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RECRUITINGEarly Phase 1INTERVENTIONAL

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

A Single-arm, Open Label, Single-dose Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-F101 Injection in the Treatment of Pediatric Fabry Disease

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

This is a single-arm, open label, single-dose clinical study to evaluate the safety, tolerability and efficacy of BBM-F101 injection in the pediatric Fabry disease participants up to 52 weeks after infusion, and the long-term safety and efficacy of BBM-F101 injection up to 5 years after infusion. BBM-F101 injection is an adeno-associated virus (AAV) gene therapy product for the treatment of pediatric Fabry disease.

Who May Be Eligible (Plain English)

Who May Qualify: 1. The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written willing to sign a consent form; If the participant is \>= 8 years old, the participant must also agree to participate in the study and sign a written willing to sign a consent form; 2. Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing; 3. Males or females aged ≥7 years and \<18 years old; 4. Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period; 5. Participants had at least one of the clinical manifestations for Fabry disease; 6. Acceptable capsid antibody titers; 7. Acceptable anti α-Gal A antibody titers; 8. Acceptable laboratory values; 9. Participant's legal guardian and participant with good cooperation and compliance; 10. Use of reliable contraception methods during the study for adolescence. Who Should NOT Join This Trial: 1. Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA), positive for hepatitis C virus RNA (HCV-RNA), positive for HIV or syphilis; 2. Have potential liver diseases; 3. Heart failure and severe arrhythmias; 4. Severe allergic reactions for enzyme replacement drugs or other medications; 5. Acute/chronic infections; 6. End-stage renal disease; 7. Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period; 8. Have received gene therapy or used other investigational drugs within four weeks prior to dosing; 9. Other conditions that make the participant not eligible for the study according to the investigator. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: 1. The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written informed consent; If the participant is \>= 8 years old, the participant must also agree to participate in the study and sign a written informed consent; 2. Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing; 3. Males or females aged ≥7 years and \<18 years old; 4. Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period; 5. Participants had at least one of the clinical manifestations for Fabry disease; 6. Acceptable capsid antibody titers; 7. Acceptable anti α-Gal A antibody titers; 8. Acceptable laboratory values; 9. Participant's legal guardian and participant with good cooperation and compliance; 10. Use of reliable contraception methods during the study for adolescence. Exclusion Criteria: 1. Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA), positive for hepatitis C virus RNA (HCV-RNA), positive for HIV or syphilis; 2. Have potential liver diseases; 3. Heart failure and severe arrhythmias; 4. Severe allergic reactions for enzyme replacement drugs or other medications; 5. Acute/chronic infections; 6. End-stage renal disease; 7. Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period; 8. Have received gene therapy or used other investigational drugs within four weeks prior to dosing; 9. Other conditions that make the participant not eligible for the study according to the investigator.

Treatments Being Tested

GENETIC

BBM-F101 injection

The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion.

Locations (1)

Children's Hospital of Fudan University
Shanghai, China