About 60% of people with hemophilia have a severe form of the condition. The severity level of hemophilia is a measure of clotting factor activity in the blood. People with severe hemophilia have less than 1% clotting factor activity. By comparison, normal factor activity is above 50% and as high as 150 to 200%.
Current standard care is replacement of clotting factor. For some people, the frequency and intravenous (IV) administration route of replacement therapy can make it a burden. For people who have developed inhibitors, the standard replacement therapy is not effective.
About 15–20% of people with hemophilia develop inhibitors that prevent standard hemophilia treatments from working effectively. The body thinks the replacement clotting factor is a foreign substance and tries to destroy it. People with inhibitors require special treatment, which can be complicated and costly.
Research is underway to find treatments that may be more effective for all hemophilia patients, with or without inhibitors. Treatment options that work differently than factor replacement therapy and work against inhibitors may help improve compliance and reduce bleeding episodes.
The BASIS study is working to find a hemophilia treatment that may be more convenient and more effective than current standard treatments. If you have severe hemophilia A or B, with or without inhibitors, you may be able to help with this research.
If you’re interested in the BASIS study, you can print this page and show it to your doctor or simply give this trial reference number to your doctor.
BASIS is testing a study drug to see if it is safe and what effects it may have on hemophilia in adolescents (12 to 17 years old) and adults (less than 75 years old) living with severe hemophilia A or B, with or without inhibitors. *
This study is different from your regular medical care. The purpose of regular medical care is to improve or otherwise manage your health, but the purpose of research is to gather information to advance science and medicine.
* Some people develop inhibitors that prevent standard hemophilia treatments from working.
The study team determines eligibility of each potential participant, but the following parameters should give you some idea of whether this trial may be an option for you.
At least 12 but less than 75 years old
Severe hemophilia A or B, with or without inhibitors
The study drug, called PF-06741086 or marstacimab, is:
Before joining the BASIS study, you will review all aspects of the trial with the study team and ask as many questions as you want. After having all your questions answered, if you want to participate in the study, you will sign the informed consent document and continue with the study screening, to see if this study is right for you. If you join the study, your participation will last about 20 months. You will visit the study site 12 times and have 9 phone calls with the study team. You may be reimbursed for your time and travel.
During study visits and phone calls, you will answer questions and review your study eDiary with the study team. At some visits, procedures such as measurement of your vital signs, blood and urine tests, and physical examinations will be required.
If you complete 12 months of study drug injections, you may have the opportunity to participate in a 12-month extension study and continue study drug injections.
You will have a final phone call with the study team about 1 month after your last dose of study drug.