Clinical trial NCT01757405: Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
Official title: A PHASE 3, PROSPECTIVE, OPEN-LABEL, RANDOMIZED STUDY TO EVALUATE SAFETY AND EFFICACY OF RECOMBINANT ACTIVATED FVII BI (rFVIIa BI) IN THE TREATMENT OF ACUTE BLEEDING EPISODES PER AN ON-DEMAND REGIMEN IN PATIENTS WITH HEMOPHILIA A OR B WITH INHIBITORS
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia A
Hemophilia B
Keywords: with Factor VIII (FVIII) or Factor IX (FIX) inhibitors
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2012-12-28
Enrollment: 40
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Main 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.

Main 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.

Primary outcomes:
measure: Percentage of Bleeding Episode With "Treatment Success"
timeFrame: within 12 hours of first dose
Secondary outcomes:
measure: Treatment Response for Each Bleeding Episode
timeFrame: within 24 hours of infusion
measure: Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes
timeFrame: 24 hours post infusion
measure: Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs)
timeFrame: 6 months (throughout study period)
measure: Safety and Tolerability of Treatment Regimens by Clinical Assessment of Adverse Events (AEs)
timeFrame: 6 months (throughout study period)
measure: Percentage of Participants With Inhibitor Development to FVII
timeFrame: 6 months (throughout study period)
Other outcomes:
Locations:
city: Tampa
country: United States
facility: Health Point Medical Group "St Joseph's Children's Hospital"
state: Florida
city: Nara
country: Japan
facility: Nara Medical University Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical University Hospital
city: Krakow
country: Poland
facility: Kracow Medical Center, LLC
city: Warszawa
country: Poland
facility: Institute of Haematology and Transfusion Medicine, Clinic of Haemostatic Disorders and Internal Diseases
city: Timisoara
country: Romania
facility: Louis Turcanu Emergency Clinical Children´s Hospital
city: Kirov
country: Russian Federation
facility: Kirov Hematology and Blood Transfusion Research Institute under the Federal Medical and Biological Agency of Russia
city: Moscow
country: Russian Federation
facility: Hematology Research Center under RAMS (State Institution), Department of Reconstructive Orthopedic Surgery for Hemophilia Patients
city: St. Petersburg
country: Russian Federation
facility: St. Petersburg City Healthcare Institution Municipal Policlinic # 37
city: Belgrade
country: Serbia
facility: Clinic for Hematology of the Clinical Center of Serbia
city: A Coruña
country: Spain
facility: Hospital Teresa Herrera Materno Infantil del C.H.U.Carretera del Pasajes/nlaboratorio de hematología
city: Sevilla
country: Spain
facility: University Hospital Virgen del Rocio
city: Taipei City
country: Taiwan
facility: Tri-Service General Hospital (TSGH)
city: Donetsk
country: Ukraine
facility: V.K. Gusak Institute of Urgent and Reconstructive Surgery within the Ukrainian National Academy of Medical Sciences, Hematology Department
city: Kiev
country: Ukraine
facility: Kyiv City Clinical Hospital #9, City Scientific-Practical Center for Diagnostics and Treatment of Patients with Hemostatic Pathlogies
city: Lviv
country: Ukraine
facility: State Institution "Institute of Blood Pathology and Transfusion Medicine within the Ukrainian National Academy of Medical Sciences", Hematology Department
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: