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

Primary Purpose

Hemophilia A

Status

Completed

Phase

Phase 4

Locations

Study Type

Interventional

Intervention

rFVIIa-FEIBA therapy for hemophilia A inhibitors

FEIBA- Activated Prothrombin Complexes

About this trial

This is an interventional treatment trial for Hemophilia A

Eligibility Criteria

16 Years - 65 Years (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

feiba-VIIa, hemophilia A-inhibitor therapy

Arm Description

COMBINED PATIENT- TAILORED THERAPY WITH CONCOMITANT ADMINISTRATION OF BOTH DRUGS , FOLLOWING EX VIVO THROMBIN GENERATION PREDICTING ASSAYS

Outcomes

Primary Outcome Measures

Hemostasis achieved post therapy

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

Safety

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

Secondary Outcome Measures

Time to Hemostasis

Following therapy patients documented time to "GOOD" response

Full Information

NCT ID

NCT00284193

First Posted

January 17, 2006

Last Updated

July 26, 2012

Sponsor

Sheba Medical Center

1. Study Identification

Unique Protocol Identification Number

NCT00284193

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2012

Overall Recruitment Status

Completed

Study Start Date

January 2005 (undefined)

Primary Completion Date

November 2008 (Actual)

Study Completion Date

November 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Sheba Medical Center

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia A

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

5 (Actual)

8. Arms, Groups, and Interventions

Arm Title

feiba-VIIa, hemophilia A-inhibitor therapy

Arm Type

Experimental

Arm Description

COMBINED PATIENT- TAILORED THERAPY WITH CONCOMITANT ADMINISTRATION OF BOTH DRUGS , FOLLOWING EX VIVO THROMBIN GENERATION PREDICTING ASSAYS

Intervention Type

Drug

Intervention Name(s)

rFVIIa-FEIBA therapy for hemophilia A inhibitors

Other Intervention Name(s)

NOVOSEVEN, APCC

Intervention Description

DOses tailored per ex vivo spiking thrombin generation

Intervention Type

Drug

Intervention Name(s)

FEIBA- Activated Prothrombin Complexes

Primary Outcome Measure Information:

Title

Hemostasis achieved post therapy

Description

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

Time Frame

6-24 hours

Title

Safety

Description

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

Time Frame

0-24 HOURS

Secondary Outcome Measure Information:

Title

Time to Hemostasis

Description

Following therapy patients documented time to "GOOD" response

Time Frame

0-24 HOURS

Other Pre-specified Outcome Measures:

Title

Coagulation Studies

Description

cbc fibrinogen and D-dimer were assessed pre and post therapy, thrombin generation was assayed when possible after 1-2 hours

Time Frame

0-24 HOURS

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

16 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Uri Martinowitz, MD

Organizational Affiliation

Sheba Medical Center

Official's Role

Principal Investigator

12. IPD Sharing Statement

Learn more about this trial

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

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