PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexMale
Age2-7
SponsorPrecision BioSciences, Inc.
About this trial
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.
Eligibility criteria
Qualifiers
Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent
Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 [inclusive])
Clinical phenotype consistent with DMD in the opinion of the Investigator
Ability to complete age-appropriate motor testing assessments requirements.
Disqualifiers
Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time.
Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1.
Prior or ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose or inability or unwillingness to refrain from initiating or resuming these therapies for at least 5 years following gene therapy administration.
Prior ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose.
Trial design
Treatments tested in this trial
- PBGENE-DMD (IV)
Treatment groups
18 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov