Study on the Safety and Effectiveness of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy

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What is this study about?

This clinical trial is focused on studying Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. The study will evaluate a treatment called Delandistrogene Moxeparvovec, also known by its code name SRP-9001. This treatment is a type of gene therapy, which involves introducing new genetic material into the body to help treat the disease. The trial will also use a placebo, which is a substance with no active therapeutic effect, to compare the results.

The purpose of the study is to assess the safety and effectiveness of Delandistrogene Moxeparvovec in both non-ambulatory (those who cannot walk) and ambulatory (those who can walk) participants with Duchenne Muscular Dystrophy. Participants will receive the treatment through an intravenous infusion, which means the solution is delivered directly into the bloodstream. The study will monitor changes in muscle function and other health indicators over a period of time, with key assessments occurring at 72 weeks after the start of the trial.

Throughout the study, researchers will track various health outcomes, including muscle strength and respiratory function, to determine the impact of the treatment. The trial will also measure the amount of dystrophin, a protein that is typically lacking in individuals with Duchenne Muscular Dystrophy, produced by the body after receiving the gene therapy. Participants will be closely monitored for any side effects or adverse reactions to ensure their safety during the trial.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including a diagnosis of Duchenne Muscular Dystrophy (DMD) and age requirements for certain groups.

Participants must have stable doses of oral corticosteroids and meet other health criteria.

2 randomization and treatment assignment

Participants are randomly assigned to receive either the gene therapy delandistrogene moxeparvovec or a placebo, which is a saline solution.

The treatment is administered through an intravenous infusion.

3 treatment administration

The gene therapy or placebo is given as a single dose through an intravenous infusion.

The infusion is a solution specifically prepared for this study.

4 monitoring and follow-up

Participants are monitored for changes in muscle function and other health indicators over a period of 72 weeks.

Regular assessments include tests of upper limb performance and lung function, as well as measurements of dystrophin protein levels.

5 safety and efficacy evaluation

The study evaluates the safety of the treatment by tracking any adverse events or side effects.

Efficacy is assessed by measuring improvements in muscle function and other health outcomes.

6 completion of the study

The study is expected to conclude in September 2027.

Final assessments will determine the overall impact of the treatment on participants’ health and quality of life.

Who Can Join the Study?

  • The patient must have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD), which is a genetic disorder that affects muscles, based on clinical findings and previous genetic tests.
  • For Cohort 1, the patient must be non-ambulatory, meaning they cannot walk, according to specific criteria in the study.
  • For Cohort 2, the patient must be able to walk and be between 8 and 18 years old at the time of screening.
  • The patient must be able to cooperate with tests that assess their ability to move.
  • The patient must have been taking a stable daily dose of oral corticosteroids (a type of medication) for at least 12 weeks before the screening, and the dose should remain the same during the study, except for changes due to weight adjustments.
  • The patient should not have elevated levels of antibodies against Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74), which is a type of virus used in gene therapy, as specified by the study requirements.
  • The patient must have a specific type of genetic mutation called a pathogenic frameshift mutation or a premature stop codon between exons 18 and 79. These are changes in the DNA that affect how proteins are made in the body.

Who Cannot Join the Study?

  • Only males can participate in the study. Females are not eligible.
  • Participants must have Duchenne Muscular Dystrophy, a specific muscle condition.
  • The study is for a specific age group, so participants must be within the required age range.
  • Participants should not belong to any vulnerable population that might be at risk during the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Hopital Des Enfants Toulouse France
IRCCS Istituto Giannina Gaslini Genoa Italy
Universitair Ziekenhuis Gent Gent Belgium
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Centre Hospitalier Regional De La Citadelle Liege Belgium
Hospital Universitario 12 De Octubre Madrid Spain
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Karolinska University Hospital Solna Sweden
Centre Hospitalier Lyon Sud Pierre Benite France
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Uovkbhgtco Moazqho Csoozw Hohypvhfnodwvlzbs Hamburg Germany
Uninheimmnmjleejzmwxw Evrmd Aaa Essen Germany
Khlxuhff dwb Udzltmikmzob Mxbgejjk Aqi Munich Germany

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

Trial status

Country Status Recruitment Start
Belgium Belgium
Not yet recruiting
22.05.2023
France France
Not yet recruiting
22.05.2023
Germany Germany
Not yet recruiting
22.05.2023
Italy Italy
Not yet recruiting
22.05.2023
Spain Spain
Not yet recruiting
22.05.2023
Sweden Sweden
Not yet recruiting
22.05.2023

Trial locations

Investigated drugs:

SRP-9001 is a gene transfer therapy being studied for its potential to treat Duchenne Muscular Dystrophy. This therapy involves delivering a specific gene to the body to help produce a protein that is missing or not working properly in patients with this condition. The goal is to improve muscle function and slow the progression of the disease.

Investigated diseases:

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive muscle weakness and degeneration. 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 performing daily activities. Over time, individuals may lose the ability to walk and require assistance with mobility. The progression of muscle weakness can also impact respiratory and cardiac functions.

Trial ID:
2024-512626-28-00
Protocol code:
SRP-9001-303
NCT ID:
NCT05881408
Trial Phase:
Therapeutic confirmatory (Phase III)

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