The HOPE-B Trial of Experimental AMT-061 For Severe or Moderately Severe Hemophilia B
Logo Image
"Volunteer for research at UC Davis and contribute to discoveries that may improve health care for you, your family, and your community!"
Age: 18 years or older
Healthy Volunteers: No
Keywords: hemophilia B, blood clotting disorder
Type: Genetic study, Phase 3
2 Participants
The purpose of this study is to test the safety and effectiveness of a single infusion of the experimental study drug AMT-061. It will be given to adults with severe or moderately severe hemophilia B.

AMT-061 is a gene therapy product. Genes contain the blueprint for the body to produce proteins. A damaged or mutated gene can cause the body to produce insufficient levels of a certain protein or stop producing a certain protein at all. In people suffering from hemophilia B, the gene containing the blue print for clotting factor IX, is damaged or mutated.

AMT-061 gene therapy targets the liver cells, where factor IX is normally produced. Once AMT061 reaches the liver cells, the capsid (“Package”) releases the gene cassette (“instruction manual”). The capsid is then broken down and disappears. The gene cassette is expected to remain in the liver cells permanently (independent from your own genetic material). The intended result is stable factor IX production in the liver for a long period of time.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

Inclusion criteria:

  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

Exclusion criteria:

  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
Benefits and risks of participating

We cannot promise any benefits to you as a result of taking part in this research study.


The study doctor will discuss the risks associated with taking part in this research study.
You will be compensated $100 for the screening visit, $1,000 for the dosing visit, and $100 for each clinic visit for your time and effort. All payments will be processed through Paragon International.
Study duration and period
We expect that you will be in this research study for at least 5 and a half years.
Recruitment period
From May 15, 2019
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Amanda Surrell
Research Topic
  • Hemophilia B

Have any questions or want to learn more? Leave your contact details below and the research team will reach out to you.


Send SMS with Study Info:

SMS Sent!

Share Study Info via Email:

E-mail Preview