Study on the Effects of Fordadistrogene Movaparvovec for Patients with Duchenne Muscular Dystrophy

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What is this study about?

This clinical trial is focused on studying a condition called Duchenne Muscular Dystrophy (DMD), which is a genetic disorder characterized by progressive muscle weakness and degeneration. The trial is testing a new treatment called Fordadistrogene Movaparvovec, also known by its code name PF-06939926. This treatment is a type of gene therapy that uses a specially designed virus to deliver a healthy version of the gene responsible for DMD into the patient’s body. The study will compare the effects of this treatment to a placebo to see if it can improve muscle function in patients with DMD.

The purpose of the study is to evaluate the safety and effectiveness of Fordadistrogene Movaparvovec in treating DMD. Participants in the study will receive the treatment through an intravenous infusion, which means it will be delivered directly into the bloodstream. The study will last for a period of time during which participants will be monitored for changes in their muscle function and overall health. The main goal is to see if the treatment can improve the ability to perform daily activities and increase muscle strength.

In addition to the main treatment, the study will also involve the use of Eculizumab, another medication that will be administered as a concentrate for infusion. This medication is being used to help understand its effects in combination with the main treatment. Throughout the study, participants will undergo various assessments to track their progress, including tests to measure muscle strength and function. The study aims to provide valuable information on the potential benefits of gene therapy for individuals with DMD.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including age, diagnosis, and current medication regimen.

Participants must be male, aged between 4 and 8 years, with a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD).

2 initial assessment

An initial assessment is conducted to establish a baseline for the North Star Ambulatory Assessment (NSAA) score, which measures physical abilities.

Participants must have an NSAA score between 16 and 30 and be able to walk 10 meters unassisted.

3 treatment administration

Participants receive either the investigational drug fordadistrogene movaparvovec or a placebo through intravenous infusion.

The treatment aims to improve muscle function and is administered as a solution for infusion.

4 ongoing assessments

Regular assessments are conducted to monitor changes in the NSAA score and other health indicators over a 52-week period.

These assessments include muscle biopsies and blood tests to measure mini-dystrophin expression and other biomarkers.

5 final evaluation

At the end of the study period, a final evaluation is conducted to assess the overall impact of the treatment on muscle function and health.

The primary outcome is the change in the NSAA score from the baseline to week 52.

Who Can Join the Study?

Male participants who are at least 4 years old but younger than 8 years at the first screening visit.
Must have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) through genetic testing, showing a specific change in the dystrophin gene. If the results are unclear, another test may be done at the screening visit.
Must have been taking a stable daily dose of glucocorticoids (a type of medication) for at least 3 months before the first screening visit and continue this dose until the third visit. The dose should not change by more than a small amount (0.2 mg/kg) during this time.
Must have a total score between 17 and 29 on the North Star Ambulatory Assessment (NSAA) at the first screening visit. This is a test that measures how well someone can move and walk.
Must be able to walk 10 meters without help at the first screening visit.
Participants and their legal representatives must be willing and able to attend all scheduled visits, follow the treatment plan, and undergo necessary tests and procedures, which may include muscle biopsies and heart scans under general anesthesia.
Participants and their legal representatives must be able to give their agreement and sign a consent form, which includes following the study’s rules and restrictions.
Participants and their legal representatives must agree not to seek certain sensitive clinical data on their own and not to share their experiences in the trial with others or on social media.

Who Cannot Join the Study?

Patients who are not male cannot participate in the study.
Patients who do not have Duchenne Muscular Dystrophy (DMD) cannot participate. DMD is a genetic disorder that causes muscle weakness.
Patients who are outside the specified age range for the study cannot participate. The age range is not specified here, but it is important for eligibility.
Patients who are part of a vulnerable population may not be eligible. A vulnerable population includes groups that might need special protection, like children or people 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
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Hopital Necker Enfants Malades	Paris	France
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Universitair Ziekenhuis Gent	Gent	Belgium
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Centre Hospitalier Universitaire De Nantes	Nantes	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Uwzqwaffyjgdiywwigrzt Ehiwd Azf	Essen	Germany
Hwdvrpcr Vgtx dkoguwdd	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	05.11.2020
France	Not recruiting	05.11.2020
Germany	Not recruiting	05.11.2020
Italy	Not recruiting	05.11.2020
Spain	Not recruiting	05.11.2020

Trial locations

Investigated drugs:

Eculizumab

PF-06939926: This is an investigational gene therapy being studied for the treatment of Duchenne Muscular Dystrophy. It is designed to deliver a copy of a gene that helps produce a protein called dystrophin, which is missing or not working properly in patients with this condition. The goal of this therapy is to improve muscle function and slow the progression of the disease.

Investigated diseases:

Duchenne muscular dystrophy

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. Symptoms usually begin in early childhood, with affected individuals experiencing difficulty in walking, running, and jumping. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to increased difficulty in movement. Over time, individuals may lose the ability to walk and require assistance with daily activities. The progression of muscle weakness can also affect the heart and respiratory muscles.

Trial ID:

2023-508510-42-00

Protocol code:

C3391003

NCT ID:

NCT04281485

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effects of Fordadistrogene Movaparvovec for Patients with Duchenne Muscular Dystrophy

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