Cooperative Study of Factor VIII Inhibitors

This study has been completed.
Sponsor:
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier:
NCT00000582
First received: October 27, 1999
Last updated: April 26, 2012
Last verified: April 2012
  Purpose

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


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

Study Type: Interventional
Study Design: Masking: Double-Blind
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by National Heart, Lung, and Blood Institute (NHLBI):

Study Start Date: July 1978
Study Completion Date: July 1979
Detailed Description:

BACKGROUND:

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.

DESIGN NARRATIVE:

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.

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Males with hemophilia, not stratified as to ethnic group.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00000582

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