Prospective Observational Study of Plasma-Derived FVIII/VWF in Immune Tolerance Induction (PRISM)

Clinical Trial

Offered by: Nemours Children's Specialty Care, Pensacola
Location: Pensacola, Fla.

Trial Name

Prospective Observational Study of Plasma-Derived FVIII/VWF in Immune Tolerance Induction (PRISM)

What is the trial about?

A study for patients with hemophilia A. Hemophilia A is an inherited bleeding disorder caused by not having enough blood clotting factor VIII. Without enough factor VIII, the blood cannot clot properly to stop bleeding. The purpose of the study is to learn about patients who develop an inhibitor.

Who can participate?

  • A patient with a diagnosis of congenital hemophilia A with Factor VIII <2 percent.
  • A patient who has a high responding inhibitor (titer greater than or equal to 5 BU/mL recorded at any time); or, if a low responding inhibitor (persistently <5 BU.mL), the patient is, in the investigator’s judgment, a candidate for ITI or has already initiated ITI with Alphanate. 
  • A patient who has initiated treatment with Alphanate as either primary or rescue ITI within the last six months OR a clinical decision has been made to start treatment on Alphanate for ITI within eight weeks.

What is involved?

Information about your medical history and hemophilia A treatments will be collected. Information will be collected after your first Alphanate treatment. Information will be gathered every three months while you are being treated with Alphanate. For patients who have a good response to Alphanate, information will be gathered every three months for one year after you stop Alphanate treatment. If you agree, information may also be collected once a year until the end of the study. The end of the study may be up to 10 years after you enroll.  In addition to the information that your doctor will gather, you will be asked to complete a questionnaire.

Contact Nemours Clinical Trials

Trial Name: Prospective Observational Study of Plasma-Derived FVIII/VWF in Immune Tolerance Induction

Contact Information

(800) 354-5690
ClinicalTrials@nemours.org