Actively Recruiting
Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
Led by Children's Hospital of Fudan University · Updated on 2024-07-09
6
Participants Needed
1
Research Sites
275 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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.
You will not qualify if you...
- 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.
AI-Screening
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Trial Site Locations
Total: 1 location
1
Children's Hospital of Fudan University
Shanghai, China
Actively Recruiting
Research Team
Q
Qian Shen, MD,PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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