Cooperative Study of Factor VIII Inhibitors
Information source: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Blood Coagulation Disorders; Hematologic Diseases; Hemophilia A; Hemorrhagic Disorders
Intervention: factor ix (Drug)
Phase: Phase 3
Status: Completed
Sponsored by: National Heart, Lung, and Blood Institute (NHLBI) Official(s) and/or principal investigator(s): Louis Aledort, Affiliation: Icahn School of Medicine at Mount Sinai J. Edson, Affiliation: University of Minnesota - Clinical and Translational Science Institute M. Eyster, Affiliation: Milton S. Hershey Medical Center Scott Goodnight, Affiliation: University of Oregon Health Sciences Center William Hathaway, Affiliation: University of Colorado Medical Center Jack Lazerson, Affiliation: Milwaukee Children's Hospital Peter Levine, Affiliation: Memorial Hospital Jeanne Lusher, Affiliation: Wayne State University Campbell McMillan, Affiliation: University of North Carolina Sandor Shapiro, Affiliation: Thomas Jefferson University
Summary
To test the efficacy of prothrombin complex concentrates (Factor IX) in the treatment of
hemophiliac patients who had inhibitors to Factor VIII.
Clinical Details
Study design: Masking: Double-Blind, Primary Purpose: Treatment
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
Minimum age: 18 Years.
Maximum age: 75 Years.
Gender(s): Male.
Criteria:
Males with hemophilia, not stratified as to ethnic group.
Locations and Contacts
Additional Information
Related publications: Eyster ME, Gill FM, Blatt PM, Hilgartner MW, Ballard JO, Kinney TR. Central nervous system bleeding in hemophiliacs. Blood. 1978 Jun;51(6):1179-88. Lusher JM, McMillan CW. Severe factor VIII and factor IX deficiency in females. Am J Med. 1978 Oct;65(4):637-48. Kasper CK. Effect of prothrombin complex concentrates on factor VIII inhibitor levels. Blood. 1979 Dec;54(6):1358-68. Lusher JM, Shapiro SS, Palascak JE, Rao AV, Levine PH, Blatt PM. Efficacy of prothrombin-complex concentrates in hemophiliacs with antibodies to factor VIII: a multicenter therapeutic trial. N Engl J Med. 1980 Aug 21;303(8):421-5. Gerety RJ, Eyster ME, Tabor E, Drucker JA, Lusch CJ, Prager D, Rice SA, Bowman HS. Hepatitis B virus, hepatitis A virus and persistently elevated aminotransferases in hemophiliacs. J Med Virol. 1980;6(2):111-8.
Starting date: July 1978
Last updated: April 26, 2012
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