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Gene Therapy

Gene Therapy for Duchenne Muscular Dystrophy

Phase 1 & 2
Recruiting
Research Sponsored by Solid Biosciences Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ambulatory as defined as 'being able to walk without the use of an assistive device.'
Cohort 1: <18 kg body weight
Must not have
Current or prior treatment with an approved or investigational gene transfer drug.
Current or prior treatment with approved or investigational dystrophin modifying drugs such as eteplirsen, golodirsen, casimersen, and viltolarsen.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 90, day 360
Awards & highlights
No Placebo-Only Group

Summary

This trial will assess the safety and effectiveness of SGT-003, an IV infusion, in treating Duchenne muscular dystrophy in children ages 4-8. All participants will be monitored for 5 years.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects are not listed here, gene therapies like SGT-003 can potentially cause immune reactions, including responses against the virus vector or newly produced proteins, as well as general symptoms such as fever or fatigue.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can walk on my own without needing help from any device.
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I weigh less than 18 kg.
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I weigh less than 30 kg.
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I am between 4 and 6 years old.
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I am between 6 and 7 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have been treated with a gene therapy drug.
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I have used drugs like eteplirsen for my condition.
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My DMD is linked to specific gene deletions, confirmed by tests.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 90, day 360
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 90, day 360 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Change from baseline in North Star Ambulatory Assessment (NSAA) total score
Change from baseline in microdystrophin protein levels
Change from baseline in stride velocity 95th centile (SV95C)

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

Find a Location

Who is running the clinical trial?

Solid Biosciences Inc.Lead Sponsor
3 Previous Clinical Trials
89 Total Patients Enrolled
Solid Bio Clinical TrialsStudy DirectorSolid Biosciences
1 Previous Clinical Trials
12 Total Patients Enrolled
~29 spots leftby May 2027
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