Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor

NCT ID: NCT00851721

Last Updated: 2021-05-19

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

52 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-03-31

Study Completion Date

2012-10-17

Brief Summary

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The purpose of the study was to determine the efficacy, safety, and health-related quality of life benefits with FEIBA NF prophylactic treatment as compared with on-demand treatment.

Detailed Description

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Conditions

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Hemophilia A or B With Inhibitors Hemophilia A Hemophilia B

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Prophylaxis arm

Group Type EXPERIMENTAL

Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)

Intervention Type BIOLOGICAL

85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period

On-demand arm

Group Type ACTIVE_COMPARATOR

Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)

Intervention Type BIOLOGICAL

FEIBA NF dose and dosing interval as prescribed by the treating physician

Interventions

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Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)

85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period

Intervention Type BIOLOGICAL

Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)

FEIBA NF dose and dosing interval as prescribed by the treating physician

Intervention Type BIOLOGICAL

Other Intervention Names

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FEIBA NF FEIBA NF

Eligibility Criteria

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Inclusion Criteria

* Signed and dated informed consent form by the participant or the participant's legally authorized representative
* The participant is ≥ 4 to ≤ 65 years of age
* The participant has a Karnofsky performance score of ≥ 60
* Hemophilia A and B of any severity, with documented history of high-titer inhibitor (\> 5 Bethesda unit (BU)) for at least 12 months; or, if inhibitor titer is ≤ 5 BU, and the participant is refractory with increased dosing of either factor VIII (FVIII) or factor IX (FIX), as demonstrated from the participant's medical history
* Currently being treated on an on-demand basis for treatment of bleeding episodes
* Adequate venous access, with or without central venous device
* ≥ 12 bleeding episodes requiring treatment with by-passing agents in the past 12 months, based on medical history
* Competent in-home treatment and infusion therapy
* Currently using bypassing agents (activated prothrombin complex concentrate (APCC) or recombinant activated factor VII (rFVIIa)) for treatment of bleeding episodes
* HCV-, either by antibody testing or polymerase chain reaction (PCR); or HCV+ with stable hepatic disease
* HIV-, or HIV+ with stable disease and CD4 count \> 200 cells/mm3 at screening
* Female participant of childbearing potential, presents with a negative serum pregnancy test, and agrees to employ adequate birth control measures for the duration of the study

Exclusion Criteria

* Currently receiving immune tolerance induction (ITI)
* Currently on regular prophylactic therapy to prevent bleeding episodes
* Clinically symptomatic liver disease (e.g. diagnosis of cirrhosis \[confirmed by liver biopsy\], portal vein hypertension, ascites, prothrombin time (PT) 5 seconds above upper limit of normal)
* Platelet count \< 100,000/ml
* Planned elective surgery during participation in this study
* Participant is currently participating in another clinical study and has received an investigational product or device within 30 days prior to study entry
* Planned use of pegylated or non-pegylated alpha-interferon with or without ribavirin for HCV infected participants or planned use of a protease inhibitor for HIV infected participants. Participants currently taking any of these medications for a 30-day course are eligible.
* Clinically significant increase in D-dimer levels from historical baseline and/or associated with chronic liver disease or clinically evident thromboembolic event
* Known hypersensitivity to anti-inhibitor coagulant complexes (AICCs)
* Currently treated with a systemic immunomodulating drug
* Prior history of thromboembolic event: acute myocardial infarction, deep vein thrombosis, or pulmonary embolism
* Diagnosis of advanced atherosclerosis, malignancy and/or other diseases that may increase the participant's risk of thromboembolic complications
* Clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance
Minimum Eligible Age

4 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Baxalta now part of Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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Chicago, Illinois, United States

Site Status

Cleveland, Ohio, United States

Site Status

Rio de Janeiro, Rio de Janeiro, Brazil

Site Status

São Paulo, São Paulo, Brazil

Site Status

Sofia, , Bulgaria

Site Status

Zagreb, , Croatia

Site Status

Kanagawa, , Japan

Site Status

Nara, , Japan

Site Status

Wellington, , New Zealand

Site Status

Krakow, , Poland

Site Status

Warsaw, , Poland

Site Status

Bucharest, , Romania

Site Status

Timișoara, , Romania

Site Status

Kirov, , Russia

Site Status

Moscow, , Russia

Site Status

Yekaterinburg, , Russia

Site Status

Lviv, , Ukraine

Site Status

Countries

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United States Brazil Bulgaria Croatia Japan New Zealand Poland Romania Russia Ukraine

References

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Antunes SV, Tangada S, Stasyshyn O, Mamonov V, Phillips J, Guzman-Becerra N, Grigorian A, Ewenstein B, Wong WY. Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors. Haemophilia. 2014 Jan;20(1):65-72. doi: 10.1111/hae.12246. Epub 2013 Aug 1.

Reference Type RESULT
PMID: 23910578 (View on PubMed)

Other Identifiers

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2008-003855-65

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

090701

Identifier Type: -

Identifier Source: org_study_id

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