A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

NCTID	NCT04394286 (View at clinicaltrials.gov)
Description	The purpose of this study is to evaluate the safety and dose escalation of SHP648 an adeno-associated viral vector for gene transfer in hemophilia B participants. (Show More)
Development Status	Inactive
Indication	Hemophilia B
Disease Ontology Term	DOID:12259
Compound Name	SHP648
Compound Alias	BAX 335, AskBio009
Compound Description	AAV8.ss-3xCRM8-TTR-FIX_R338Lopt
Sponsor	Baxalta now part of Shire
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	2
Results Posted	View Results

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	AAV8
Editor Type
Dose 1	2E11 vg/kg
Dose 2	1.0E12 vg/kg
Dose 3	3E12 vg/kg
Dose 4
Dose 5

No.of Trial Sites	2
Locations	Turkey,Spain

Has US IND	True
FDA Designations
Recent Updates	Takeda decided to terminate the SHP648 (TAK-748) program prior to dosing additional subjects. In the previous clinical trial, there was little evidence of sustained efficacy