A Phase 1/2a, Multi-center, Open-label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of PBGENE-DMD in Participants With Duchenne Muscular Dystrophy (FUNCTION-DMD)
Precision BioSciences, Inc.
Summary
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.
Description
This is a Phase 1/2a, open-label, multicenter trial designed to evaluate the safety, tolerability, and primary efficacy of a single IV dose of PBGENE-DMD in male participants with DMD presenting with mutations that may be amenable to treatment with PBGENE-DMD. A structured, multimodal, short-term immunomodulatory regimen will be administered around the time of dosing to mitigate the risk of potential immune-mediated responses. The trial consists of two parts: Part 1 is to confirm a safe and well-tolerated single dose of PBGENE-DMD that may be further evaluated in Part 2 (expansion). A total…
Eligibility
- Age range
- 2–7 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: 1. Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent 2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\]) 3. Clinical phenotype consistent with DMD in the opinion of the Investigator 4. Ability to complete age-appropriate motor testing assessments requirements. Participants aged 2 to \< 4 years at the time of screening must: 1. Be able to walk at least 10 meters independently (without assistive devices). 2. Be able to rise from the floor without physical assistance (use of a Gowers' m…
Interventions
- BiologicalPBGENE-DMD (IV)
Participants will receive a single dose of PBGENE-DMD
Location
- Arkansas Children's HospitalLittle Rock, Arkansas