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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 90, day 360
Awards & highlights

Study 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.Check my eligibility
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.See study design
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 have been on a stable dose of prednisone or deflazacort for at least 12 weeks.
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I am between 4 and 6 years old.
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I am between 6 and 7 years old.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of treatment-emergent adverse events (AEs)
Secondary outcome measures
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)

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
93 Total Patients Enrolled
Solid Bio Clinical TrialsStudy DirectorSolid Biosciences

Frequently Asked Questions

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

Is recruitment for this experiment still in progress?

"The information hosted on clinicaltrials.gov states that no participants are necessary for this particular medical trial at the moment, despite it being first posted on January 15th 2024 and last edited November 14th 2023. However, there are 91 other studies actively recruiting right now."

Answered by AI

Are octogenarians eligible to participate in this trial?

"To be eligible for this particular trial, applicants must fall between the ages of 4 and 7. Simultaneously, there are 61 studies aimed at children under 18 years old as well as 54 other trials targeting elderly people over 65."

Answered by AI

Is it possible for me to participate in this clinical investigation?

"To fulfill the eligibility criteria of this research, participants need to be diagnosed with duchenne muscular dystrophy and between 4-7 years old. Approximately 6 individuals must take part in the trial for it to proceed."

Answered by AI

Who else is applying?

What site did they apply to?
Nationwide Children's Hospital
What portion of applicants met pre-screening criteria?
Did not meet criteria
~4 spots leftby Jan 2026