Summary

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

Description

Summary

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

Keywords

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

Locations

  • University of California, San Diego accepting new patients
    San Diego California 92122 United States
  • University of California, Davis not yet accepting patients
    Sacramento California 95817 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
UniQure Biopharma B.V.
ID
NCT03489291
Phase
Phase 2
Study Type
Interventional
Last Updated
July 11, 2018