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



This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase. The IMP 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 IMP is identified as AAV5-hFIXco-Padua (AMT- 061). The pharmaceutical form of AMT-061 is a solution for intravenous infusion. The administered dose of AMT-061 will be 2 x 1013 gc/kg.

Official Title

Phase IIb, Open-label, Single-dose, Single-arm, Multi-center Trial to Confirm the Factor IX Activity Level of the 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


Hemophilia B Hemophilia, Gene Therapy Bleeding Factor IX FIX viral vector Padua Hemophilia A AAV5-hFIXco-Padua (AMT-061) Single infusion of AMT-061


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
  4. >20 previous exposure days of treatment with FIX protein

You CAN'T join if...

  1. History of FIX inhibitors
  2. Positive FIX inhibitor test at screening
  3. Select screening laboratory values > 2 times upper normal limit:
  4. Positive human immunodeficiency virus (HIV) 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


  • University of California, San Diego
    San Diego California 92122 United States
  • Los Angeles Orthopedic Hospital
    Los Angeles California 90007 United States


in progress, not accepting new patients
Start Date
Completion Date
UniQure Biopharma B.V.
Phase 2
Study Type
Last Updated