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

NCT07673809 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2026-06-29

No results posted yet for this study

Summary

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.

Conditions

Interventions

GENETIC

GNR-097

Single IV infusion of GNR-097 (recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (micro-dystrophin)).

GENETIC

Placebo followed by GNR-097

Single IV infusion of matching placebo followed by single IV infusion of GNR-097 at the beginning of the second year.

Sponsors & Collaborators

  • AO GENERIUM

    lead INDUSTRY

Principal Investigators

  • Oksana A. Markova, M.D. · AO GENERIUM

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
9 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-09-30
Primary Completion
2029-08-02
Completion
2029-08-02

Countries

  • Belarus
  • Russia

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07673809 on ClinicalTrials.gov