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

2 1

What is this study about?

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.

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 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 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 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 Study?

  • 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 Study?

  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Universitaetsmedizin Goettingen Goettingen Germany
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy

Other Sites

Site Name City Country Status
Friedrich Baur Institute An Der Neurologischen Klinik Und Poliklinik Munich Germany
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Stichting Radboud University Medical Center Nijmegen The Netherlands
Centre Hospitalier Universitaire De Nantes Nantes France
Hopital Beaujon Clichy France
Czndhf Cqtvrtn Nbyx Milan Italy

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not recruiting
08.03.2023
Germany Germany
Not recruiting
08.03.2023
Italy Italy
Not recruiting
08.03.2023
The Netherlands The Netherlands
Not recruiting
08.03.2023

Trial locations

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.

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 ID:
2023-510353-42-00
NCT ID:
NCT05481879
Trial Phase:
Phase I and Phase II (Integrated) – First administration to humans

Other Trials to Consider

  • A study to evaluate the efficacy and safety of DYNE-101 in patients with Myotonic Dystrophy Type 1

    Recruiting

    3 1 1
    Investigated diseases:
    Belgium Denmark France Germany Italy The Netherlands +1
  • Study of intravenous AOC 1001 to evaluate long-term safety and effectiveness in patients with myotonic dystrophy type 1

    Recruiting

    3 1 1
    Denmark France Germany Italy The Netherlands Spain