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.
Interested in this trial?
Submit an InquiryInterventions
GNR-097GENETIC
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-097GENETIC
Single IV infusion of matching placebo followed by single IV infusion of GNR-097 at the beginning of the second year.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Republican Scientific and Practical Center Mother and Child | Minsk | Belarus | |
| Russian Children's Clinical Hospital | Moscow | Russia | |
| National Medical Research Center for Children | Moscow | Russia | |
| Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University | Moscow | Russia | |
| Saint Petersburg State Pediatric Medical University | Saint Petersburg | Russia | |
| Regional Children's Clinical Hospital | Yekaterinburg | Russia |