Health Condition 1: null- Hemophilia B Pediatric Patients With Age less than 12 years
Conditions
Interventions
Intervention1: recombinant factor ix: Prophylactic treatment: twice weekly.
Planned duration of subject participation: 7 months
ROute of administration: Intravenous
Control Intervention1: NA: NA
Sponsors
Baxter Innovation GmBH
Eligibility
Inclusion criteria
Inclusion criteria: Inclusion criteria: • Subject and/or legal representative has/have voluntarily provided signed informed consent • Subject has severe (FIX level • Subject is • Subject 6 to If a subject does not have a verifiable, documented history of 150 EDs, s/he can be enrolled if the following criteria are met: 1) there are an estimated 100 - 150 EDs to any FIX product (plasma-derived or recombinant FIX concentrate(s), prothrombin complex concentrate ï??PCCï?? or fresh frozen plasma ï??FFPï?? that are not fully documented (assumption based on the severity of disease and treatment history), and 2) s/he has participated in Immunine Study 050901 and accumulated either at least 50 EDs to Immunine or a total of at least 150 EDs to a plasma-derived and/or recombinant FIX concentrate prior to enrollment • Subject 50 EDs (based on the subjectâ??s medical records). If a subject does not have a verifiable, documented history of >50 EDs, s/he can be enrolled if the following criteria are met: 1) there are approximately 20 - 50 EDs to any FIX product (plasma-derived or recombinant FIX concentrate(s), PCC or FFP) that are not fully documented, and 2) s/he has participated in Immunine Study 050901 and accumulated a minimum of 30 EDs to Immunine or a total of > 50 EDs to a plasma-derived and/or recombinant FIX concentrate prior to enrollment • Subject has no evidence of a history of FIX inhibitors (based on the subjectâ??s medical records). If a verifiable, documented history is unavailable, the subject can be enrolled if s/he has participated in Study 050901 for at least 30 EDs ( • Subject and/or legal representative accepts prophylactic treatment over a period of 6 months
Exclusion criteria
Exclusion criteria: • Subject has a history of FIX inhibitors with a titer >= 0.6 BU (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory with the corresponding detection limit) at any time prior to screening • 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 • Subject has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s) • Subject has a known hypersensitivity to hamster proteins or rFurin • Subject has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC) • Subject has an abnormal renal function (serum creatinine 1.5 times the upper limit of normal) • Subject has an International Normalized Ratio (INR) 1.4 • Subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels 5 times the upper limit of normal • Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B • Subjectâ??s platelet count is 100,000/mL • Subject has a clinically significant medical, psychiatric, or cognitive illness, that, in the opinion of the Investigator, would affect subjectâ??s safety or compliance • Subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than anti-retroviral chemotherapy • Subject has participated in another investigational study within 30 days of enrollment. However, participation in Study 050901 with Immunine is allowed.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| All AEs possibly or probably related to BAX326Timepoint: 8 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Annualized bleeding rateTimepoint: 8 months;Area under the plasma concentration versus time curve from 0 to 72 hours post-infusion Timepoint: 8 months;Efficacy assessments: Determination of factor IX level and pharmacokinetic parameters at certain time points, including incremental recovery; rating scale for treatment of bleeding episodes (excellent / good / fair / none)Timepoint: 8 months;Hemostatic efficacy of the acute management of bleeding episodesTimepoint: 8 months | — |
Countries
Bulgaria, India, Poland, Romania, Russian Federation, Ukraine, United Kingdom
Contacts
Public ContactDr Ashok Moharana
Baxter (India) Private Limited
Outcome results
None listed