Gene Therapy for Duchenne Muscular Dystrophy

Recruiting at 12 trial locations

Overseen By Aravindhan Veerapandiyan, MD

Age: < 18

Sex: Male

Trial Phase: Phase 1 & 2

Sponsor: Solid Biosciences Inc.

Must be taking: Prednisone, Deflazacort

Must not be taking: Dystrophin modifying drugs

Disqualifiers: Gene transfer drugs, Exon 1-11, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 6

Duration: 1 day

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the safety and effectiveness of a one-time gene therapy treatment called SGT-003 for individuals with Duchenne muscular dystrophy (DMD). DMD is a genetic disorder that causes progressive muscle weakness and loss. The trial includes different groups based on age and walking ability. Individuals with a confirmed diagnosis of DMD who are not currently on certain other treatments may qualify for this study. Participants will remain in the trial for five years to monitor long-term effects. As a Phase 1/Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group.

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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that SGT-003, a gene therapy for Duchenne muscular dystrophy, was generally well tolerated in earlier studies. These studies used only steroids to manage the body's immune response. This approach appears to have protected the heart, which is crucial for overall heart health. Testing SGT-003 in a larger group now suggests that earlier results supported its potential safety. However, ongoing monitoring is essential to ensure it remains safe and tolerable for all participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about SGT-003 because it represents a groundbreaking approach to treating Duchenne Muscular Dystrophy (DMD). Unlike traditional treatments, which often focus on managing symptoms or slowing disease progression with corticosteroids, SGT-003 is a gene therapy that aims to address the root cause of the disease. It delivers a functional version of the dystrophin gene directly into the patient's muscle cells, potentially restoring the missing protein that causes muscle degeneration in DMD. This innovative mechanism of action offers hope for a more effective and lasting treatment, which could significantly improve quality of life for those affected by this condition.

What evidence suggests that SGT-003 might be an effective treatment for Duchenne muscular dystrophy?

Research has shown that SGT-003 is a promising gene therapy for treating Duchenne muscular dystrophy. It delivers a protein called microdystrophin, which helps muscles function better, using a specially designed virus. Early findings suggest that participants have generally tolerated SGT-003 well, with no major safety issues reported in earlier tests. The therapy aims to slow muscle weakening, offering hope for improved muscle strength and function. While more information is needed to confirm its efficacy, these early results are encouraging. Participants in this trial will receive a single IV infusion of SGT-003, with different cohorts based on age and ambulatory status. ^{2 4 6 7 8}

Who Is on the Research Team?

Solid Bio Clinical Trials

Principal Investigator

Solid Biosciences

Are You a Good Fit for This Trial?

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.

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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

Group I: Cohort 5: SGT-003 Experimental Treatment 1 Intervention

All non-ambulatory participants from age 10 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1.

Group II: Cohort 4: SGT-003 Experimental Treatment 1 Intervention

All ambulatory participants from age 12 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1.

Group III: Cohort 3: SGT-003 Experimental Treatment 1 Intervention

All participants from age 0 to \< 4 years will receive a single IV infusion of SGT-003 on Day 1.

Group IV: Cohort 2: SGT-003 Experimental Treatment 1 Intervention

All ambulatory participants from age 7 to \< 12 years will receive a single IV infusion of SGT-003 on Day 1.

Group V: Cohort 1: SGT-003 Experimental Treatment 1 Intervention

All ambulatory participants from age 4 to \< 7 years 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

Who Is Running the Clinical Trial?

Solid Biosciences Inc.

Lead Sponsor

Trials

4

Recruited

130+

Published Research Related to This Trial

Gene therapy for Duchenne muscular dystrophy (DMD) is being actively researched, with promising results from using modified adeno-associated viruses to deliver a mini-gene of dystrophin into muscle cells, which is expected to enter clinical trials soon.

Various methods, including viral and nonviral delivery systems, have been tested in biological models, highlighting the need for collaboration among Russian laboratories to advance these gene therapy experiments towards clinical application.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Current state and prospects for gene therapy of Duchenne muscular dystrophy in the world and in Russia]. Baranov, VS., Baranov, AN., Zelenin, AV.[2014]

Gene therapy using dystrophin cDNAs in dystrophin-deficient mice shows promise for treating Duchenne muscular dystrophy (DMD), with regional restoration of muscle morphology observed in transgenic models.

Direct injection methods yielded low expression rates of dystrophin in adult muscle fibers, but recombinant adenovirus vectors showed greater efficiency in young mice, highlighting the need for improved delivery systems for effective treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Gene therapy in Duchenne muscular dystrophy. Inui, K., Okada, S., Dickson, G.[2019]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular dystrophy. McKim, DA., Cripe, TP., Cripe, LH.[2021]

Implanting normal muscle precursor cells into dystrophin-deficient muscles in mice shows promise for treating Duchenne muscular dystrophy (DMD), but similar approaches in human patients have not been successful.

Gene therapy techniques, such as introducing dystrophin constructs via viral vectors, have been demonstrated in mice, but their efficiency and safety need thorough evaluation before being tested in humans.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cell and gene therapy in Duchenne muscular dystrophy. Morgan, JE.[2012]

Recent advancements in gene therapy for muscular dystrophy highlight the potential of plasmid DNA as a treatment option, overcoming previous challenges like low transfection efficiency and immune rejection.

A groundbreaking clinical trial using a human dystrophin plasmid and a novel intravascular delivery method has shown promise in achieving widespread muscle transfection, paving the way for effective treatments for Duchenne muscular dystrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Naked plasmid DNA for the treatment of muscular dystrophy. Braun, S.[2012]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

Recent advancements in gene delivery methods, particularly using adeno-associated viral vectors, show great promise for treating muscular dystrophies, although a complete cure has not yet been achieved.

Systemic administration of anti-sense oligonucleotides has successfully induced dystrophin expression and improved muscle health in canine models, highlighting a potential therapeutic approach for conditions like Duchenne muscular dystrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Therapeutic strategy for muscular dystrophies]. Shin, JH., Takeda, S.[2013]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Strategies for Bottlenecks of rAAV-Mediated Expression in Skeletal and Cardiac Muscle of Duchenne Muscular Dystrophy. Li, N., Song, Y.[2022]

Gene therapy using a 6.3 kb minidystrophin cDNA delivered via adenovirus was effective in treating muscular dystrophy in neonatal mdx mice, suggesting a potential approach for Duchenne muscular dystrophy (DMD).

However, adult mice experienced strong immune reactions to the adenovirus, indicating that using a modified adenovirus with dystrophin cDNA or upregulating endogenous utrophin could be safer and more effective strategies for treating DMD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Gene therapy to muscle diseases: perspective and issues on basic research]. Takeda, S.[2012]

Citations

1. investors.solidbio.com investors.solidbio.com/news-releases/news-release-details/solid-biosciences-reports-third-quarter-2025-financial-results

Solid Biosciences Reports Third Quarter 2025 Financial ... SGT-003 has been generally well tolerated using a steroid-only prophylactic immunomodulation regimen; cardiac safety monitoring continued to ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06138639

A Study of SGT-003 Gene Therapy in Duchenne Muscular ... This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of ...

3. parentprojectmd.org parentprojectmd.org/solid-biosciences-shares-update-on-inspire-duchenne-trial-of-sgt-003/

Solid Biosciences Shares Update on INSPIRE ... SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their novel AAV capsid designed to ...

4. dmdhub.org dmdhub.org/trials/a-study-of-sgt-003-gene-therapy-in-duchenne-muscular-dystrophy1-sgt-003-301/

A Study of SGT-003 Gene Therapy in Duchenne Muscular ... This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896625009150

22OUpdate on INSPIRE DUCHENNE: a phase 1/2 study of ... As of a data cut of March 13, 2025, SGT-003 has been well tolerated in the 7 participants who received SGT-003 at the single study dose level of 1E14 vg/kg, ...

6. parentprojectmd.org parentprojectmd.org/solid-biosciences-shares-interim-data-from-inspire-duchenne-trial-of-sgt-003/

Solid Biosciences Shares Interim Data from INSPIRE ... SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their novel AAV capsid, which is designed ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT07160634

NCT07160634 | A Study of SGT-003 Gene Therapy in ... A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE). ClinicalTrials.gov ID NCT07160634.

8. clinicaltrials.gov clinicaltrials.gov/study/NCT06138639

NCT06138639 | A Study of SGT-003 Gene Therapy in ... 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 ...