This study is in progress, not accepting new patients

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

Eligibility: for males ages 18 years and up (full criteria)
Location: at UC Davis UCSD
Dates: study started June 2018
completion around March 2025

Description

Summary

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 10¹³ gc/kg.

Official Title

Phase III, Open-label, Single-dose, Multi-center, Multinational Trial Investigating a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, AMT-061) Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B

Keywords

Hemophilia B, Gene therapy, FIX, factor IX, Bleeding, Viral vector, Padua, hemophilia, AAV (adeno-associated virus), serotype 5 AAV (adeno-associated virus), serotype 5, Hemophilia A, AAV5-hFIXco-Padua, Factor IX (FIX)

Eligibility

You can join if…

Open to males ages 18 years and up

Male
Age ≥18 years
Subjects with congenital hemophilia B, classified as severe or moderately severe, and are currently on factor IX prophylaxis
>150 previous exposure days of treatment with factor IX protein

You CAN'T join if...

History of factor IX inhibitors
Positive factor IX inhibitor test at screening
Select screening laboratory value >2 times upper limit of normal
Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy
Active infection with hepatitis B or C virus at screening
History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase
Previous gene therapy treatment
Receipt of an experimental agent within 60 days prior to screening
Current participation or anticipated participation within one year after study drug administration in this trial in any other interventional clinical trial involving drugs or devices

Locations

University of California, Davis
Sacramento California 95817 United States
University of California, San Diego
San Diego California 92161 United States
Los Angeles Orthopedic Hospital
Los Angeles California 90007 United States
Children's Hospital of Los Angeles
Los Angeles California 90027 United States

Details

Status: in progress, not accepting new patients
Start Date: June 2018
Completion Date: March 2025 (estimated)
Sponsor: CSL Behring
ID: NCT03569891
Phase: Phase 3 Hemophilia Research Study
Study Type: Interventional
Participants: About 67 people participating
Last Updated: March 20, 2025