Study on the Safety and Efficacy of Delandistrogene Moxeparvovec and Imlifidase for Patients with Duchenne Muscular Dystrophy with Pre-existing Antibodies

1 1 1

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 explore the effects of a treatment called Delandistrogene moxeparvovec, also known by its code name SRP-9001. This treatment is a type of gene therapy that aims to deliver a functional version of the dystrophin gene to muscle cells. Participants in the study will also receive Imlifidase, a medication that helps reduce antibodies that might interfere with the gene therapy. The purpose of the study is to evaluate the safety and effectiveness of this combined treatment approach in individuals with DMD who have pre-existing antibodies to the virus used in the gene therapy.

During the study, participants will receive the treatments through an intravenous infusion, which means the medication is delivered directly into the bloodstream. The study will monitor changes in the amount of dystrophin protein in muscle tissue, as this protein is crucial for muscle function. The study will also assess the presence of the gene therapy in muscle tissue and the body’s response to the treatment over time. Participants will be observed for any side effects or adverse reactions to ensure the treatment is safe and well-tolerated.

The study will take place over several weeks, with regular check-ups and assessments to track the progress and effects of the treatment. The goal is to determine if the combination of Delandistrogene moxeparvovec and Imlifidase can improve muscle function and slow down the progression of Duchenne muscular dystrophy in those with specific antibodies. This research could provide valuable insights into new treatment options for individuals living with this challenging condition.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying a diagnosis of Duchenne muscular dystrophy (DMD) and checking for elevated rAAVrh74 antibody titers.

Participants must be able to cooperate with motor assessment testing and have a stable daily dose of oral corticosteroids for at least 12 weeks prior to the study.

2 imlifidase administration

Participants receive an infusion of Imlifidase, a medication administered intravenously. This step is crucial for reducing pre-existing antibodies to the gene therapy vector.

The maximum observed plasma concentration of Imlifidase is monitored up to Day 7, and total IgG levels in serum are measured up to Week 12.

3 gene therapy administration

Following the Imlifidase infusion, participants receive Delandistrogene moxeparvovec (SRP-9001) intravenously. This gene therapy aims to increase dystrophin protein expression in muscle tissue.

The concentration of vector genome copies in serum is measured up to Day 7 after administration.

4 monitoring and follow-up

Participants undergo regular monitoring to assess the safety and efficacy of the treatment. This includes measuring changes in dystrophin protein expression in muscle biopsies at Baseline and Week 12.

The study also tracks any treatment-emergent adverse events, adverse events of special interest, and serious adverse events up to Week 104.

5 study completion

The study is estimated to conclude by June 30, 2027. Participants will have contributed valuable data on the safety and efficacy of the gene therapy for Duchenne muscular dystrophy.

Who Can Join the Study?

The patient must be able to walk, as specified by the study’s criteria.
The patient must have a confirmed diagnosis of Duchenne muscular dystrophy (DMD) before the screening, based on clinical findings and genetic testing.
The patient must be able to cooperate with tests that assess movement abilities.
The patient must have certain levels of antibodies, which are proteins made by the immune system, as required by the study.
The patient must have a specific type of genetic change in the DMD gene that leads to the absence of a protein called dystrophin.
The patient must have been taking a stable daily dose of oral corticosteroids, which are medications that reduce inflammation, for at least 12 weeks before the screening. The dose should stay the same during the study, except for changes due to weight adjustments.
The patient must be male.

Who Cannot Join the Study?

Only male participants are allowed, so females cannot participate.
Participants must be within a specific age range, so those outside this range cannot join.
Individuals who do not have Duchenne muscular dystrophy (DMD) cannot participate.
Participants must not belong to certain vulnerable groups that are not specified for this study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name	City	Country	Status
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain

Trial status

Country	Status	Recruitment Start
Spain	Not recruiting	17.01.2024

Trial locations

Investigated drugs:

SRP-9001 is a gene therapy designed to deliver a specific gene to muscle cells in patients with Duchenne Muscular Dystrophy. This therapy aims to help the body produce a protein called dystrophin, which is typically lacking in individuals with this condition. The goal is to improve muscle function and slow the progression of the disease.

Imlifidase is an enzyme used to reduce the levels of certain antibodies in the body. In this trial, it is used to help patients who have pre-existing antibodies that might otherwise interfere with the effectiveness of the gene therapy SRP-9001. By lowering these antibodies, imlifidase helps ensure that the gene therapy can work more effectively.

Duchenne muscular dystrophy (DMD) – Duchenne muscular dystrophy 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, frequent falls, and challenges in climbing stairs. 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 the use of a wheelchair. The progression of muscle weakness can also affect the heart and respiratory muscles.

Trial ID:

2024-512624-11-00

Protocol code:

SRP-9001-104

NCT ID:

NCT06241950

Trial Phase:

Human Pharmacology (Phase I) – Other

Other Trials to Consider

#1 Clinical Trials Search in Europe

Study on the Safety and Efficacy of Delandistrogene Moxeparvovec and Imlifidase for Patients with Duchenne Muscular Dystrophy with Pre-existing Antibodies

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?