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

Fabry Disease Clinical Trial

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06207552
• Other study ID #: BBM017-IIT1001
• Status: Not yet recruiting
• Phase: Early Phase 1
• Start date: February 2024
• Est. completion date: June 2029

Study information

• Verified date: February 2024
• Source: Children's Hospital of Fudan University
• Contact: Qian Shen, MD,PhD
• Phone: +86 13701923307
• Email: shenqian[@]shmu.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 6
• Est. completion date: June 2029
• Est. primary completion date: June 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years to 18 Years

Eligibility

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 a-Gal A (a-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 a-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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Genetic:
• BBM-F101 injection
The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion.

Locations

Country	Name	City	State
China	Children's Hospital of Fudan University	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Fudan University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of dose limited toxicity	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	12 weeks
Primary	Incidence of adverse events and serious adverse events	The incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following the BBM-F101 injection	52 weeks