SGT-003 Gene Therapy for Duchenne Muscular Dystrophy

Overseen By Solid Bio Clinical Trials

Age: < 18

Sex: Male

Trial Phase: Phase 3

Sponsor: Solid Biosciences Inc.

Must be taking: Prednisone, Deflazacort

Must not be taking: Gene therapy, Dystrophin drugs

Disqualifiers: Gene therapy, Dystrophin drugs, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy treatment called SGT-003 for boys with Duchenne Muscular Dystrophy (DMD), a genetic disorder that causes muscle weakness. The study aims to evaluate the effectiveness of SGT-003 when administered as a single IV infusion and to assess its long-term safety. Participants will receive either SGT-003 first and a placebo later, or vice versa, and will be monitored for at least five years. Boys who can walk, have a confirmed DMD diagnosis, are on a stable steroid regimen, and meet certain physical criteria might be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

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 6 months before joining. If you're taking other medications, especially investigational drugs or certain DMD treatments, you may need to stop them before enrolling.

Is there any evidence suggesting that SGT-003 is likely to be safe for humans?

Research has shown that SGT-003, a gene therapy for Duchenne Muscular Dystrophy, is generally safe. In earlier studies, participants tolerated SGT-003 well. These studies also demonstrated that the therapy successfully increased the production of microdystrophin, a protein essential for proper muscle function.

Some side effects were reported, but they were mostly mild and manageable. No serious safety issues have emerged in the available data. The trial's progression to a later phase indicates that earlier testing confirmed its safety, which is a positive sign for potential participants. ^{1 2 3 4}

Why do researchers think this study treatment might be promising for Duchenne Muscular Dystrophy?

Researchers are excited about SGT-003 for Duchenne Muscular Dystrophy (DMD) because it uses a novel gene therapy approach, which is different from the standard steroid treatments that only help manage symptoms. SGT-003 targets the root cause by delivering a functional version of the dystrophin gene, which is missing or defective in DMD patients. This approach has the potential to significantly alter the course of the disease by addressing the genetic issue directly, offering hope for more lasting improvements in muscle function compared to existing therapies.

What evidence suggests that SGT-003 might be an effective treatment for Duchenne Muscular Dystrophy?

Research shows that SGT-003 gene therapy could help treat Duchenne muscular dystrophy (DMD). Earlier studies found that SGT-003 significantly increased microdystrophin, a smaller version of the protein dystrophin, which people with DMD lack. This protein helps protect muscles. SGT-003 also reduced signs of muscle damage in recipients. Other studies have shown that SGT-003 can improve blood flow to muscles, potentially preventing muscle breakdown. In this trial, participants will receive either SGT-003 followed by a placebo or a placebo followed by SGT-003. These findings suggest that SGT-003 could effectively treat DMD. ^{1 3 4 5 6}

Are You a Good Fit for This Trial?

This trial is for ambulant males with Duchenne Muscular Dystrophy (DMD) who can walk, have a confirmed diagnosis and specific gene mutation, are on stable steroids for at least 6 months, can rise from lying down within a set time, weigh ≤50 kg, and don't have antibodies against adeno-associated virus.

Inclusion Criteria

I have been diagnosed with DMD and have a gene mutation linked to it.

I don't have antibodies against adeno-associated virus.

I have been on a stable dose of prednisone or deflazacort for at least 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part 1 Treatment

Participants receive a single IV infusion of SGT-003 or placebo

1 day

1 visit (in-person)

Part 2 Treatment

Participants receive a single IV infusion of placebo or SGT-003

1 day

1 visit (in-person)

Long-term Follow-up

Participants are monitored for safety and effectiveness for at least 5 years after SGT-003 dosing

5 years

What Are the Treatments Tested in This Trial?

Interventions

• SGT-003

Trial Overview

The study tests SGT-003 gene therapy's effectiveness in improving DMD symptoms. Participants will receive either SGT-003 or placebo via IV infusion in a randomized order across two parts of the trial. They'll be monitored for at least five years after dosing.

How Is the Trial Designed?

Group I: SGT-003 followed by Placebo Experimental Treatment 2 Interventions

Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2.

Group II: Placebo followed by SGT-003 Experimental Treatment 2 Interventions

Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2.

Who Is Running the Clinical Trial?

Solid Biosciences Inc.

Lead Sponsor

Trials

4

Recruited

130+

Citations

1.

investors.solidbio.com

investors.solidbio.com/news-releases/news-release-details/solid-biosciences-reports-positive-initial-clinical-data-next

Solid Biosciences Reports Positive Initial Clinical Data from ...

About SGT-003​​ Nonclinical studies have shown that nNOS can improve blood flow to the muscle thereby reducing muscle breakdown from ischemia and ...

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 SGT-003 ...

3.

neurologylive.com

neurologylive.com/view/solid-reports-positive-data-sgt-003-gene-therapy-phase-1-2-trial-of-duchenne

Solid Reports Positive Data for SGT-003 Gene Therapy in ...

SGT-003 gene therapy showed significant microdystrophin expression and reduced muscle injury markers in Duchenne muscular dystrophy patients.

4.

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, ...

5.

contemporarypediatrics.com

contemporarypediatrics.com/view/craig-mcdonald-md-breaks-down-positive-sgt-003-data-for-duchenne-muscular-dystrophy

Craig McDonald, MD, breaks down positive SGT-003 data ...

In the phase 1/2 INSPIRE DUCHENNE trial, data from the first 3 patients dosed revealed an average microdystrophin expression of 110%, as ...

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 ...