Study on the Effects and Safety of RO7204239 for Patients with Facioscapulohumeral Muscular Dystrophy

What is this study about?

This clinical trial is focused on studying Facioscapulohumeral Muscular Dystrophy (FSHD), a genetic disorder that causes muscle weakness and wasting. The study will evaluate a treatment called RO7204239, which is a type of protein known as a humanised IgG1 monoclonal antibody. This protein is designed to target and interact with a specific substance in the body called latent myostatin, which is involved in muscle growth and development. The trial will compare the effects of RO7204239 with a placebo to understand its impact on the body and its safety.

The purpose of the study is to assess how RO7204239 affects the muscles of participants with FSHD, using imaging techniques like Magnetic Resonance Imaging (MRI). Participants will receive the treatment as a solution for injection under the skin. The study will monitor various health indicators, including muscle volume and any side effects, over a period of time. The trial is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo, to ensure unbiased results.

Throughout the study, participants will undergo regular health checks, including assessments of their vital signs, heart function, and laboratory tests. The trial aims to provide valuable information on the safety and potential benefits of RO7204239 for individuals with FSHD, contributing to the understanding and management of this condition. The study is expected to continue until the end of 2025.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria such as age, genetic confirmation of facioscapulohumeral muscular dystrophy (FSHD), and ability to walk unassisted for 10 meters.

Participants agree to maintain their current level of physical activity throughout the study.

2 initial assessment

An initial assessment is conducted to establish baseline measurements. This includes magnetic resonance imaging (MRI) to evaluate muscle volume and fat content, as well as blood tests to measure levels of myostatin, a protein related to muscle growth.

3 treatment administration

Participants receive either the investigational drug RO7204239 or a placebo. The drug is a humanized monoclonal antibody administered via subcutaneous injection.

The frequency and dosage of the injections are determined by the study protocol and are administered over a specified period.

4 ongoing monitoring

Throughout the study, participants undergo regular monitoring to assess the effects of the treatment. This includes repeated MRI scans, blood tests, and evaluations of vital signs and physical health.

The study aims to observe changes in muscle volume, fat content, and the presence of any adverse effects.

5 final assessment

At the end of the study period, a final assessment is conducted. This includes a comprehensive review of all collected data to evaluate the safety and effectiveness of the treatment.

Participants’ health and any changes in their condition are documented and analyzed.

Who Can Join the Study?

Must be between 18 and 65 years old at the time of signing the consent form.
Must have a genetic confirmation of either FSHD1 or FSHD2. This means a test has shown you have a specific type of muscular dystrophy.
Must have clinical findings that match FSHD, as judged by the doctor conducting the study.
Must be able to walk 10 meters without help. This is checked using a test called the Timed 10-Meter Walk Test.
Must have a score between 2.5 and 4 on the Ricci Clinical Severity Scale. This is a way to measure how severe the condition is.
Must agree to keep the same level and type of physical therapy, occupational therapy, and exercise during the study.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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

Other Sites

Site Name	City	Country	Status
Rigshospitalet	Copenhagen	Denmark
Cglodf Cuqxtel Nykw	Milan	Italy

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	07.02.2023
Italy	Not recruiting	07.02.2023

Trial locations

RO7204239 is an investigational medication being studied for its effects on facioscapulohumeral muscular dystrophy, a condition that causes muscle weakness. The trial aims to understand how this medication works in the body, its safety, and how well it is tolerated by participants. Researchers are using magnetic resonance imaging (MRI) to observe changes in the muscles and assess the medication’s impact.

Investigated diseases:

Facioscapulohumeral muscular dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) – This is a genetic muscle disorder characterized by progressive muscle weakness and wasting. It primarily affects the muscles of the face, shoulders, and upper arms, leading to difficulties in facial expressions, lifting the arms, and other movements. The progression of muscle weakness can vary significantly among individuals, with some experiencing mild symptoms and others facing more severe muscle loss. Over time, the weakness may extend to other muscle groups, including those in the lower body. The disease often begins in adolescence or early adulthood, but the age of onset and rate of progression can differ widely. FSHD is caused by genetic changes that affect the DUX4 gene, leading to muscle cell damage.

Trial ID:

2022-503117-36-00

Protocol code:

BN43703

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effects and Safety of RO7204239 for Patients with Facioscapulohumeral Muscular Dystrophy

What is this study about?

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