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Safety and effectiveness study of DYNE-101 in patients with Myotonic Dystrophy Type 1 (DM1)

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on people with Myotonic Dystrophy Type 1, a genetic condition that causes progressive muscle weakness and difficulty relaxing muscles after use. The study will test a new medication called DYNE-101, which is given through intravenous administration. Some participants will receive DYNE-101 while others will receive a placebo solution.

The purpose of this research is to evaluate how safe DYNE-101 is and how well patients tolerate it when given multiple doses. The study will also examine how the medication affects muscle tissue in people with Myotonic Dystrophy Type 1. The medication being tested is a specially designed antibody that targets specific proteins in the body.

During the study, participants will receive multiple doses of either DYNE-101 or placebo through an intravenous line. The study will involve various assessments of muscle strength and function, including tests of hand grip strength and walking ability. Participants will also undergo muscle tissue sampling to evaluate how the medication affects their muscle cells.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment period

    You will receive either DYNE-101 or placebo (saline solution) through intravenous infusion

    The treatment involves multiple doses over time

    You will be assigned to either the Multiple Ascending Dose (MAD) group or the Dose Expansion group

  2. Step 2

    Medical assessments

    Your muscle strength will be measured through various tests:

    - Hand grip strength test

    - 10-meter walk/run test

    - Stair climbing test

    - Five times sit-to-stand test

    - 9-hole peg test

    Multiple muscle tissue samples (biopsies) will be collected during the study

  3. Step 3

    Monitoring period

    Your muscle function will be regularly evaluated up to week 25

    Blood samples will be taken to measure drug levels in your body

    You will be monitored for any side effects throughout the study

    Your muscle tissue will be examined for changes at week 13

  4. Step 4

    Study completion

    The study is expected to continue until February 2030

    Regular assessments will track changes in your muscle strength and function

    Your overall health status will be monitored throughout the study duration

Who can join the trial?

13 criteria

  • Must be between 18 and 50 years old for MAD group, or 18 to 65 years old for Dose Expansion group
  • Must have confirmed DM1 diagnosis with more than 100 trinucleotide repeats through genetic testing
  • First muscle symptoms must have started at age 12 or later
  • Must have muscle stiffness (myotonia) with hand opening time of at least 2 seconds
  • Hand grip strength must be between 20% and 80% of normal range for age, sex, and height
  • Ankle strength must be between 20% and 80% of normal range for age, sex, and height
  • Must be able to complete walking test, climb stairs, and stand from sitting position without using mobility aids (ankle supports are allowed)
  • Body Mass Index (BMI) must be less than 35 kg/m²
  • If taking testosterone, must be on stable dose for at least 30 days before screening
  • Must agree to follow contraception requirements during the study
  • Women must not be pregnant or breastfeeding
  • Must be able to provide informed consent
  • Must be willing to attend all scheduled visits and complete study procedures, including multiple muscle biopsies

Who cannot join the trial?

15 criteria

  • Age below 18 years or over 65 years
  • Inability to provide informed consent
  • Pregnancy or breastfeeding
  • Participation in another clinical trial within the past 30 days
  • History of severe allergic reactions to medications
  • Significant heart, liver, or kidney problems
  • Use of medications that could interfere with the study drug
  • Active infections or major illnesses
  • History of blood clotting disorders (conditions that affect blood's ability to form clots)
  • Unstable medical conditions that require frequent medication changes
  • History of drug or alcohol abuse within the past year
  • Mental health conditions that could affect study participation
  • Previous adverse reactions to similar treatments
  • Inability to comply with study procedures or follow-up visits
  • Abnormal laboratory test results that indicate health concerns
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Investigated drugs

DYNE-101 is an investigational medication administered intravenously (through a vein) that is being studied for the treatment of Myotonic Dystrophy Type 1 (DM1), a genetic muscle disorder. This medication is designed to target muscle tissue in people with DM1. The drug is currently being tested to understand how safe it is, how well patients tolerate it, and how effectively it works in treating muscle symptoms in people with DM1.

What is already known about the treatment

DYNE-101 - An investigational medication administered intravenously (IV) that is currently being studied in clinical trials for the treatment of Myotonic Dystrophy Type 1 (DM1), a genetic muscle disorder. The drug is being evaluated in multiple ascending dose studies to assess its safety, tolerability, and effectiveness in treating muscle tissue symptoms in DM1 patients. While the exact molecular mechanism is still under investigation through clinical trials, DYNE-101 is specifically designed to target the underlying causes of muscle dysfunction in DM1, with researchers evaluating its pharmacodynamic effects on muscle tissue through controlled, placebo-compared studies.

Investigated diseases

Myotonic Dystrophy Type 1 - A genetic disorder that affects muscle function, causing progressive muscle weakness and wasting. The condition is characterized by myotonia, which means muscles have difficulty relaxing after contraction. It typically begins in adulthood and affects multiple body systems, including skeletal muscles, heart, eyes, and brain. The disease causes increasing weakness in the muscles of the face, neck, hands, and feet. People with this condition may also experience excessive daytime sleepiness, cataracts, and irregular heartbeats.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2023-510353-42-00Estimated enrolment186 patientsSponsorDyne Therapeutics Inc.

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