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

Early Phase 1

Recruiting

• Conditions: Fabry Disease
• Interventions: Genetic: BBM-F101 injection

• Registration Number: NCT06207552
• Lead Sponsor: Children's Hospital of Fudan University

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-12-18
• Study First Submitted QC: 2024-01-04
• Study First Posted: 2024-01-17
• Study Start: 2024-02-20
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-07
• Last Update Posted: 2024-07-09
• Primary Completion: 2025-06
• Study Completion: 2029-06

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

1. 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;
2. Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
3. Males or females aged ≥7 years and <18 years old;
4. Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
5. Participants had at least one of the clinical manifestations for Fabry disease;
6. Acceptable capsid antibody titers;
7. Acceptable anti α-Gal A antibody titers;
8. Acceptable laboratory values;
9. Participant's legal guardian and participant with good cooperation and compliance;
10. Use of reliable contraception methods during the study for adolescence.

Exclusion Criteria

1. 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;
2. Have potential liver diseases;
3. Heart failure and severe arrhythmias;
4. Severe allergic reactions for enzyme replacement drugs or other medications;
5. Acute/chronic infections;
6. End-stage renal disease;
7. Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period;
8. Have received gene therapy or used other investigational drugs within four weeks prior to dosing;
9. Other conditions that make the participant not eligible for the study according to the investigator.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Arm of BBM-F101 injection	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

Name	Time	Method
Incidence of dose limited toxicity	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	52 weeks	The incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following the BBM-F101 injection

Trial Locations

Locations (1)

Children's Hospital of Fudan University; 🇨🇳 Shanghai, China