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
This trial does not accept healthy volunteersQualifiers
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
Design model
Single group
Treatments tested in this trial
PBGENE-DMD (IV)
Biological/VaccineParticipants will receive a single dose of PBGENE-DMD
Treatment groups
18 Participants
are divided into 1 treatment groupGroup A: Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohortExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events
Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment
Time frame
From Dosing through Week 104Secondary outcomes
1
Dystrophin expression in skeletal muscle
Measurement of Biologic activity of PBGENE-DMD through dystrophin expression in skeletal muscle
Time frame
Week 12, Week 52Other outcomes
1
Developmental Motor Function 1
Change from baseline in the digital mobility outcome (DMO) of Stride Velocity 95th Centile (SV95C)
Time frame
Week 52, Week 1042
Developmental Motor Function 2
Change from baseline in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Motor Gross Motor and Fine Motor scores (\<3 years of age)
Time frame
Week 52, Week 1043
Developmental Motor Function 3
Change from baseline (defined as the first assessment after the participant has reached 3 years of age) in North Star Ambulatory Assessment (NSAA) total score (≥ 3 years of age)
Time frame
Week 52, Week 1044
Developmental Motor Function 4
Change from baseline in participants ≥ 3 years of age in timed performance in 4-stair climb, time to rise (TTR), 10-meter walk/run (10MWR), 100-meter walk/run (only age \>/= 4 years of age)
Time frame
Week 52, Week 104Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov