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Gene Therapy Trial Report

Summary

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


NCTID NCT06207552 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Fabry Disease
Disease Ontology Term DOID:14499
Compound Name BBM-F101
Sponsor Children's Hospital of Fudan University
Funder Type Other
Recruitment Status
Enrollment Count 6 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant GLA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 Undisclosed dose

Study Record Dates


Current Stage Early phase1
Submit Date 2023-12-18
Completion Date 2029-06
Last Update 2024-07-09

Participation Criteria


Eligible Age 7 Years - 18 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links