Cooperative Study of Factor VIII Inhibitors

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00000582
Recruitment Status : Completed
First Posted : October 28, 1999
Last Update Posted : April 27, 2012
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)

Brief Summary:
To test the efficacy of prothrombin complex concentrates (Factor IX) in the treatment of hemophiliac patients who had inhibitors to Factor VIII.

Condition or disease Intervention/treatment Phase
Blood Coagulation Disorders Hematologic Diseases Hemophilia A Hemorrhagic Disorders Drug: factor ix Phase 3

Detailed Description:


Despite major advances in the treatment of patients with hemophilia, a serious remaining challenge was presented by the occurrence of circulating inhibitors to Factor VIII. Because of lack of information on the natural course of patients with Factor VIII inhibitors, the relative efficacy of various modes of therapy was not established. The Division of Blood Diseases and Resources decided to sponsor a clinical investigation which would evaluate populations of hemophilia patients for Factor VIII inhibitors, follow up these patients to provide information on the natural history of the inhibitor in the hemophilia patients, and make available a reference center to monitor results and attain uniformity.

Treatment of a patient with a severe inhibitor and consequent bleeding remained a problem. Management included protracted treatment with Factor VIII, use of immunosuppressive agents and prothrombin complex (or Factor IX) concentrates. The rationale for Factor IX was that it bypassed the defect in Factor VIII caused by the inhibitor. This method of therapy attracted wide popularity, but the success was greatly debated. It was intended at the very outset of the Factor VIII study that therapeutic trials involving patients with inhibitors would not be a prime function, but that such studies would be monitored if necessary. A controlled trial of Factor IX concentrates therapy was strongly advised by the DBDR Advisory Committee. Accordingly, during fiscal year 1978, a protocol for a double-blind control study was developed by the Factor VIII inhibitor group. The trial began in the spring of 1978, and the intervention terminated about one year later.


Double-blind study; patients served as their own controls. A total of 51 patients each received a single large dose of Konyne, Proplex, or diluted albumin (as a control). Joint bleeding of the elbow, knee, and ankle was evaluated six hours after each dose.

Study Type : Interventional  (Clinical Trial)
Masking: Double
Primary Purpose: Treatment
Study Start Date : July 1978
Actual Study Completion Date : July 1979

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Males with hemophilia, not stratified as to ethnic group.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00000582

Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
OverallOfficial: Louis Aledort Icahn School of Medicine at Mount Sinai
OverallOfficial: J. Edson University of Minnesota - Clinical and Translational Science Institute
OverallOfficial: M. Eyster Milton S. Hershey Medical Center
OverallOfficial: Scott Goodnight University of Oregon Health Sciences Center
OverallOfficial: William Hathaway University of Colorado Medical Center
OverallOfficial: Jack Lazerson Milwaukee Children's Hospital
OverallOfficial: Peter Levine Memorial hospital
OverallOfficial: Jeanne Lusher Wayne State University
OverallOfficial: Campbell McMillan University of North Carolina
OverallOfficial: Sandor Shapiro Thomas Jefferson University