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ACTIVE NOT RECRUITINGPhase1/Phase2

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

Sponsor: Dyne Therapeutics

NCT ID: NCT05481879

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
DYNE-101 (drug), Placebo (drug)
How long the study runs
Study runs about 82 months (dates as stated)
About the drug or intervention
DYNE-101 — drug: Administered by IV infusion · Placebo — drug: Administered by IV infusion
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
18 Years to 65 Years
Who
All
Number of participants
127
Started
2022-09-05
Last checked
2026-09

Plain English Summary

What is this study?

  • • Testing a new treatment for myotonic dystrophy type 1 (dm1)
  • • Phase1/Phase2 - 127 participants
  • • 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)

Who can take part?

  • • Ages 18 Years to 65 Years
  • • Diagnosed with myotonic dystrophy type 1 (dm1)

Where?

  • • London - University College London Hospitals
  • • Newcastle upon Tyne - John Walton Muscular Dystrophy Research Centre
  • • Salford - Salford Royal Hospital

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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 (208 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.

Myotonic Dystrophy Type 1 (DM1)

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Still need:

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years - 65 Years
  • Who can join: All genders

Biomarkers mentioned

PR

What the study is looking for

  • ✓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...

Who cannot take part

  • ✗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...
  • ✗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...
See the full criteria
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.

Where Is This Study? (3 UK sites)

University College London Hospitals

London NW1 2BU, United Kingdom

Hospital R&D contact (matched)

Rajinder Sidhu - Associate Director, Research Governance and Operations

uclh.jro-communications@nhs.net020 3447 9825

John Walton Muscular Dystrophy Research Centre

Newcastle upon Tyne, United Kingdom

Hospital R&D contact (matched)

Jonathan McGregor

wcft.rdi@nhs.net0151 556 3748

Salford Royal Hospital

Salford M6 8HD, United Kingdom

Data sourced from ClinicalTrials.gov · Last verified: 2026-09