Status:
RECRUITING
Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
Lead Sponsor:
Children's Hospital of Fudan University
Conditions:
Fabry Disease
Eligibility:
All Genders
7-18 years
Phase:
EARLY_PHASE1
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 in...
Eligibility Criteria
Inclusion
- 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;
- Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
- Males or females aged ≥7 years and \<18 years old;
- Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
- Participants had at least one of the clinical manifestations for Fabry disease;
- Acceptable capsid antibody titers;
- Acceptable anti α-Gal A antibody titers;
- Acceptable laboratory values;
- Participant's legal guardian and participant with good cooperation and compliance;
- Use of reliable contraception methods during the study for adolescence.
Exclusion
- 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;
- Have potential liver diseases;
- Heart failure and severe arrhythmias;
- Severe allergic reactions for enzyme replacement drugs or other medications;
- Acute/chronic infections;
- End-stage renal disease;
- Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period;
- Have received gene therapy or used other investigational drugs within four weeks prior to dosing;
- Other conditions that make the participant not eligible for the study according to the investigator.
Key Trial Info
Start Date :
February 20 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 1 2029
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT06207552
Start Date
February 20 2024
End Date
June 1 2029
Last Update
July 9 2024
Active Locations (1)
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1
Children's Hospital of Fudan University
Shanghai, China