Duchenne Muscular DystrophyPhase 1/2NCT07673809
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
A Phase 1/2 study testing GNR-097 in people with Duchenne Muscular Dystrophy, run by AO GENERIUM at 6 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 9 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- GNR-097
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 30 Sep 2025to 2 Aug 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 9 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Written informed consent for participation in the trial.
- Ambulatory boys aged 4-9 years with a documented diagnosis of DMD and clinical manifestations of the disease.
- A frameshift mutation or nonsense mutation in the DMD gene.
- Сreatine phosphokinase level >5000 U/L.
- Binding antibody titer to AAV9 ≤1:50 [method: ELISA].
- The patient is able to interact with the study physician and perform tests to assess functional activity.
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- Results of functional activity assessment tests at screening (at least in one of the two attempts performed on different days):
- NSAA ≥22;
- time to rise from a supine position without using surrounding objects or furniture <5 sec;
- 6MWT distance ≥350 m.
- The patient received oral glucocorticosteroids at a stable dose for ≥12 weeks prior to signing the Informed Consent Form, and it is planned that glucocorticosteroids will be continued during the screening stage and after the patient's inclusion in the study.
- For patients receiving deflazacort at study entry: switching the patient from deflazacort to prednisolone, in the opinion of the investigator, will not result in a significant deterioration in the patient's health.
- The patient has been immunized with a vaccine against meningococcal serotypes A, C, Y, W135 (and B, if available) no later than 4 weeks prior to administration of GNR-097/placebo, and the immunization period expires no more than three months after the expected date of administration of GNR-097/placebo.
Who cannot join
- Hypersensitivity to any component of GNR-097 or placebo.
- Patient with cognitive impairment or a sedentary lifestyle that, in the opinion of the investigator, may interfere with the development or manifestation of motor activity.
- Mutations in exons 8 and/or 9 of the DMD gene; for patients planned for inclusion in Cohort A, additionally: mutations in exons 1-17 and/or 59-71 of the DMD gene.
- Clinical signs of cardiomyopathy, including left ventricular ejection fraction (Simpson) <40% based on echocardiography performed during screening.
- Contraindications to magnetic resonance imaging.
- History of any autoimmune disease, with the exception of drug-compensated autoimmune thyroiditis.
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- History of tuberculosis; positive or indeterminate result of Diaskintest® TigraTest® or T-SPOT.TB screening.
- Positive results of tests for hepatitis B, hepatitis C, or HIV screening.
- Acute infectious diseases that resolved less than 4 weeks before administration of GNR-097/placebo.
- Immunization with a live attenuated vaccine less than 3 months before administration of GNR-097/placebo OR immunization with any inactivated vaccine less than 4 weeks before administration of GNR-097/placebo.
- Abnormal laboratory parameters:
- GGT level is more than three upper limits of normal;
- total bilirubin >50.0 μmol/L (except for patients with a confirmed diagnosis of Gilbert's syndrome);
- creatinine >160.0 μmol/L;
- hemoglobin <80 or >180 g/L;
- white blood cell count >18,500/μL;
- platelet count below the lower limit of normal.
- History of taking antisense oligonucleotides, ataluren, gene therapy using vector constructs, or cell therapy.
- Use of immunosuppressive drugs other than glucocorticosteroids less than 12 weeks prior to signing the Informed Consent Form.
- Participation in clinical trials less than 6 months prior to signing the Informed Consent Form.
- Unwillingness or inability of the patient and/or their parent/legal guardian to comply with the protocol requirements and/or the trial procedures.
- Other diseases or conditions not listed above that, in the opinion of the physician investigator and/or the Sponsor, prevent the patient from participating in the trial, including for safety reasons.
Trial locations and contacts
6 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 2 countries
Russia5 sites, 5 recruiting
- Recruiting
Russian Children's Clinical Hospital
Moscow, 117513
Svetlana V. Mikhailova, Dr. Med. Sci.
- Recruiting
National Medical Research Center for Children
Moscow, 119991
Lyudmila M. Kuzenkova, Dr. Med. Sci., professor
- Recruiting
Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University
Moscow, 125412
Dmitry V. Vlodavets, Dr. Med. Sci.
- Recruiting
Saint Petersburg State Pediatric Medical University
Saint Petersburg, 194100
Vasily M. Suslov, Ph.D.
- Recruiting
Regional Children's Clinical Hospital
Yekaterinburg, 620149
Olga A. Lvova, Dr. Med. Sci.
Belarus1 site, 1 recruiting
- Recruiting
Republican Scientific and Practical Center Mother and Child
Minsk, 220053
Irina V. Zhevneronok, Ph.D.
About this study
Official title: Multicenter, Single-blind, Randomized, Placebo-controlled Study of a Single Intravenous Infusion of a Gene Therapy Product GNR-097 in Pediatric Patients With Duchenne Muscular Dystrophy
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
- Duchenne Muscular Dystrophy
- Being tested
- GNR-097, Placebo followed by GNR-097
- Keywords
- Duchenne Muscular Dystrophy, DMD, Ambulatory, Gene Therapy, Micro-dystrophin, AAV, AAV9
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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 group | Role | What they receive |
|---|---|---|
| GNR-097 | Experimental | Participants will receive single intravenous (IV) infusion of GNR-097 on Day 1. Receives: Genetic: GNR-097 |
| Placebo followed by GNR-097 | Placebo comparator | 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. Receives: Genetic: Placebo followed by GNR-097 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and percentage of participants with treatment-emergent adverse events (AEs), AEs of special interest and serious adverse events (SAEs)
AEs of special interest include immune-mediated myositis, myocarditis, thrombotic microangiopathy and hemolytic uremic syndrome
Measured: Baseline to End of Study (Week 104)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2026
- Study start30 Sep 2025
- Primary completion2 Aug 2029 (estimated)
- Study completion2 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- In phase I participants are included sequentially. In phase II participants are randomized to two groups.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AO GENERIUM
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Oksana A. Markova, M.D., Study chair, AO GENERIUM
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07673809
- Sponsor's protocol ID
- GNR097-DMD01
- Status last verified
- Jun 2026
- First submitted
- 23 Jun 2026
- Last updated
- 29 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Single-blind, Randomized, Placebo-controlled Study of a Single Intravenous Infusion of a Gene Therapy Product GNR-097 in Pediatric Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT07673809
- Acronym / sponsor ID
- GNR097-DMD01
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, DMD, Ambulatory, Gene Therapy, Micro-dystrophin, AAV, AAV9
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- AO GENERIUMIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2025 (actual)
- Primary completion
- 2 Aug 2029 (estimated)
- Study completion
- 2 Aug 2029 (estimated)
- First posted
- 29 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
- Model description
- In phase I participants are included sequentially. In phase II participants are randomized to two groups.
| Arm | Type | Receives |
|---|---|---|
| GNR-097 | Experimental | Genetic: GNR-097Participants will receive single intravenous (IV) infusion of GNR-097 on Day 1. |
| Placebo followed by GNR-097 | Placebo comparator | Genetic: Placebo followed by GNR-097In 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. |
Who may qualify
- Ages
- 4 to 9 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (13 listed)
- Written informed consent for participation in the trial.
- Ambulatory boys aged 4-9 years with a documented diagnosis of DMD and clinical manifestations of the disease.
- A frameshift mutation or nonsense mutation in the DMD gene.
- Сreatine phosphokinase level >5000 U/L.
- Binding antibody titer to AAV9 ≤1:50 [method: ELISA].
Key exclusion criteria (22 listed)
- Hypersensitivity to any component of GNR-097 or placebo.
- Patient with cognitive impairment or a sedentary lifestyle that, in the opinion of the investigator, may interfere with the development or manifestation of motor activity.
- Mutations in exons 8 and/or 9 of the DMD gene; for patients planned for inclusion in Cohort A, additionally: mutations in exons 1-17 and/or 59-71 of the DMD gene.
- Clinical signs of cardiomyopathy, including left ventricular ejection fraction (Simpson) <40% based on echocardiography performed during screening.
- Contraindications to magnetic resonance imaging.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GNR-097Single IV infusion of GNR-097 (recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (micro-dystrophin)). | Genetic | GNR-097 |
| Placebo followed by GNR-097Single IV infusion of matching placebo followed by single IV infusion of GNR-097 at the beginning of the second year. | Genetic | Placebo followed by GNR-097 |
Primary outcome
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)
AEs of special interest include immune-mediated myositis, myocarditis, thrombotic microangiopathy and hemolytic uremic syndrome
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Oksana A. Markova, M.D. study chair
AO GENERIUM
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GNR-097 in people with Duchenne Muscular Dystrophy, run by AO GENERIUM at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Russia, Belarus. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Elena I. Zagoruyko, M.D. (+7 (926) 344 84 48, eizagoruiko@generium.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jun 2026.