A Study Testing AOC 1020 to Improve Movement in Adults with Facioscapulohumeral Muscular Dystrophy

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What is this study about?

This study is looking at a condition called Facioscapulohumeral Muscular Dystrophy, also known as FSHD, which is a genetic disorder that causes progressive weakness of the muscles, particularly affecting the face, shoulder blades, and upper arms. The study will test a medication called AOC 1020, which is given through a vein as an infusion. Some participants will receive AOC 1020 while others will receive placebo or 0.9% Saline for IV administration. The purpose of the study is to evaluate how well AOC 1020 works on functional mobility, which means the ability to move and perform daily activities.

Participants in this study will be randomly assigned to receive either the active medication or placebo, and neither the participants nor the doctors will know which treatment is being given during the study. The study will last for 78 weeks, which is about 18 months. During this time, participants will receive regular infusions of the study medication and will be monitored to see how their condition changes. The main focus will be on measuring changes in walking speed over a distance of 10 meters, which helps doctors understand if the treatment is helping with movement and mobility.

Throughout the study, various assessments will be performed to measure different aspects of the disease, including how well the upper and lower body muscles are working, how long it takes to stand up and walk a short distance, and how the condition affects quality of life. Participants will also be asked about pain levels, fatigue, and their overall impression of how severe their symptoms are and whether they notice any changes. Blood samples will be taken to measure certain markers that can indicate disease activity and muscle damage.

1 Treatment period begins

Your participation in the study will begin once you have completed all required procedures before the treatment phase starts.

This study will compare the effects of AOC 1020 with a placebo (an inactive substance that looks like the study medication but contains no active ingredient). You will be randomly assigned to receive either AOC 1020 or placebo. Neither you nor your doctor will know which treatment you are receiving during the study.

2 Receiving the study medication

You will receive the assigned treatment through an intravenous infusion (a slow injection directly into your vein).

The study medication AOC 1020 is a specially designed antibody that targets a specific protein and delivers genetic material to help reduce the production of a protein called DUX4, which is involved in facioscapulohumeral muscular dystrophy.

If you are assigned to the placebo group, you will receive 0.9% saline solution (salt water) through the same intravenous infusion method.

The infusions will be administered at regular intervals throughout the study period.

3 Regular assessments and measurements

Throughout the study, various assessments will be performed to measure how the treatment affects your condition.

You will undergo a 10-meter walk or run test where your speed will be measured as you walk or run a distance of 10 meters. This test will be performed multiple times during the study.

A Timed-Up-and-Go test will be performed, which measures the time it takes for you to stand up from a chair, walk a short distance, turn around, walk back, and sit down again.

Your muscle strength will be tested using quantitative muscle testing, which measures the strength of different muscle groups in your arms and legs.

You will be asked to complete questionnaires about your upper body function, your ability to perform daily activities, and how fatigue affects you.

You will be asked to rate your pain levels using a numerical scale and provide your overall impression of how severe your condition is and whether it has changed.

Blood samples will be collected to measure specific markers related to the disease, including a protein called creatine kinase and other biological indicators.

4 Assessment at Week 78

At Week 78 (approximately 18 months after starting treatment), a comprehensive evaluation will be performed.

The primary measurement will be the change in your walking speed on the 10-meter walk or run test compared to your speed at the beginning of the study.

Additional measurements at this time point will include the Timed-Up-and-Go test, upper body function questionnaire, and muscle strength testing.

These assessments will help determine the effectiveness of the treatment on your functional mobility and overall condition.

5 Ongoing monitoring throughout the study

Between the start of treatment and Week 78, you will have multiple visits where the same assessments will be repeated to track changes over time.

Your safety will be monitored continuously throughout the study, and any side effects or changes in your health will be recorded.

The total duration of your participation in the study will extend until approximately April 2028, as this is when the study is expected to be completed.

Who Can Join the Study?

  • You must provide written informed consent, which means you sign and date a form showing you agree to join the study, and follow all study requirements. If you are under 18 years old, a parent or legal guardian must also give consent, and the study will be explained to you in a way that matches your age and understanding.
  • You must have Facioscapulohumeral Muscular Dystrophy, also called FSHD, confirmed by a genetic test. This means a laboratory test of your genes has proven you have this muscle condition. The test must show either FSHD1 or FSHD2, which are the two types of this condition.
  • You must be between 18 and 70 years old at the time you sign the consent form to join the study.
  • You must be able to walk on your own for at least 10 meters, which is about 33 feet. You may use orthoses, which are supportive devices like braces, or ankle braces to help you walk, but you should not need a walker or wheelchair for this distance.

Who Cannot Join the Study?

  • The source data does not contain specific exclusion criteria, which are reasons why a patient cannot participate in the study
  • Without detailed exclusion criteria information, it is not possible to list the specific conditions or situations that would prevent participation in this clinical trial
  • Exclusion criteria typically include things like other medical conditions, certain medications being taken, pregnancy status, or previous treatments, but none are specified in the provided information

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
Bellvitge University Hospital L'hospitalet De Llobregat Spain
Hospital Universitario Y Politecnico La Fe Valencia Spain
Universitaetsmedizin Goettingen Goettingen Germany

Other Sites

Site Name City Country Status
Azienda Ospedaliero-Universitaria Sant Andre Rome Italy
Stichting Radboud University Medical Center Nijmegen The Netherlands
Centre Hospitalier Universitaire De Montpellier Montpellier France
Rheinische Friedrich-Wilhelms-Universitaet Bonn Bonn Germany
Centre Hospitalier Universitaire De Nice Nice France
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH Ulm Germany
Hopital Beaujon Clichy France
Roskilde University Roskilde Denmark
Azienda Ospedaliero Universitaria Pisana Pisa Italy
Aarhus Universitethospital Aarhus Denmark
Czduzf Ckjkeql Nrfg Milan Italy
Lmbot Uziwfgsuvded Mfatycg Cseyexf (viqsd Leiden The Netherlands
Afpucvodzr Pcppmkdg Hytmqaln Db Myezvbqqs Marseille France
Hznluxhb Ueprymwiswtav Dheywlky Donostia / San Sebastian Spain
Klxhdhqg dmm Uznvkqtptgom Mvvvtwvg Ady Munich Germany
Hunayuqe Vrve dovjagtr Barcelona Spain

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

Trial status

Country Status Recruitment Start
Denmark Denmark
Recruiting
01.10.2025
France France
Not yet recruiting
01.10.2025
Germany Germany
Recruiting
01.10.2025
Italy Italy
Recruiting
01.10.2025
Spain Spain
Recruiting
01.10.2025
The Netherlands The Netherlands
Recruiting
01.10.2025

Trial locations

AOC 1020 is an investigational medication given through a vein (intravenously) that is being studied as a potential treatment for facioscapulohumeral muscular dystrophy, a genetic condition that causes progressive muscle weakness. This medication is designed to help improve how well patients can move and perform daily activities.

Placebo is an inactive substance that looks like the real medication but contains no active treatment. It is used in this study to compare against the actual medication to help determine if the medication being tested is truly effective.

Facioscapulohumeral Muscular Dystrophy – Facioscapulohumeral Muscular Dystrophy is a genetic disorder that causes progressive weakening of muscles. The condition typically begins by affecting the muscles of the face, shoulders, and upper arms. As the disease advances, weakness gradually spreads to the muscles of the abdomen, hips, and legs. The pattern of muscle weakness is often asymmetric, meaning one side of the body may be more affected than the other. People with this condition may experience difficulty raising their arms, climbing stairs, or walking. The progression of the disease varies significantly among individuals, with some experiencing mild symptoms while others develop more severe muscle weakness over time.

Trial ID:
2025-521012-18-00
Protocol code:
AOC 1020-CS3
NCT ID:
NCT07038200
Trial Phase:
Therapeutic confirmatory (Phase III)

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