Gene Therapy for Duchenne Muscular Dystrophy
Trial Summary
Will I have to stop taking my current medications?
The trial requires participants to be on a stable dose of prednisone or deflazacort for at least 12 weeks before joining. It does not specify if you need to stop other medications, but you cannot have taken certain drugs within 3 months before the trial.
What data supports the effectiveness of the treatment SGT-003 Gene Therapy for Duchenne Muscular Dystrophy?
Gene therapy using adeno-associated virus (AAV) to deliver dystrophin has shown promise in early studies for improving muscle function in Duchenne Muscular Dystrophy (DMD). Initial reports suggest potential benefits for movement, although more research is needed to understand its effects on heart and lung complications associated with DMD.12345
How is the SGT-003 treatment for Duchenne Muscular Dystrophy different from other treatments?
SGT-003 Gene Therapy is unique because it uses a modified virus to deliver a mini-gene version of the dystrophin gene directly to muscle cells, aiming for more efficient and targeted delivery compared to traditional methods. This approach is designed to overcome challenges like immune reactions and low gene expression seen with other gene therapy techniques.46789
What is the purpose of this trial?
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study. Cohort 1 will include participants 4 to \<7 years of age. Cohort 2 will include participants 7 to \<12 years of age. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
Research Team
Solid Bio Clinical Trials
Principal Investigator
Solid Biosciences
Eligibility Criteria
The INSPIRE DUCHENNE trial is for young boys with Duchenne muscular dystrophy. Cohort 1 includes ages 4 to under 6, weighing less than 18 kg and able to walk unaided. Cohort 2 will be ages 6 to under 8, weighing less than 30 kg. Participants must not have used certain other treatments for DMD or been in another study recently.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravenous infusion of SGT-003 on Day 1
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- SGT-003
SGT-003 is already approved in United States for the following indications:
- Duchenne muscular dystrophy
Find a Clinic Near You
Who Is Running the Clinical Trial?
Solid Biosciences Inc.
Lead Sponsor