NCT01757405CompletedPhase 3
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
Baxalta now part of Shire31 sites in 9 countries40 target enrollmentStarted: February 20, 2013Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Enrollment
- 40
- Locations
- 31
- Primary Endpoint
- Percentage of Bleeding Episode With "Treatment Success"
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Years to 65 Years (Child, Adult, Older Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion 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 ≥
- •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
- •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.
Outcomes
Primary Outcomes
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
- Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs)(6 months (throughout study period))
- Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes(24 hours post infusion)
- Treatment Response for Each Bleeding Episode(within 24 hours of infusion)
- 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))
Investigators
Study Sites (31)
Loading locations...
Similar Trials
Completed
Phase 3
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia AHemophilia ANCT02234323Bioverativ, a Sanofi company108
Completed
Phase 2
Randomized, Placebo Controlled, Crossover Study in an Environmental Challenge Chamber to Assess Safety & Efficacy of Three Oral Doses of BI 671800 Versus Fluticasone Propionate and Montelukast in Sensitive Seasonal Allergic Rhinitis Patients Out of SeasonRhinitis, Allergic, SeasonalAsthmaNCT01007721Boehringer Ingelheim146
Terminated
Phase 3
Ribavirin to Prevent RSV Pneumonia in Bone Marrow Transplant PatientsRespiratory Syncytial Virus InfectionsNCT00031473National Institute of Allergy and Infectious Diseases (NIAID)90
Withdrawn
Phase 3
Open Label Trial to Compare BI 207127 to Telaprevir in HCV PatientsHepatitis C, ChronicNCT01858961Boehringer Ingelheim
Terminated
Phase 2
This Study Tests Whether BI 409306 Prevents Patients With Schizophrenia From Becoming Worse. This Study Looks at How Well Patients Tolerate BI 409306 and How Effective it is Over 6 MonthsSchizophreniaNCT03351244Boehringer Ingelheim264