Fabry Disease
Conditions
Keywords
Fabry
Brief summary
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.
Interventions
GENETICBBM-F101 injection
The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion.
Sponsors
Children's Hospital of Fudan University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of dose limited toxicity | 12 weeks | The incidence of dose limited toxicity (DLT) events as determined by the safety review committee (SRC) within DLT observation period following the BBM-F101 injection |
| Incidence of adverse events and serious adverse events | 52 weeks | The incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following the BBM-F101 injection |
Countries
China
Contacts
Primary ContactQian Shen, MD,PhD
Outcome results
None listed