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Completed

NCT Number: NCT01757405

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

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.

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Key information

Conditions

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

Age range

12 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Nara Medical University Hospital, Nara, Japan

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Recombinant Factor VIIa BI (rFVIIa BI)

Biological

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

Primary outcomes

  1. Percentage of Bleeding Episode With "Treatment Success"

    Time frame: within 12 hours of first dose

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

Secondary outcomes

  1. Treatment Response for Each Bleeding Episode

    Time frame: within 24 hours of infusion

    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.

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

    Time frame: 24 hours post infusion

    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).

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

    Time frame: 6 months (throughout study period)

    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).

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

    Time frame: 6 months (throughout study period)

    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]).

  5. Percentage of Participants With Inhibitor Development to FVII

    Time frame: 6 months (throughout study period)

    Development of rFVII inhibitors or FVIIa binding antibodies during the study.

Sponsors and collaborators

Lead sponsor

Baxalta now part of Shire

Industry

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2014
Study completion
2014
First posted
Dec 28, 2012
Registry last updated
May 11, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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