Tests a gene therapy injection in children with Fabry disease
Tests a gene therapy injection given once to children with Fabry disease.
Plain-language title generated from the registry record
Registry title: Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
Key facts
- Where
- Shanghai, ChinaSee the site
- Phase
- Early phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 7 and 18 years old?
Source: NCT06207552, who can take part
Where in China
- Children's Hospital Of Fudan UniversityShanghai, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Children's Hospital of Fudan University
- Trial code
- NCT06207552
- Record updated
- 9 July 2024
- Original description
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.Read full original descriptionShow less
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
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.
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: 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; 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; Other conditions that make the participant not eligible for the study according to the investigator.