Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Fudan University

Status and phase

Enrolling

Early Phase 1

Conditions

Fabry Disease

Treatments

Genetic: BBM-F101 injection

Study type

Interventional

Funder types

Other

Identifiers

NCT06207552

BBM017-IIT1001

Details and patient eligibility

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.

Enrollment

6 estimated patients

Sex

All

Ages

7 to 18 years old

Volunteers

No Healthy Volunteers

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.