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

NCT01757405 | Completed Phase 3 Results DMC

• 2 other identifiers: 0211012011-006294-26
• Study Type: interventional
• Target: 40
• Locations: 9 countries
• Sites: 16

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

Keywords

with Factor VIII (FVIII) or Factor IX (FIX) inhibitors

Outcome Measures

Primary Outcomes (1)

• Percentage of Bleeding Episode With "Treatment Success"

  No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.

  within 12 hours of first dose

Secondary Outcomes (5)

• Treatment Response for Each Bleeding Episode

  within 24 hours of infusion

• Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes

  24 hours post infusion

• Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs)

  6 months (throughout study period)

• Safety and Tolerability of Treatment Regimens by Clinical Assessment of Adverse Events (AEs)

  6 months (throughout study period)

• Percentage of Participants With Inhibitor Development to FVII

  6 months (throughout study period)

Study Arms (2)

≤ 3 doses of 90 µg/kg rFVIIa BI

EXPERIMENTAL

Biological: Recombinant Factor VIIa BI (rFVIIa BI)

One dose of 270 µg/kg rFVIIa BI

EXPERIMENTAL

Biological: Recombinant Factor VIIa BI (rFVIIa BI)

Interventions

Recombinant Factor VIIa BI (rFVIIa BI) BIOLOGICAL

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

≤ 3 doses of 90 µg/kg rFVIIa BI

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (16)

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

Tampa, Florida, 33607, United States

Location

Nara Medical University Hospital

Nara, 6348522, Japan

Location

Tokyo Medical University Hospital

Tokyo, 1600023, Japan

Location

Kracow Medical Center, LLC

Krakow, 31-501, Poland

Location

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

Warsaw, 02-776, Poland

Location

Louis Turcanu Emergency Clinical Children´s Hospital

Timișoara, 300011, Romania

Location

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

Kirov, 610027, Russia

Location

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

Moscow, 125167, Russia

Location

St. Petersburg City Healthcare Institution Municipal Policlinic # 37

Saint Petersburg, 195213, Russia

Location

Clinic for Hematology of the Clinical Center of Serbia

Belgrade, 11000, Serbia

Location

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

A Coruña, 15006, Spain

Location

University Hospital Virgen del Rocio

Seville, 41013, Spain

Location

Tri-Service General Hospital (TSGH)

Taipei, 11490, Taiwan

Location

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

Donetsk, 83045, Ukraine

Location

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

Kiev, 79044, Ukraine

Location

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

Lviv, Ukraine

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Interventions

recombinant FVIIa

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2012
• First Posted: December 28, 2012
• Study Start: February 20, 2013
• Primary Completion: November 11, 2014
• Study Completion: November 11, 2014
• Last Updated: May 11, 2021
• Results First Posted: October 25, 2016

Record last verified: 2021-04

Locations

JP (2); RU (3); US (1); SE (1); ES (2); TW (1); UA (3); PL (2); RO (1)