A Study of the Long-Term Safety and Efficiency of Experimental Medicine SPK-9001 For Hemophilia B

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Age: 18 years or older
Healthy Volunteers: No
Keywords: hemophilia B
Type: Genetic study
3 Participants
The purpose of this research study is to find out the long term effects after having participated in the gene transfer study (SPK-9001-101). In the gene transfer study you received a single dose of SPK-9001.

Hemophilia B is an inherited (genetic) disease in which a protein (factor IX) that promotes blood clotting is missing or does not work properly. Hemophilia B is caused by a defect in the gene that produces the factor IX protein. Factor IX protein is important in blood clotting. Patients with severe hemophilia B have very low levels of the normal factor IX protein. Such low levels can result in spontaneous bleeding events that cause destruction of bone and tissue. Bleeding events can include hemorrhage in the brain and can cause severe symptoms (like stroke) and death.

The gene transfer you were given in the SPK-9001-101 study may increase levels of factor IX protein in the blood. It may keep them elevated to a stop or reduce spontaneous bleeding episodes.
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. Ability to understand the purpose and risks of the study, as well as provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local study participant privacy regulations.
  2. Subjects who have received a single administration of SPK-9001, and completed the required assessments in the SPK-9001-101 clinical study.

Exclusion criteria:

  1. Subjects who will not consent for up to 5 years of safety follow-up.
  2. Subjects who are unable or unwilling to comply with the study visits and requirements.
  3. Subjects who have planned to participate in another interventional investigational gene transfer study.
  4. Subjects with any clinically significant medical condition that the Investigator believes would pose as a safety risk or are not capable of the performing endpoints of this study.
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 not be paid to take part in this research study.
Study duration and period
We expect that you will be in this research study for up to 5 years if you agree to take part in this extension study.
Recruitment period
From Feb. 16, 2018
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
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.


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