centro Clinico Nemo
Milan, , Italy
Fondazione Policlinico Universitario A Gemelli-Rome
Rome, , Italy
Radboud Medical Center
Nijmegen, , Netherlands
NZCR Auckland
Auckland, , New Zealand
University College London Hospitals
London, , United Kingdom
John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, , United Kingdom
Salford Royal Hospital
Salford, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Patti Hogan, BSN RN, CCRA
Role: primary
Andrea Ramirez
Role: primary
Alan Lai
Role: primary
Marie-Agnès Sourdril
Role: primary
Asma Balloumi
Role: primary
Corinna Wirner-Piotrowski
Role: primary
Maribel Evoli
Role: primary
Maria Pirozzoli
Role: primary
Eline Sanders
Role: primary
Miriam Rodrigues
Role: primary
Nikoletta Nikolenko
Role: primary
Nicola McLarty
Role: primary
Bethan Blackledge
Role: primary
Other Identifiers
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2023-510353-42-00
Identifier Type: CTIS
Identifier Source: secondary_id
DYNE101-DM1-201
Identifier Type: -
Identifier Source: org_study_id