Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen

NCT ID: NCT01757405

Last Updated: 2021-05-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-02-20
• Study Completion Date: 2014-11-11

Brief Summary

The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.

Conditions

Hemophilia A; Hemophilia B

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

≤ 3 doses of 90 µg/kg rFVIIa BI

Group Type: EXPERIMENTAL

Recombinant Factor VIIa BI (rFVIIa BI)

Intervention Type: BIOLOGICAL

Administered approximately every 3 hours as an intravenous bolus injection on-demand

One dose of 270 µg/kg rFVIIa BI

Group Type: EXPERIMENTAL

Recombinant Factor VIIa BI (rFVIIa BI)

Intervention Type: BIOLOGICAL

Administered as a single intravenous bolus injection on-demand

Interventions

Recombinant Factor VIIa BI (rFVIIa BI)

Administered approximately every 3 hours as an intravenous bolus injection on-demand

Intervention Type: BIOLOGICAL

Recombinant Factor VIIa BI (rFVIIa BI)

Administered as a single intravenous bolus injection on-demand

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Participant is male with hemophilia A or B with inhibitors, with a high titer (≥5 Bethesda unit (BU)) or a historical high anamnestic response.
• Participant is 12 to 65 years old at the time of screening.
• Participant is currently using or has used bypassing agents for treatment of bleeding episodes.
• Participant has an annualized bleed rate of 5 or more bleeding episodes per year on average over the 2 years prior to the Screening visit.
• Participant has a Karnofsky Performance Score ≥60.
• Participant is hepatitis C virus negative (HCV-) either by antibody testing or polymerase chain reaction (PCR); or hepatitis C virus positive (HCV+) with stable hepatic disease.
• Participant is human immunodeficiency virus negative (HIV-) or HIV+ with stable disease, CD4+ count ≥200 cells/mm^3 at screening.
• Participant is willing and able to comply with the requirements of the protocol.

Exclusion Criteria

• Participant is not willing to go on an on-demand treatment scheme.
• Participant is positive for a FVII inhibitor at screening.
• Participant has clinically symptomatic liver disease.
• Participant has a platelet count <100,000/µL.
• The use of α-interferon with or without ribavirin is planned for an HCV-infected participant or the use of a protease inhibitor is planned for an HIV-infected participant.

• Participants currently taking any of these medications for ≥30 days are eligible.
• Participant has a known hypersensitivity to rFVIIa, hamster or murine proteins, or Tween 80.
• Participant has a known history of being non-responsive to rFVIIa treatment of bleeding episodes.
• Participant has a prior history of thromboembolic event or diagnosis of other diseases that may increase the participant's risk of thromboembolic complications.
• Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
• Participant is a family member or employee of the investigator.
• Participant is scheduled for surgery during the study period.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Shire

Locations

Health Point Medical Group "St Joseph's Children's Hospital"

Tampa, Florida, United States

Nara Medical University Hospital

Nara, , Japan

Tokyo Medical University Hospital

Tokyo, , Japan

Kracow Medical Center, LLC

Krakow, , Poland

Institute of Haematology and Transfusion Medicine, Clinic of Haemostatic Disorders and Internal Diseases

Warsaw, , Poland

Louis Turcanu Emergency Clinical Children´s Hospital

Timișoara, , Romania

Kirov Hematology and Blood Transfusion Research Institute under the Federal Medical and Biological Agency of Russia

Kirov, , Russia

Hematology Research Center under RAMS (State Institution), Department of Reconstructive Orthopedic Surgery for Hemophilia Patients

Moscow, , Russia

St. Petersburg City Healthcare Institution Municipal Policlinic # 37

Saint Petersburg, , Russia

Clinic for Hematology of the Clinical Center of Serbia

Belgrade, , Serbia

Hospital Teresa Herrera Materno Infantil del C.H.U.Carretera del Pasajes/nlaboratorio de hematología

A Coruña, , Spain

University Hospital Virgen del Rocio

Seville, , Spain

Tri-Service General Hospital (TSGH)

Taipei, , Taiwan

V.K. Gusak Institute of Urgent and Reconstructive Surgery within the Ukrainian National Academy of Medical Sciences, Hematology Department

Donetsk, , Ukraine

Kyiv City Clinical Hospital #9, City Scientific-Practical Center for Diagnostics and Treatment of Patients with Hemostatic Pathlogies

Kiev, , Ukraine

State Institution "Institute of Blood Pathology and Transfusion Medicine within the Ukrainian National Academy of Medical Sciences", Hematology Department

Lviv, , Ukraine

Countries

United States; Japan; Poland; Romania; Russia; Serbia; Spain; Taiwan; Ukraine

Other Identifiers

2011-006294-26

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

021101

Identifier Type: -

Identifier Source: org_study_id