Contact SCGE




Clinical Trial Report

Gene Therapy Trial Report

Summary

Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B


NCTID NCT00979238 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Hemophilia B
Disease Ontology Term DOID:12259
Compound Name ScAAV2/8-LP1-hFIXco
Sponsor St. Jude Children's Research Hospital
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 14
Results Posted Not Available

Therapy Information


Target Gene/Variant F9
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2/8
Editor Type
Dose 1 2E11 vg/kg (n=2)
Dose 2 6E11 vg/kg (n=2)
Dose 3 2E12 vg/kg (n=6)
Dose 4 Extension phase with new process: 2E12 vg/kg (n=2)
Dose 5 Extension phase with new process: 5E12 vg/kg (n=2)

Study Record Dates


Current Stage Phase1
Submit Date 2009-09-16
Completion Date 2032-12-31
Last Update 2024-11-22

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 8
Locations United States,United Kingdom

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Was the first Hemophilia B trial to show sustained efficacy

Resources/Links