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Gene Therapy

Gene Therapy for Duchenne Muscular Dystrophy (IGNITE DMD Trial)

Phase 1 & 2
Waitlist Available
Research Sponsored by Solid Biosciences Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
Adolescents must be non-ambulatory by protocol-specified criteria
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights

IGNITE DMD Trial Summary

This trial is testing a new drug for Duchenne muscular dystrophy in children and adolescents. The trial will last for approximately 5 years, and subjects will receive a single infusion of the drug.

Who is the study for?
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.Check my eligibility
What is being tested?
The study tests SGT-001, a gene therapy delivered through an IV infusion once. It aims to assess safety, tolerability, and effectiveness over about 5 years in DMD patients. Initially designed as controlled study but now all new participants receive the active treatment.See study design
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions related to immune response against the AAV9 vector used in gene transfer such as fever or muscle pain; liver enzyme elevations due to liver targeting of the vector; or other unforeseen complications.

IGNITE DMD Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with DMD and have a gene mutation linked to it.
Select...
I am a teenager who cannot walk.
Select...
My child can walk as required by the study.
Select...
My muscle biopsy showed I lack dystrophin.

IGNITE DMD Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Primary efficacy endpoint
Primary safety endpoint

IGNITE DMD Trial Design

3Treatment groups
Experimental Treatment
Active Control
Group I: SGT-001 - Dose Level 2Experimental Treatment1 Intervention
Single IV infusion of SGT-001 at next ascending dose
Group II: SGT-001 - Dose Level 1Experimental Treatment1 Intervention
Single IV infusion of SGT-001 at starting dose
Group III: Untreated ControlActive Control1 Intervention
Untreated control group. After 1 year, treatment-eligible control patients will receive SGT-001 at the selected dose.

Find a Location

Who is running the clinical trial?

Solid Biosciences Inc.Lead Sponsor
3 Previous Clinical Trials
83 Total Patients Enrolled
Solid Biosciences, LLCLead Sponsor
1 Previous Clinical Trials
60 Total Patients Enrolled
Roxana Donisa Dreghici, MDStudy DirectorSolid Biosciences

Media Library

SGT-001 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03368742 — Phase 1 & 2
Duchenne Muscular Dystrophy Research Study Groups: SGT-001 - Dose Level 2, SGT-001 - Dose Level 1, Untreated Control
Duchenne Muscular Dystrophy Clinical Trial 2023: SGT-001 Highlights & Side Effects. Trial Name: NCT03368742 — Phase 1 & 2
SGT-001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03368742 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Could you tell me how many hospitals are participating in this clinical trial?

"Five locations in totale are recruiting for this trial, these include University of Massachusetts Medical School-Worcester in Worcester, Massachusetts, David Geffen School of Medicine at UCLA in Los Angeles, California and University of Florida in Gainesville, Florida."

Answered by AI

Are participants still being enrolled in this research project?

"The information on clinicaltrials.gov reveals that this particular trial is not currently looking for patients. This study was originally posted on December 6th, 2017 and updated as recently as April 29th, 2022. However, there are 88 other ongoing trials that might be of interest."

Answered by AI

Does this research involve elderly participants?

"According to the inclusion criteria, people aged 4 to 17 are eligible for this particular study. Out of the total 121 studies, 61 focus on pediatrics while 50 investigate geriatrics."

Answered by AI

How can I sign up for this research opportunity?

"For this particular study, we are looking for 16 children and adolescents who have muscular dystrophies. These potential participants must also meet the following requirements: Adolescents - those that cannot walk by protocol-specified criteria."

Answered by AI

Who else is applying?

How old are they?
< 18
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
2017. "Microdystrophin Gene Transfer Study in Adolescents and Children With DMD". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03368742.
All > Muscular Dystrophy
~5 spots leftby Dec 2026
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