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Clinical Trials in Italy / NCT05481879
Active, not recruiting Phase 1/2

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

NCT05481879 · tracked via the Priya Life Science Italy tracker
Phase
Phase 1/2
Started
2022-09-05
Last updated
2026-09-23

Condition(s) studied

Myotonic Dystrophy Type 1 (DM1)

Investigational drug(s) / intervention(s)

DYNE-101 →Placebo

DYNE-101: Administered by IV infusion

Placebo: Administered by IV infusion

Study summary

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
65 Years
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.

Primary outcome measure(s)

Trial sites (20)

FacilityCityRegionStatus
Stanford University Stanford California
University of Florida College of Medicine Gainesville Florida
Indiana University School of Medicine Indianapolis Indiana
University of Iowa Iowa City Iowa
Washington University in St. Louis St Louis Missouri
University of Rochester Medical Center Rochester New York
Neurology Rare Disease Center Denton Texas
Virginia Commonwealth University (VCU) Richmond Virginia
St. Vincent's Hospital Fitzroy Victoria
CHU de Nantes Nantes France
Institut de Myologie Paris France
Charité - Universitätsmedizin Berlin Berlin Germany
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

More Dyne Therapeutics trials in Italy

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05481879 on ClinicalTrials.gov ↗ ← All trials in Italy