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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01757405
Other study ID # 021101
Secondary ID 2011-006294-26
Status Completed
Phase Phase 3
First received
Last updated
Start date February 20, 2013
Est. completion date November 11, 2014

Study information

Verified date April 2021
Source Takeda
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 40
Est. completion date November 11, 2014
Est. primary completion date November 11, 2014
Accepts healthy volunteers No
Gender Male
Age group 12 Years to 65 Years
Eligibility 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 a-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.

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
Recombinant Factor VIIa BI (rFVIIa BI)
Administered approximately every 3 hours as an intravenous bolus injection on-demand
Recombinant Factor VIIa BI (rFVIIa BI)
Administered as a single intravenous bolus injection on-demand

Locations

Country Name City State
Japan Nara Medical University Hospital Nara
Japan Tokyo Medical University Hospital Tokyo
Poland Kracow Medical Center, LLC Krakow
Poland Institute of Haematology and Transfusion Medicine, Clinic of Haemostatic Disorders and Internal Diseases Warszawa
Romania Louis Turcanu Emergency Clinical Children´s Hospital Timisoara
Russian Federation Kirov Hematology and Blood Transfusion Research Institute under the Federal Medical and Biological Agency of Russia Kirov
Russian Federation Hematology Research Center under RAMS (State Institution), Department of Reconstructive Orthopedic Surgery for Hemophilia Patients Moscow
Russian Federation St. Petersburg City Healthcare Institution Municipal Policlinic # 37 St. Petersburg
Serbia Clinic for Hematology of the Clinical Center of Serbia Belgrade
Spain Hospital Teresa Herrera Materno Infantil del C.H.U.Carretera del Pasajes/nlaboratorio de hematología A Coruña
Spain University Hospital Virgen del Rocio Sevilla
Taiwan Tri-Service General Hospital (TSGH) Taipei City
Ukraine 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
United States Health Point Medical Group "St Joseph's Children's Hospital" Tampa Florida

Sponsors (1)

Lead Sponsor Collaborator
Baxalta now part of Shire

Countries where clinical trial is conducted

United States,  Japan,  Poland,  Romania,  Russian Federation,  Serbia,  Spain,  Taiwan,  Ukraine, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 Treatment Response for Each Bleeding Episode Participants rated the treatment of each bleeding episode. If treatment occurred under direct supervision of treating physician, the physician rated the response. Ratings based on a 4 point scale; EXCELLENT - full relief of pain and cessation of objective signs of bleeding (swelling, tenderness, decrease in range of motion [for muscle bleeds]) within 9 hours of treatment initiation. No additional infusion required to control bleeding, other than prescribed dosing regimen. GOOD - Substantial relief of pain and/or cessation of objective signs of bleeding within 9 hours of treatment initiation. No additional infusion required to control bleeding, other than prescribed dosing regimen. MODERATE - slight relief of pain and slight improvement of signs of bleeding within 9 hours of treatment initiation. Requires additional infusion beyond treatment regimen. NONE - No improvement or condition worsens. SUCCESSFUL = EXCELLENT or GOOD. within 24 hours of infusion
Secondary Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes Clinical responders defined as sustained bleeding control, (no additional hemostatic medication including rFVIIa BI required between 12 and 24 hours after first infusion of the successfully treated bleeding episode). 24 hours post infusion
Secondary Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs) Safety was determined by the number of AEs (both serious AEs [SAEs] and non-serious AEs [nsAE]). Tolerability was determined by the number of AEs related to rFVIIa BI (both SAEs and nsAEs) as determined by causality assessment of the AEs by the investigator. An AE was deemed Related if the investigator judged the AE to be "possibly related" or "probably related" to rFVIIa BI. The percentage of participants with AEs were presented by seriousness (SAE, nsAE), severity (Mild, Moderate or Severe) and causality (Related or Not Related to rFVIIa BI). 6 months (throughout study period)
Secondary Safety and Tolerability of Treatment Regimens by Clinical Assessment of Adverse Events (AEs) Safety was determined by the number of AEs (both serious AEs [SAEs] and non-serious AEs [nsAE]). Tolerability was determined by the number of AEs related to rFVIIa BI (both SAEs and nsAEs) as determined by causality assessment of the AEs by the investigator. An AE was deemed Related if the investigator judges the AE to be "possibly related" or "probably related" to rFVIIa BI. The percentage of AEs were presented by seriousness (SAE, nsAE), severity (Mild, Moderate or Severe) and causality (Related or Not Related [to rFVIIa BI]). 6 months (throughout study period)
Secondary Percentage of Participants With Inhibitor Development to FVII Development of rFVII inhibitors or FVIIa binding antibodies during the study. 6 months (throughout study period)
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