A One-time Infusion of a Adeno-associated Virus (AAV) Vector Carrying a Microdystrophin Gene as a Therapy for Boys with Duchenne Muscular Dystrophy Aged 4 Through 11 Years
Phase: Recruiting
First Posted: April
Condition(s): Duchenne Muscular Dystrophy (DMD)
NCT Number: NCT06138639 Other Study ID Number(s): SGT-003-101
What Is the Purpose of This Study?
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of the microdystrophin gene therapy SGT-003 in participants with Duchenne muscular dystrophy (DMD).
Who Can Take Part in This Study?
- Cohort 1: 4 to <7 years of age
- Cohort 2: 7 to <12 years of age
- Participants who are ambulatory. (Ambulatory is defined as "being able to walk without the use of an assistive device.")
- Have an established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
- The mutation will be confirmed by genetic testing at trial screening.
- Boys will be excluded from the trial with any deletion mutation in exons 1 to 11 or 42 to 45 in the DMD gene.
- Negative for antibodies to the AAV virus (the SBL-103 virus)
- On a stable dose of at least 0.5 mg/kg/day of oral daily prednisone, or 0.75 mg/kg/day deflazacort, for ≥12 weeks prior to entering the study.
- Meet 10-meter walk/run time criteria
- Meet time to rise from supine criteria
- Participant has body weight: ≤50 kg
For a full list of eligibility requirements, visit https://clinicaltrials.gov/study/NCT06138639
What Will Happen During This Study?
- If determined to be eligible after screening is performed, participants will receive a single IV infusion of SGT-003.
- Study duration for each participant is 5 years, during which multiple visits to Nationwide Children's Hospital will be required.
Will We Be Paid for This Study?
No, participants will not be compensated for their time.
Principal Investigator
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Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare condition defined by worsening muscle weakness and damage. It is the most common inherited neuromuscular disorder that affects all races and ethnicities. DMD only affects males and children with DMD may lose the ability to walk as early as 7 years of age.
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