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Clinical Trial Report

Gene Therapy Trial Report

Summary

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A


NCTID NCT05265767 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemophilia A
Disease Ontology Term DOID:12134
Compound Name CD68-ET3-LV
Compound Description Auto CD34+PBSC transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene
Sponsor Christian Medical College, Vellore, India
Funder Type Other
Recruitment Status
Completed
Enrollment Count 6
Results Posted Not Available

Therapy Information


Target Gene/Variant F8
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type
Dose 1 5.0-6.1E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2022-02-16
Completion Date 2024-06-28
Last Update 2024-07-12

Participation Criteria


Eligible Age 18 Years - 45 Years
Standard Ages Adult
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 1
Locations India

Regulatory Information


Has US IND True
FDA Designations
Recent Updates

Resources/Links