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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
Drug Product 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
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type AAV
Dose 1 Undisclosed dose

Study Record Dates


Current Phase 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