NCT05481879

Brief 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 (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
116

participants targeted

Target at P75+ for phase_1

Timeline
38mo left

Started Sep 2022

Longer than P75 for phase_1

Geographic Reach
8 countries

17 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress54%
Sep 2022Jul 2029

First Submitted

Initial submission to the registry

July 28, 2022

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 1, 2022

Completed
1 month until next milestone

Study Start

First participant enrolled

September 5, 2022

Completed
6.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2029

Last Updated

March 5, 2026

Status Verified

August 1, 2025

Enrollment Period

6.8 years

First QC Date

July 28, 2022

Last Update Submit

March 3, 2026

Conditions

Myotonic Dystrophy Type 1 (DM1)

Outcome Measures

Primary Outcomes (2)

  • MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    Through study completion, up to Week 217

  • Dose Expansion Cohorts: Change From Baseline in Myotonia as Measured by Video Hand Opening Time (vHOT)

    Baseline up to Week 25

Secondary Outcomes (33)

  • MAD Cohorts: Change From Baseline in Composite Alternative Splicing Index (CASI) in Skeletal Muscle Tissue

    Baseline up to Week 45

  • MAD Cohorts: Change From Baseline in Dystrophia Myotonica Protein Kinase (DMPK) Ribonucleic Acid (RNA) Expression in Muscle Tissue

    Baseline up to Week 45

  • MAD Cohorts: Change From Baseline in Hand Grip Relaxation Time

    Baseline up to Week 121

  • MAD Cohorts: Change From Baseline in Myotonia as Measured by vHOT

    Baseline up to Week 193

  • MAD Cohorts: Change From Baseline in Quantitative Myometry Testing (QMT)

    Baseline up to Week 193

  • +28 more secondary outcomes

Study Arms (8)

MAD Cohort: Placebo-Controlled Period: DYNE-101

EXPERIMENTAL

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

MAD Cohort: Placebo-Controlled Period: Placebo

PLACEBO COMPARATOR

Participants will be randomized to receive DYNE-101 matching placebo, Q4W or Q8W for up to 24 weeks.

Drug: Placebo

MAD Cohort: Treatment Period: DYNE-101

EXPERIMENTAL

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-101Drug: Placebo

MAD Cohort: Long-Term Extension Period: DYNE-101

EXPERIMENTAL

Participants will receive DYNE-101, Q4W or Q8W for up to 168 weeks.

Drug: DYNE-101

Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101

EXPERIMENTAL

Participants will receive DYNE-101, Q8W for up to 24 weeks.

Drug: DYNE-101

Dose Expansion Cohort: Placebo-Controlled Period: Placebo

EXPERIMENTAL

Participants will receive DYNE-101 matching placebo, Q8W for up to 24 weeks.

Drug: Placebo

Dose Expansion Cohort: Treatment Period: DYNE-101

EXPERIMENTAL

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-101Drug: Placebo

Dose Expansion Cohort: Long-Term Extension Period: DYNE-101

EXPERIMENTAL

Participants will receive DYNE-101, Q8W for up to 168 weeks.

Drug: DYNE-101

Interventions

DYNE-101DRUG

Administered by IV infusion

Dose Expansion Cohort: Long-Term Extension Period: DYNE-101Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101Dose Expansion Cohort: Treatment Period: DYNE-101MAD Cohort: Long-Term Extension Period: DYNE-101MAD Cohort: Placebo-Controlled Period: DYNE-101MAD Cohort: Treatment Period: DYNE-101
PlaceboDRUG

Administered by IV infusion

Dose Expansion Cohort: Placebo-Controlled Period: PlaceboDose Expansion Cohort: Treatment Period: DYNE-101MAD Cohort: Placebo-Controlled Period: PlaceboMAD Cohort: Treatment Period: DYNE-101

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

RECRUITING

University of Iowa

Iowa City, Iowa, 52242, United States

RECRUITING

University of Rochester Medical Center

Rochester, New York, 14642, United States

RECRUITING

Neurology Rare Disease Center

Denton, Texas, 76208, United States

RECRUITING

Virginia Commonwealth University (VCU)

Richmond, Virginia, 23298, United States

RECRUITING

St. Vincent's Hospital

Fitzroy, Victoria, 3065, Australia

RECRUITING

CHU de Nantes

Nantes, 44093, France

RECRUITING

Institut de Myologie

Paris, 75013, France

RECRUITING

Charité - Universitätsmedizin Berlin

Berlin, 10117, Germany

RECRUITING

Ludwig Maximilians University, Munich - Friedrich Baur Institut

Munich, 80336, Germany

RECRUITING

Centro Clinico Nemo

Milan, 20162, Italy

RECRUITING

Fondazione Policlinico Universitario A Gemelli-Rome

Rome, 00168, Italy

RECRUITING

Radboud Medical Center

Nijmegen, Netherlands

RECRUITING

NZCR Auckland

Auckland, 1023, New Zealand

RECRUITING

University College London Hospitals

London, NW1 2BU, United Kingdom

RECRUITING

John Walton Muscular Dystrophy Research Centre

Newcastle upon Tyne, United Kingdom

RECRUITING

Salford Royal Hospital

Salford, M6 8HD, United Kingdom

RECRUITING

MeSH Terms

Conditions

Myotonic Dystrophy

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesMyotonic DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Dyne Clinical Trials

CONTACT

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

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 28, 2022

First Posted

August 1, 2022

Study Start

September 5, 2022

Primary Completion (Estimated)

July 1, 2029

Study Completion (Estimated)

July 1, 2029

Last Updated

March 5, 2026

Record last verified: 2025-08

Data Sharing

IPD Sharing
Will not share

Locations

NL(1)IT(2)NZ(1)US(5)AU(1)GB(3)FR(2)DE(2)