Brief Title
Cooperative Study of Factor VIII Inhibitors
Brief Summary
To test the efficacy of prothrombin complex concentrates (Factor IX) in the treatment of
hemophiliac patients who had inhibitors to Factor VIII.
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.
Study Phase
Phase 3
Study Type
Interventional
Condition
Blood Coagulation Disorders
Intervention
factor ix
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Start Date
July 1978
Completion Date
July 1979
Eligibility Criteria
Males with hemophilia, not stratified as to ethnic group.
Gender
Male
Ages
18 Years - 75 Years
Accepts Healthy Volunteers
No
Contacts
Louis Aledort, ,
Administrative Informations
NCT ID
NCT00000582
Organization ID
302
Study Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study Sponsor
Louis Aledort, , Icahn School of Medicine at Mount Sinai
Verification Date
April 2012