Actively Recruiting
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Led by Precision BioSciences, Inc. · Updated on 2026-04-30
18
Participants Needed
1
Research Sites
188 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.
CONDITIONS
Official Title
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male participants aged 2 to 7 years at the time of consent
- Molecularly confirmed diagnosis of DMD with mutation between exons 45 to 55
- Clinical features consistent with DMD as determined by the investigator
- Ability to perform age-appropriate motor tests: for ages 2 to <4 years, walk at least 10 meters independently and rise from the floor without assistance; for ages 4 to 7 years, walk at least 100 meters independently and have an NSAA score between 16 and 29
- Received routine childhood immunizations appropriate for age according to local schedules
- Parent(s) or legally authorized representative willing and able to provide informed consent; participant assent when applicable
- Willingness to participate in long-term follow-up after trial completion
You will not qualify if you...
- Prior treatment with any gene therapy, gene editing, or cell-based therapy
- Use of any investigational medication or experimental therapy within 6 months before dosing
- Prior or current use of products to increase dystrophin expression, including exon-skipping therapies, within 6 months before dosing, or inability/unwillingness to avoid such therapies for 5 years after gene therapy
- Concurrent enrollment in another clinical trial except observational studies
- Positive test for antibodies to AAV9
- Any condition contraindicating immunosuppression treatment
- Pathogenic mutations outside exons 45 to 55 (exons 1-44 or 56-79)
- Evidence of cardiomyopathy or left ventricular dysfunction with LVEF below 50% on screening echocardiogram
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
Research Team
P
Precision BioSciences Clin Ops
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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