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

NCT06207552 | Recruiting Early Phase 1

• 1 other identifier: BBM017-IIT1001
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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

Keywords

Fabry

Outcome Measures

Primary Outcomes (2)

• 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

• 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

Study Arms (1)

Arm of BBM-F101 injection

EXPERIMENTAL

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

Genetic: BBM-F101 injection

Interventions

BBM-F101 injection GENETIC

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

Arm of BBM-F101 injection

Eligibility Criteria

• Age: 7 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, China

RECRUITING

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Hong Xu, MD,PhD

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Qian Shen, MD,PhD

CONTACT

+86 13701923307; shenqian@shmu.edu.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2023
• First Posted: January 17, 2024
• Study Start: February 20, 2024
• Primary Completion: June 1, 2025
• Study Completion (Estimated): June 1, 2029
• Last Updated: July 9, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)