Status:

TERMINATED

Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A

Lead Sponsor:

University of Pittsburgh

Collaborating Sponsors:

National Heart, Lung, and Blood Institute (NHLBI)

Conditions:

Severe Hemophilia A

Eligibility:

MALE

18+ years

Phase:

PHASE3

Brief 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 V...

Detailed Description

The purpose of this 52-week pilot R34 randomized, open-label, non-inferiority, cross-over study is to determine the feasibility of a large single dose Phase III study of hemophilia adult prophylaxis. ...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

July 1 2012

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

November 1 2013

Estimated Enrollment :

4 Patients enrolled

Trial Details

Trial ID

NCT01405742

Start Date

July 1 2012

End Date

November 1 2013

Last Update

October 7 2016

Active Locations (6)

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Page 1 of 2 (6 locations)

1

Georgetown University

Washington D.C., District of Columbia, United States, 20057-1168

2

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104-4206

3

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104-4318

4

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States, 15213-4306