A Gene Transfer Study Using Experimental SPK-8011 for Hemophilia A (poor blood clotting)

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"This research study will help us learn if the study product (SPK-8011) is safe and effective in reducing bleeding for treating hemophilia A."
Age: 18 years or older
Gender:
Male
Healthy Volunteers: No
Keywords: hemophilia A, Gene-transfer
Type: Genetic study, Phase [1, 2]
Target:
2 Participants
Investigator:
Description
A gene is a piece of DNA, which is found in cells in the human body, that makes proteins. Gene transfer is an approach to transfer normal genes into patients. Scientists have been looking for ways to transfer the factor VIII gene into the cells of hemophilia A patients. After transferring the gene, patients may make factor VIII protein in the blood, which may stop or lower the risk of bleedings. This type of approach is called factor VIII gene transfer. The vehicles bringing a gene inside the cells are called ‘vectors’. Many vectors are made from viruses. In nature, viruses must get inside the cells in order to reproduce.

There is a non-disease causing virus called AAV (adeno-associated virus). AAV is a virus that infects people, but has not been known to cause illness. Using special methods of cutting and pasting the DNA, scientists have changed this harmless virus so that it can transfer genes into cells. The genes of the AAV virus are removed then replaced with a factor VIII gene. Once the AAV virus is changed, it is called vector. Think of the ‘vector’ as a car and the ‘factor VIII gene’ as the passenger. The AAV vector carrying the factor VIII gene injects into your blood. Once in the blood, the vector travels to your liver where the factor VIII protein is normally made.

The purpose of this research study is to learn if a study product (SPK-8011) is safe and effective (how well it prevents bleeding) for treating hemophilia A.
This study requires

This study requires several medical visits, tests, and procedures. This includes but is not limited to:

  • Lab tests to establish a baseline
  • Safety monitoring and observation
  • Infusions

Your physician will discuss in detail with you the visits, tests, and procedures you will need to undergo in this study.

Who can participate

Inclusion Criteria:

  • Males age18 years or older

  • Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma FVIII activity levels ≤ 2% of normal

  • Have received >150 exposure days (EDs) to FVIII concentrates or cryoprecipitate

  • Have experienced >10 bleeding events over the previous 12 months only if receiving on-demand therapy and having FVIII baseline level 1-2% of normal

  • Have no prior history of allergic reaction to any FVIII product

  • Have no measurable inhibitor against factor VIII inhibitor as assessed by the central laboratory and have no prior history of inhibitors to FVIII protein

  • Agree to use reliable barrier contraception

Exclusion Criteria:

  • Evidence of active hepatitis B or C

  • Currently on antiviral therapy for hepatitis B or C

  • Have significant underlying liver disease

  • Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 ( participants who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll)

  • Have detectable antibodies reactive with AAV-Spark200 capsid

  • Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks

Benefits and risks of participating
BENEFITS:

We can promise no benefits to you from participating in this research.


RISKS:

Your physician will review all the risks of this study with you.
Compensation
We will compensate you $1500 for the infusion day and $150 for all other study visits for your time and effort. You will be reimbursed up to $100 per study visit for travel costs. Your companion’s travel costs on the Dosing Day will also be compensated, if needed.
Resources
Schedule
Study duration and period
You will take part in this study for about 64 weeks. An optional additional 15 years of follow up is recommended.
Recruitment period
From Sept. 7, 2017
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Research Topic
Conditions:
  • Hemophilia A

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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