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

Children's Hospital of Fudan University1 site in 1 country6 target enrollmentFebruary 20, 2024

ConditionsFabry Disease

Overview

Phase: Early Phase 1
Intervention: Not specified
Conditions: Fabry Disease
Sponsor: Children's Hospital of Fudan University
Enrollment: 6
Locations: 1
Primary Endpoint: Incidence of dose limited toxicity
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

February 20, 2024

End Date

June 2029

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Children's Hospital of Fudan University

Responsible Party

Sponsor

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.

Outcomes

Primary Outcomes

Incidence of dose limited toxicity

Time Frame: 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

Time Frame: 52 weeks

The incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following the BBM-F101 injection