This study is in progress, not accepting new patients

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

a study on Hemophilia

Summary

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

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

  1. Male
  2. Age ≥18 years
  3. Subjects with congenital hemophilia B, classified as severe or moderately severe, and are currently on factor IX prophylaxis
  4. >150 previous exposure days of treatment with factor IX protein

You CAN'T join if...

  1. History of factor IX inhibitors
  2. Positive factor IX inhibitor test at screening
  3. Select screening laboratory value >2 times upper limit of normal
  4. Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy
  5. Active infection with hepatitis B or C virus at screening
  6. History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase
  7. Previous gene therapy treatment
  8. Receipt of an experimental agent within 60 days prior to screening
  9. 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
Completion Date
(estimated)
Sponsor
CSL Behring
ID
NCT03569891
Phase
Phase 3 Hemophilia Research Study
Study Type
Interventional
Participants
About 67 people participating
Last Updated