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

NCT ID: NCT06207552

Last Updated: 2024-07-09

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-02-20
• Study Completion Date: 2029-06-30

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.

Conditions

Fabry Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm of BBM-F101 injection

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

Group Type: EXPERIMENTAL

BBM-F101 injection

Intervention Type: GENETIC

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

Interventions

BBM-F101 injection

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

Intervention Type: GENETIC

Eligibility Criteria

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.

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital of Fudan University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Hong Xu, MD,PhD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Fudan University

Locations

Children's Hospital of Fudan University

Shanghai, , China

Site Status: RECRUITING

Countries

China

Central Contacts

Qian Shen, MD,PhD

Role: CONTACT

shenqian@shmu.edu.cn

+86 13701923307

Facility Contacts

Qian Shen, MD,PhD

Role: primary

shenqian@shmu.edu.cn

+86 13701923307

Other Identifiers

BBM017-IIT1001

Identifier Type: -

Identifier Source: org_study_id