Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

NCTID	NCT02618915 (View at clinicaltrials.gov)
Description	A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B. (Show More)
Development Status	Inactive
Indication	Hemophilia B
Disease Ontology Term	DOID:12259
Compound Name	DTX101
Sponsor	Ultragenyx Pharmaceutical Inc
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	6
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	AAVrh10
Editor Type
Dose 1	1.6E12 GC/kg
Dose 2	5.0E12 GC/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	9
Locations	United States,United Kingdom,Bulgaria

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	DTX101 did not demonstrate sufficient efficacy, program was terminated in 2017