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Tests treatment safety and results for Duchenne Muscular Dystrophy
Official title A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
ClinicalTrials.gov ID: NCT07673809
What this study is testing
What is GNR-097?
GNR-097 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 9, men only
You may be able to join if
- 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].
You likely can't join if
- 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...
- 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...
- 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.
See the full eligibility criteria
- 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.
- 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...
- 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...
- 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.
- 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 \ 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.
The study team makes the final eligibility decision.
Where it's taking place
- Minsk, Belarus
- Moscow, Russia
- Saint Petersburg, Russia
- Yekaterinburg, Russia
Compensation & support
A stipend or compensation may be offered.
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 4 years to 9 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Minsk, Belarus; Moscow, Russia; Saint Petersburg, Russia; Yekaterinburg, Russia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.