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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B


NCTID NCT00076557 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Hemophilia B
Disease Ontology Term DOID:12259
Compound Name AAV2-hFIX16
Compound Description adeno-associated virus serotype 2 capsid carrying optimized human F9 cDNA, under the control of the APOE enhancer and SERPINA1 promotor
Sponsor Avigen
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 15
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 Hepatic artery infusion
Drug Product Type Viral vector
Target Tissue/Cell Hepatocyte
Delivery System Viral transduction
Vector Type AAV2
Editor Type
Dose 1 8E10 vg/kg (n=2)
Dose 2 4E11 vg/kg (n=3)
Dose 3 2E12 vg/kg (n=2)
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2004-01-26
Completion Date
Last Update 2007-04-04

Participation Criteria


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

Locations


No.of Trial Sites 3
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Avigen suspended enrollment in May 2004; transferred AAV-based product rights to Genzyme Corporation in December 2005, LTFU was sponsored by CHOP from 2009-2013, then Spark Therapeutics afterwards

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