Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A

• Status: Terminated
• Trial end date: 2013-11-01
• Target enrollment: 0
• Participant gender: Male

Summary

The purpose of this pilot R34 trial is to determine the feasibility of a large single dose Phase III study of hemophilia adult prophylaxis comparing once weekly with thrice-weekly recombinant factor VIII. Efficacy will measured by bleeding frequency, factor usage, joint range of motion, cost, quality-of-life, F.VIII level, and inter-dose hypocoagulability by thrombin generation. Safety will be measured by inhibitor formation and bleeding events unresponsive to up to two rescue doses.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Pittsburgh
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI)
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

• Adult males 18 years or older

• Severe hemophilia A (F.VIII < 0.01 U/ml)

• At least 150 exposure days to F.VIII products

• No detectable inhibitor

• No history of allergic reaction

• Platelets at least 150,000/ul

• If HIV(+), CD4 at least 200/ul, HIV-VL <48 copies/ml,and cART compliant

• If HCV(+), no splenomegaly,varices,GI bleed,ascites,edema,encephalopathy

• Willingness to comply with cross-over design, randomization schema

• Willingness to keep a personal diary of bleeding frequency and factor use

• Willingness to make every 3 month visits, coagulation testing at wks 2, 28

Exclusion Criteria:

• Acquired hemophilia

• Any bleeding disorder other than hemophilia A

• Presence of an inhibitor to factor VIII

• Historic platelet count < 100,000

• Use of experimental drugs

• Surgery anticipate in the next 52 weeks

• Symptomatic HCV(splenomegaly,varices,GI bleed,ascites,edema,encephalopathy)

• Symptomatic HIV(CD4<200/ul or HIV VL 48 or more copy/ml,cART noncompliant)

• Life expectancy less than 5 years

• Investigational drug or study within 4 weeks prior to study

• Inability to comply with study requirements