Study on AOC 1001 for Treating Myotonic Dystrophy Type 1 in Patients

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What is this study about?

This clinical trial is focused on studying a condition called Myotonic Dystrophy Type 1, which is a genetic disorder that affects muscle function. The study is testing a new treatment called AOC 1001, which is given as a solution through an intravenous infusion. This means the treatment is delivered directly into the bloodstream through a vein. AOC 1001 is a special type of medication that combines a humanized antibody with a small piece of genetic material designed to target and reduce the effects of the disease.

The purpose of the study is to evaluate how effective AOC 1001 is in improving hand function in people with Myotonic Dystrophy Type 1. Participants in the study will receive either the AOC 1001 treatment or a placebo, which is a substance with no active medication. The study will last for a period of 54 weeks, during which participants will have regular visits to monitor their progress and assess any changes in their condition.

Throughout the study, researchers will focus on measuring changes in hand function, as well as other aspects of muscle strength and daily activities. The goal is to determine if AOC 1001 can help improve the quality of life for those living with Myotonic Dystrophy Type 1. Participants will be closely monitored to ensure their safety and to gather important information about the treatment’s effects.

1 joining the study

Upon joining the study, eligibility is confirmed based on criteria such as age (16 to 65 years, or 18 to 65 years in Denmark and Germany), a clinical and genetic diagnosis of myotonic dystrophy type 1, and the ability to walk independently for at least 10 meters.

2 treatment administration

The treatment involves the administration of AOC 1001, a solution for infusion, given intravenously. This medication is a humanized antibody linked to a specific genetic material designed to target myotonic dystrophy type 1.

The infusion is administered under controlled conditions to ensure safety and effectiveness.

3 monitoring and assessment

Throughout the study, regular assessments are conducted to monitor changes in hand function, specifically the time taken to open the hand, known as video Hand Opening Time (vHOT).

Additional assessments include hand grip strength, muscle strength scores, and overall health and quality of life evaluations.

4 evaluation of results

Primary evaluation occurs at Week 30, focusing on changes in hand function from the start of the study.

Secondary evaluations include various muscle strength and health assessments at Week 30 and Week 54, providing a comprehensive view of the treatment’s impact.

5 completion of the study

The study is expected to conclude by October 2026, with all data collected and analyzed to determine the efficacy and safety of AOC 1001 for treating myotonic dystrophy type 1.

Who Can Join the Study?

  • Age between 16 and 65 years old. In Denmark and Germany, the age range is 18 to 65 years old.
  • Have a clinical and genetic diagnosis of Myotonic Dystrophy Type 1. This means having a specific genetic change called a CTG repeat length of 100 or more.
  • Be able to walk on your own for at least 10 meters during the screening. You can use supports like orthoses or ankle braces if needed.

Who Cannot Join the Study?

  • Patients who have a different condition than Myotonic Dystrophy Type 1 cannot participate. This is a specific genetic disorder that affects muscles and other systems in the body.
  • Individuals who are not within the specified age range for the study are excluded. The age range is not specified here, but it means that only people of certain ages can join.
  • People who are not part of the specified clinical trial groups are excluded. This means that only certain groups of people, based on specific criteria, can participate.
  • Both females and males can participate, but if someone does not fit the gender criteria, they cannot join.
  • Individuals who are considered part of a vulnerable population are excluded. This term refers to groups of people who might be at a higher risk of harm or exploitation, such as children, pregnant women, or those with certain disabilities.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
University Hospital Maastricht Maastricht The Netherlands

Other Sites

Site Name City Country Status
Hopitaux Universitaires Pitie Salpetriere Paris France
Stichting Radboud University Medical Center Nijmegen The Netherlands
Roskilde University Roskilde Denmark
Beaumont Hospital Dublin Ireland
Cahjhq Cyufxvv Nguv Milan Italy
Axnkfiwlra Pyqnkzux Hokjqdmi Dm Mnxibnfay Marseille France
Aylzta Ualaxwdrhf Hhozyswq Aarhus Denmark
Hwlewvez Uhhctjzfyayel Ddevzdeo Donostia / San Sebastian Spain
Kvxihpvb dud Uyfebsdljhmc Mhdhmzgs Awd Munich Germany

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

Trial status

Country Status Recruitment Start
Denmark Denmark
Not recruiting
01.10.2024
France France
Not recruiting
01.10.2024
Germany Germany
Not recruiting
01.10.2024
Ireland Ireland
Not recruiting
01.10.2024
Italy Italy
Not recruiting
01.10.2024
Spain Spain
Not recruiting
01.10.2024
The Netherlands The Netherlands
Not recruiting
01.10.2024

Trial locations

AOC 1001 is an investigational medication being studied for its potential to improve hand function in patients with Myotonic Dystrophy Type 1. It is administered intravenously, meaning it is given directly into a vein. The study aims to determine how effective and safe this medication is for treating this specific condition.

Myotonic Dystrophy Type 1 – This is a genetic disorder characterized by progressive muscle wasting and weakness. It often begins in adulthood and affects the muscles of the face, neck, and limbs. Individuals with this condition may experience prolonged muscle contractions and have difficulty relaxing certain muscles after use. Over time, the disease can lead to muscle stiffness and weakness, impacting daily activities such as hand function. Other symptoms may include cataracts, heart conduction problems, and endocrine changes. The severity and progression of symptoms can vary widely among affected individuals.

Trial ID:
2024-511378-60-00
Protocol code:
AOC 1001-CS3
NCT ID:
NCT06411288
Trial Phase:
Therapeutic confirmatory (Phase III)

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