Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

NCTID	NCT05164471 (View at clinicaltrials.gov)
Description	Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study. (Show More)
Development Status	Inactive
Indication	Hemophilia B
Disease Ontology Term	DOID:12259
Compound Name	FLT180a
Compound Alias	Verbrinacogene setparvovec
Compound Description	AAV2/S3.FRE1.Ti.FIXco1
Sponsor	Spur Therapeutics
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	6
Results Posted	Not Available

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	Liver
Delivery System	Viral transduction
Vector Type	AAVS3
Editor Type
Dose 1	3.84E11 vg/kg
Dose 2	6.40E11 vg/kg
Dose 3	8.32E11 vg/kg
Dose 4	1.28E12 vg/kg
Dose 5

No.of Trial Sites	7
Locations	United States,United Kingdom

Has US IND	True
FDA Designations
Recent Updates	Study was terminated early in Phase 1/2 trial, Freeline was acquired by Syncona