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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
The purpose of this study is to determine the safety of giving a normal factor IX gene to treat individuals who have an abnormal or no factor IX gene. Recruitment will be limited to adults (≥ 18 years) with a confirmed diagnosis of hemophilia B (HB), resulting from a missense mutation in the coagulation factor IX (FIX) gene or a nonsense mutation that has not been associated with an inhibitor. Only subjects who have no evidence of active hepatitis or anti-hFIX antibodies, and who have been treated/exposed to Factor IX concentrates for at least ten years and have had an average of 3 bleeding episodes per year requiring FIX administration will be enrolled. Patients will be recruited within the United States for treatment at St. Jude Children's Research Hospital, and patients will be recruited in England and other countries for treatment in London by our British collaborators.
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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
Clinical Publications
(Abstract) Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer - ASH 2018
(Abstract) Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy - ASH 2023
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
Protocol
Clinical Trial Protocol