Hemophilia B

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

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

Phase 3


Doctors at 27 locations seek male patients with Hemophilia B currently under factor IX prophylaxis treatment, referred to as “FIX.”

Study participants will receive a single infusion of an investigatory gene therapy drug. This new treatment utilizes a harmless virus to deliver a gene designed to help the patient’s body produce FIX and reduce bleeding events. The virus works to transfer the gene into the patient’s DNA and cannot cause infection. For the first year, follow up will be minimal, tracking annualized bleeding rate and use of Factor IX, and continue for ~5 years.


  • Be male and 18 years or older
  • Have congenital severe or moderately severe hemophilia B
  • Not have received previous gene therapy treatment
  • Be HIV- negative


  1. Screening before the treatment
  2. A single infusion of AMT-061 will be administered
  3. Monitoring of the patient for a few hours after dosing, then minimal follow up for the next year


27 Locations in the US are recruiting participants for this study.

Contact: Alison Long, MD  |  [email protected]  |  339-970-7578

Contact: uniqure HOPE=B  |  [email protected]


UniQure Biopharma B.V.

Or go online: https://clinicaltrials.gov/ct2/show/NCT03569891