HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCTID	NCT03569891 (View at clinicaltrials.gov)
Description	This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg. (Show More)
Development Status	Approved
Indication	Hemophilia B
Disease Ontology Term	DOID:12259
Compound Name	HEMGENIX
Compound Alias	etranacogene dezaparvovec, AMT-061
Sponsor	CSL Behring
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	67
Results Posted	View Results

Target Gene/Variant	F9R338L
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	AAV5
Editor Type	none
Dose 1	2E13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	33
Locations	Netherlands,Sweden,Belgium,United States,Ireland,Denmark,United Kingdom,Germany

Has US IND	True
FDA Designations	Breakthrough Therapy, Orphan Drug Designation, Priority Review
Recent Updates	FDA approved 11/22/22; Price/treatment: $3.5M