43 Participants Needed

Gene Therapy for Duchenne Muscular Dystrophy

Recruiting at 5 trial locations
SB
Overseen BySolid Bio Clinical Trials
Age: < 18
Sex: Male
Trial Phase: Phase 1 & 2
Sponsor: Solid Biosciences Inc.
Must be taking: Prednisone, Deflazacort
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

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

SB

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

I can walk on my own without needing help from any device.
I weigh less than 18 kg.
I weigh less than 30 kg.
See 4 more

Exclusion Criteria

I have been treated with a gene therapy drug.
I have used drugs like eteplirsen for my condition.
Exposure to another investigational drug within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
See 1 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous infusion of SGT-003 on Day 1

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years
Multiple visits over 5 years

Treatment Details

Interventions

  • SGT-003
Trial Overview This study tests SGT-003 gene therapy given through an IV infusion once. It's divided into two age groups dosed one after the other, with a long-term follow-up of five years to check safety and effectiveness.
Participant Groups
2Treatment groups
Experimental Treatment
Group I: Cohort 2: SGT-003Experimental Treatment1 Intervention
All participants will receive a single IV infusion of SGT-003 on Day 1.
Group II: Cohort 1: SGT-003Experimental Treatment1 Intervention
All participants will receive a single IV infusion of SGT-003 on Day 1.

SGT-003 is already approved in United States for the following indications:

🇺🇸
Approved in United States as SGT-003 for:
  • Duchenne muscular dystrophy

Find a Clinic Near You

Who Is Running the Clinical Trial?

Solid Biosciences Inc.

Lead Sponsor

Trials
4
Recruited
130+

Findings from Research

Gene therapy shows promise in preventing muscle deterioration and improving ambulation in patients with Duchenne muscular dystrophy (DMD), although clinical trials are still in early stages.
There is a significant gap in understanding how gene therapy may impact cardiomyopathy and respiratory failure, which are major causes of mortality in DMD patients, highlighting the need for future studies to include respiratory endpoints as outcome measures.
The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular dystrophy.McKim, DA., Cripe, TP., Cripe, LH.[2021]
Duchenne muscular dystrophy (DMD) is a rare genetic disease affecting young boys, caused by defects in the dystrophin gene, which is essential for muscle stability, and currently has no cure.
Two promising therapeutic strategies being explored are AAV-mediated gene augmentation to provide a functional dystrophin and AON-mediated exon skipping to correct the gene's defects, with ongoing preclinical and clinical trials assessing their efficacy.
Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD).Kawecka, K., Theodoulides, M., Hasoglu, Y., et al.[2019]
Gene therapy using adeno-associated virus (rAAV) to deliver mini/micro-dystrophin shows promise for treating Duchenne Muscular Dystrophy (DMD), but high doses of rAAV can lead to immune responses and liver toxicity, especially in older patients.
Research suggests that using utrophin, a protein similar to dystrophin, along with a novel MyoAAV delivery system, could enhance therapeutic effects while reducing the required rAAV dose, potentially making gene therapy safer and more effective for a broader range of patients.
Strategies for Bottlenecks of rAAV-Mediated Expression in Skeletal and Cardiac Muscle of Duchenne Muscular Dystrophy.Li, N., Song, Y.[2022]

References

The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular dystrophy. [2021]
2.United Arab Emiratespubmed.ncbi.nlm.nih.gov
Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD). [2019]
Strategies for Bottlenecks of rAAV-Mediated Expression in Skeletal and Cardiac Muscle of Duchenne Muscular Dystrophy. [2022]
Gene therapy in Duchenne muscular dystrophy. [2019]
[Therapeutic strategy for muscular dystrophies]. [2013]
[Gene therapy to muscle diseases: perspective and issues on basic research]. [2012]
7.Russia (Federation)pubmed.ncbi.nlm.nih.gov
[Current state and prospects for gene therapy of Duchenne muscular dystrophy in the world and in Russia]. [2014]
Cell and gene therapy in Duchenne muscular dystrophy. [2012]
Naked plasmid DNA for the treatment of muscular dystrophy. [2012]
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top
Terms of Service·Privacy Policy·Cookies·Security