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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
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
Dose 2 4E11 vg/kg
Dose 3 2E12 vg/kg
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

Resources/Links