Hemophilia B
Conditions
Interventions
Trade Name: Rixubis
Pharmaceutical Form: Powder for injection
Sponsors
Baxalta Innovations GmbH
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. The subject or legally authorized representative (in case of study participants =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject has known hypersensitivity or presence of any contraindication to RIXUBIS or its excipients including hamster protein. 2. Subject has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC). 3. Subject has a history of FIX inhibitors with a titer = 0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay, employed in the respective local laboratory) at any time prior to screening. 4. Subject has a detectable FIX inhibitor at screening, with a titer = 0.6 BU as determined by the Nijmegen modification of the Bethesda assay in the central laboratory. 5. Subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4 hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices. 6. Subject has severe chronic hepatic dysfunction [eg, = 5 times upper limit of normal alanine aminotransferase (ALT), as confirmed by central laboratory at screening, or a documented INR > 1.5]. 7. Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening. 8. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B. 9. Subject’s platelet count is < 100,000/mL. 10. Subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject’s safety or compliance. 11. Subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or a-interferon) other than antiretroviral chemotherapy. 12. Subject has participated in another clinical study involving an 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. 13. Subject is a family member or employee of the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to assess the safety of RIXUBIS based on SAEs (including FIX inhibitors);Primary end point(s): Incidence of SAEs (including FIX inhibitors) possibly or probably related to RIXUBIS;Timepoint(s) of evaluation of this end point: From start of study drug administration up to end of treatment (EOT) (up to 6 months);Secondary Objective: - To determine the safety of RIXUBIS based on AEs - To determine the safety of RIXUBIS based on changes in laboratory parameters - To determine the immunogenicity of RIXUBIS (excluding FIX inhibitors) - To assess the efficacy of prophylactic treatment with RIXUBIS - To assess the efficacy of RIXUBIS in the control of bleeding episodes | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Incidence of AEs possibly or probably related to RIXUBIS - Clinically significant changes in clinical laboratory parameters (hematology and clinical chemistry) - Incidence of binding IgG and IgM antibodies to FIX - Incidence of antibodies to CHO proteins and rFurin - Annualized bleeding rate with prophylactic use of RIXUBIS - Rate of success of RIXUBIS for treatment of bleeding episodes;Timepoint(s) of evaluation of this end point: From start of study drug administration up to EOT (up to 6 months) | — |
Countries
India
Contacts
Public ContactClinical Trial Transparency
Baxalta
Outcome results
None listed