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Completed

NCT Number: NCT00284193

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

16 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 4

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

rFVIIa-FEIBA therapy for hemophilia A inhibitors

Drug

DOses tailored per ex vivo spiking thrombin generation

Other names: NOVOSEVEN, APCC

FEIBA- Activated Prothrombin Complexes

Drug

Primary outcomes

  1. Hemostasis achieved post therapy

    Time frame: 6-24 hours

    Following acute bleeding therapy hemostasis was defined as good, partial or non-satisfactory

  2. Safety

    Time frame: 0-24 HOURS

    Following therapy presence of any adverse events, especially thromboembolic complications was assessed

Secondary outcomes

  1. Time to Hemostasis

    Time frame: 0-24 HOURS

    Following therapy patients documented time to "GOOD" response

Other outcomes

  1. 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

Sponsors and collaborators

Lead sponsor

Sheba Medical Center

Other Gov

Registry information

Important dates

Study start
2005
Primary completion
2008
Study completion
2009
First posted
Jan 31, 2006
Registry last updated
Jul 27, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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