Summary

for males ages 18 years and up (full criteria)
at San Diego, California and other locations
study started
estimated completion
Annette von Drygalski

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 AMT-061

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, San Diego accepting new patients
    San Diego California 92161 United States
  • Los Angeles Orthopedic Hospital accepting new patients
    Los Angeles California 90007 United States

Lead Scientist

  • Annette von Drygalski
    Dr. von Drygalski is the Director of the Hemophilia and Thrombosis Treatment Center at UCSD (http://health.ucsd.edu/specialties/hemophilia/Pages/default.aspx) and her research interests involve basic, translational and clinical science in the field of hemostasis and thrombosis, specifically hemophilia. Dr. von Drygalski collaborates with Laurent O. Mosnier and John H.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
UniQure Biopharma B.V.
ID
NCT03569891
Phase
Phase 3
Study Type
Interventional
Last Updated