Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: BBM-F101 injection.
- Who it may be relevant to
- Registry conditions: Fabry Disease. Basic parameters: 7 years — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
Overview
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
- Genetic BBM-F101 injection
The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion.
Primary outcome measures
- Incidence of dose limited toxicity [Time frame: 12 weeks]
- Incidence of adverse events and serious adverse events [Time frame: 52 weeks]
Eligibility criteria
Inclusion criteria
- 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 criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Children's Hospital of Fudan University — Shanghai
Identifiers
NCT: NCT06207552 · BBM017-IIT1001