FIXtendz is a clinical research study that will test an investigational in vivo genome editing therapy called SB-FIX for severe hemophilia B.
Listed below are some questions and answers about the study. If you have general questions about clinical research, please visit the Clinical research FAQ tab.
See if you qualify, to be referred to a doctor participating in the FIXtendz study.
You may qualify for the FIXtendz study if you:
- Are a male at least 18 years old
- Have severe hemophilia B and are currently free of inhibitor
- Have no history of an allergic reaction to Factor IX (FIX) products
If you fit these criteria, we encourage you to take the pre-qualification questionnaire. If you meet the basic study requirements, a study representative will be in touch with you to confirm your eligibility.
The FIXtendz clinical research study is looking for adult men with severe hemophilia B to test an investigational genome editing therapy named SB-FIX. (“Investigational” means it isn’t approved yet for use by the public.)
SB-FIX consists of a substance (called a vector or carrier) that carries the therapeutic Factor 9 gene for making the clotting protein called Factor IX (FIX). Study participants will receive a single injection of SB-FIX into a vein. The working gene for FIX will then be carried through the bloodstream to the liver.
Doctors are studying SB-FIX to see if it’s safe and tolerable, and learn more about its effectiveness at enabling your body to produce FIX on its own and improve blood clotting.
Click About in vivo Genome Editing to learn more about SB-FIX and in vivo Genome Editing.
Yes. Study participants will be randomly assigned into 1 of 3 treatment groups. Each treatment groups will receive a different dose (low, medium, or high) of SB-FIX. This study is open-label, which means that you and your doctor will know which dose of SB-FIX you received.
All study medication and study-related care will be at no cost to you. You’ll also receive reimbursement for travel. Transportation assistance may be available.
After you receive the infusion of SB-FIX, your study staff will follow your health for up to 3 years. You’ll make regular study appointments with the study staff, and the staff will work with you to find convenient times for these visits. At these visits, the study staff will check your health, including taking blood, urine, saliva, stool, and semen samples to study the effects of the investigational therapy.
If for some reason you need to leave the study, you may do so at any time, for any reason.
See if you may qualify for the FIXtendz study by taking this short questionnaire.