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Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00284193
First Posted: January 31, 2006
Last Update Posted: July 27, 2012
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.
Information provided by (Responsible Party):
Prof. Uriel Martinowitz, Sheba Medical Center
  Purpose

Patients with severe hemophilia and inhibitors can be treated effectively by Activated Prothrombin Complex Concentrates (APCC, eg. FEIBA) or High dose recombinant factor VIIa (rFVIIa). Rarely, such patients develop refractoriness to these products for whom therapy with sequential FEIBA and rFVIIa has been recently suggested.

The impetus for the present report was a hemophilia A patient with high titer inhibitor (1300BU) who had life threatening hematuria that was resistant to repeated doses of 400µg/kg rFVIIa up to a cumulative dose of 1200 µg/kg given over 6-9 hours.

Thrombin generation (TG) tested in vitro was consistent with resistance to high concentrations of rFVIIa but yielded good response to combinations of low doses of rFVIIa+FEIBA. In a desperate attempt to control the bleeding, concomitant therapy of 25 U/kg FEIBA and 40µg/kg rFVIIa was infused and resulted in arrest of bleeding within minutes. Over a span of about one year the patient has been successfully treated by this combination for more than 200 bleeding episodes in muscles and joints.


Condition Intervention Phase
Hemophilia A Drug: rFVIIa-FEIBA therapy for hemophilia A inhibitors Drug: FEIBA- Activated Prothrombin Complexes Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII

Resource links provided by NLM:


Further study details as provided by Prof. Uriel Martinowitz, Sheba Medical Center:

Primary Outcome Measures:
  • Hemostasis achieved post therapy [ Time Frame: 6-24 hours ]
    Following acute bleeding therapy hemostasis was defined as good, partial or non-satisfactory

  • Safety [ Time Frame: 0-24 HOURS ]
    Following therapy presence of any adverse events, especially thromboembolic complications was assessed


Secondary Outcome Measures:
  • Time to Hemostasis [ Time Frame: 0-24 HOURS ]
    Following therapy patients documented time to "GOOD" response


Other Outcome Measures:
  • Coagulation Studies [ Time Frame: 0-24 HOURS ]
    cbc fibrinogen and D-dimer were assessed pre and post therapy, thrombin generation was assayed when possible after 1-2 hours


Enrollment: 5
Study Start Date: January 2005
Study Completion Date: November 2009
Primary Completion Date: November 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: feiba-VIIa, hemophilia A-inhibitor therapy
COMBINED PATIENT- TAILORED THERAPY WITH CONCOMITANT ADMINISTRATION OF BOTH DRUGS , FOLLOWING EX VIVO THROMBIN GENERATION PREDICTING ASSAYS
Drug: rFVIIa-FEIBA therapy for hemophilia A inhibitors
DOses tailored per ex vivo spiking thrombin generation
Other Names:
  • NOVOSEVEN
  • APCC
Drug: FEIBA- Activated Prothrombin Complexes

Detailed Description:

Inhibitor patients with HR inhibitors were eligible for study enrollment. After consent blood was drawn and ex- vivo spiked with rFVIIa/FEIBA and combinations, assayed by thrombin generation tests.

The combination yielding sufficient hemostasis was depicted for patients' therapy of future bleeding episodes.

Following actual therapy hemostasis and safety parameters were monitored.

  Eligibility

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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Hemophilia patients with inhibitors
  • Patients signing informed consent

Exclusion Criteria:

  • Patients under 16 or above 65
  • Patients with allergic reaction or adverse events in previous use the concentrates
  • Patients with high risk of thrombosis
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT00284193


Sponsors and Collaborators
Sheba Medical Center
Investigators
Principal Investigator: Uri Martinowitz, MD Sheba Medical Center
  More Information

Responsible Party: Prof. Uriel Martinowitz, Prof. Uri Martinowitz, Sheba Medical Center
ClinicalTrials.gov Identifier: NCT00284193     History of Changes
Other Study ID Numbers: SHEBA-05-3768-UM-CTIL
First Submitted: January 17, 2006
First Posted: January 31, 2006
Last Update Posted: July 27, 2012
Last Verified: July 2012

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Feiba
Factor VIII
Thrombin
Coagulants
Hemostatics