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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05481879
Other study ID # DYNE101-DM1-201
Secondary ID 2022-000889-18
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date September 5, 2022
Est. completion date July 2026

Study information

Verified date December 2023
Source Dyne Therapeutics
Contact Dyne Clinical Trials
Phone +1-781-317-1919
Email clinicaltrials@dyne-tx.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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 multiple-ascending dose (MAD) Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (96 weeks).


Recruitment information / eligibility

Status Recruiting
Enrollment 72
Est. completion date July 2026
Est. primary completion date July 2026
Accepts healthy volunteers No
Gender All
Age group 18 Years to 49 Years
Eligibility 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, 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. Note: Other inclusion and exclusion criteria may apply.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
DYNE-101
Administered by IV infusion
Placebo
Administered by IV infusion

Locations

Country Name City State
France Institut de Myologie Paris
Germany Ludwig Maximilians University, Munich - Friedrich Baur Institut Munich
Italy Centro Clinico Nemo Milan
Italy Fondazione Policlinico Universitario A Gemelli-Rome Rome
Netherlands Radboud Medical Center Nijmegen
New Zealand NZCR Auckland Auckland
United Kingdom University College London Hospitals London
United Kingdom John Walton Muscular Dystrophy Research Centre Newcastle-Upon-Tyne
United Kingdom Salford Royal Hospital Salford

Sponsors (1)

Lead Sponsor Collaborator
Dyne Therapeutics

Countries where clinical trial is conducted

France,  Germany,  Italy,  Netherlands,  New Zealand,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Through study completion, up to Week 145
Secondary Change From Baseline in Splicing Index in Skeletal Muscle Tissue Baseline up to Week 97
Secondary Change From Baseline in Dystrophia Myotonica Protein Kinase (DMPK) Ribonucleic Acid (RNA) Expression in Muscle Tissue Baseline up to Week 97
Secondary Change From Baseline in Hand Grip Relaxation Time Baseline up to Week 145
Secondary Change From Baseline in Myotonia as Measured by Video Hand Opening Time (vHOT) Baseline up to Week 145
Secondary Change From Baseline in Quantitative Myometry Testing (QMT) Baseline up to Week 145
Secondary Change From Baseline in 10-Meter Walk/Run Test (10-MWRT) Baseline up to Week 145
Secondary Change From Baseline in Stair-Ascend/Descend Test Baseline up to Week 145
Secondary Change From Baseline in 5 Times Sit to Stand (5×STS) Baseline up to Week 145
Secondary Change From Baseline in 9-Hole Peg Test (9-HPT) Baseline up to Week 145
Secondary Maximum Observed Plasma Drug Concentration (Cmax) of DYNE-101 Pre-dose, and at multiple timepoints up to Week 145
Secondary Time to Maximum Observed Plasma Concentration (tmax) of DYNE-101 Pre-dose, and at multiple timepoints up to Week 145
Secondary Area Under the Concentration-time Curve From Hour 0 to the Last Measurable Plasma Concentration (AUCtlast) of DYNE-101 Pre-dose, and at multiple timepoints up to Week 145
Secondary Area Under the Concentration-time Curve Extrapolated to Infinity (AUC8) of DYNE-101 in Plasma Pre-dose, and at multiple timepoints up to Week 145
Secondary Apparent Terminal Elimination Rate Constant (?z) of DYNE-101 in Plasma Pre-dose, and at multiple timepoints up to Week 145
Secondary Apparent Terminal Elimination Half-Life (t½) of DYNE-101 in Plasma Pre-dose, and at multiple timepoints up to Week 145
Secondary Clearance (CL) of DYNE-101 in Plasma Pre-dose, and at multiple timepoints up to Week 145
Secondary Volume of Distribution at Steady State (Vss) of DYNE-101 in Plasma Pre-dose, and at multiple timepoints up to Week 145
Secondary Volume of Distribution at the Terminal Phase (Vz) of DYNE-101 in Plasma Pre-dose, and at multiple timepoints up to Week 145
Secondary Antisense Oligonucleotide (ASO) Concentration of DYNE-101 in Muscle Tissue Up to Week 97
Secondary Percentage of Participants With Antidrug Antibodies (ADAs) Up to Week 145
See also
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