Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A

Stopped early · Phase 3

Conditions studied: Severe Hemophilia A

In brief

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.

Key facts

Study ID

NCT01405742

Run by

University of Pittsburgh

People needed

4

Starts

2012-07-01

Expected to finish

2013-11-01

Last updated by the study team

2016-10-07

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Georgetown University — Washington D.C., District of Columbia, United States
• Hospital of the University of Pennsylvania — Philadelphia, Pennsylvania, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Hemophilia Center of Western Pennsylvania — Pittsburgh, Pennsylvania, United States
• Vanderbilt University — Nashville, Tennessee, United States
• Puget Sound Blood Center — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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