Gene Therapy for Duchenne Muscular Dystrophy
(IGNITE DMD Trial)
Trial Summary
Do I have to stop taking my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications, but it does require a stable daily dose of oral corticosteroids for at least 12 weeks before joining. It's best to discuss your specific medications with the trial team.
What data supports the effectiveness of the treatment SGT-001 for Duchenne Muscular Dystrophy?
Research shows that gene therapy using a virus to deliver micro-dystrophin, a smaller version of the dystrophin protein, can help treat Duchenne Muscular Dystrophy. In studies, this approach has shown promise in increasing dystrophin levels in muscle cells, which is important for muscle function.12345
Is gene therapy for Duchenne Muscular Dystrophy generally safe in humans?
Research shows that gene therapy using adeno-associated virus (AAV) vectors for Duchenne Muscular Dystrophy has been well tolerated in animal studies, with no significant adverse effects observed. Early human trials are focused on safety, and while some immune responses have been noted, the therapy is considered promising and generally safe.12678
How is the treatment SGT-001 for Duchenne Muscular Dystrophy different from other treatments?
SGT-001 is a gene therapy that uses a viral vector to deliver a micro-dystrophin gene to muscle cells, aiming to produce a functional version of the dystrophin protein, which is missing in Duchenne Muscular Dystrophy. This approach is unique because it targets the root genetic cause of the disease, unlike traditional treatments that mainly address symptoms.147910
What is the purpose of this trial?
This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years.The protocol was amended to drop the control arm after 4 participants were dosed. Participants currently enrolling are assigned to active treatment. Control participants enrolled under original protocol will continue through the study per the original protocol.
Research Team
Solid Bio Clinical Trials
Principal Investigator
Solid Biosciences
Eligibility Criteria
This trial is for children and adolescents with Duchenne muscular dystrophy (DMD), who have a confirmed diagnosis, lack dystrophin protein, and meet specific criteria regarding antibody levels, heart and lung function. Participants must be on stable corticosteroid treatment. Those with significant health issues or recent exposure to certain drugs are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravenous (IV) infusion of SGT-001
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- SGT-001
Find a Clinic Near You
Who Is Running the Clinical Trial?
Solid Biosciences Inc.
Lead Sponsor
Solid Biosciences, LLC
Lead Sponsor