A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy

Sponsor
AO GENERIUM
Study ID
NCT07673809
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
MALE
Age
4 Years - 9 Years
Healthy Volunteers
Not accepted

Interventions

  • GNR-097 — GENETIC
    Single IV infusion of GNR-097 (recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (micro-dystrophin)).
  • Placebo followed by GNR-097 — GENETIC
    Single IV infusion of matching placebo followed by single IV infusion of GNR-097 at the beginning of the second year.

Study Details

The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD). In Phase I the participants will be included in two sequential dose cohorts with increasing doses of the investigational product. Based on the results of Phase I, the dose of the investigational product for use in Phase II will be determined. Phase II is a randomized, single-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.

Key Dates

Start date
Sep 30, 2025
Status verified
Jun 2026
Primary completion
Aug 2, 2029
Completion
Aug 2, 2029

Study Design

Enrollment
32 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: GNR-097
    Participants will receive single intravenous (IV) infusion of GNR-097 on Day 1.
  • Placebo Comparator: Placebo followed by GNR-097
    In Phase II participants will receive matching placebo IV infusion on Day 1. Then, they will have the opportunity to receive a single IV infusion of GNR-097 at the beginning of the second year.

Primary Outcome Measure

Number and percentage of participants with treatment-emergent adverse events (AEs), AEs of special interest and serious adverse events (SAEs) [ Time Frame: Baseline to End of Study (Week 104) ]

Central Contacts

Related Studies