BAX 326 (recombinant cogulation factor IX): Continuation of a study of safety and effectiveness of the product in previously treated patients with severe or moderately severe hemophilia B.
- Conditions
- Previously treated patients (PTPs) with severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B.MedDRA version: 17.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2010-022726-33-CZ
- Lead Sponsor
- Baxter Innovations GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 100
FOR SUBJECTS TRANSITIONING FROM BAX STUDIES 250901 and 251101
• The subject and/or legal representative has/have voluntartily provided signed informed consent.
• The subject has completed Baxter Pivotal Study 250901 or Pediatric Study 251101. The subject was 12 to 65 years old at the time of screening for 250901 study or <12 years old at the time of screening for Pediatric Study 251101.
• Subject has not developed an inhibitory FIX antibody during Baxter clinical study 250901 or Pediatric Study 251101.
• Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200 particles/µL ~ <400,000 copies/mL
• Subject is immunocompetent as evidenced by a CD4 count = 200 cells/mm3.
• If female of childbearing potential, subject presents with a negative pregnancy test and agrees to continue employing adequate birth control measures for the duration of the study.
• Subject is willing and able to comply with the requirements of the protocol.
FOR NEW SUBJECTS
- Subject and/or legal representative has/have provided signed informed consent.
- Subject is 2 to 70 years old at the time of screening.
- Subject is naïve to BAX326
- Subject has severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory.
- Subject aged = 6 years is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 150 EDs.
- Subject aged < 6 years year is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 50 EDs.
- Subject has no evidence of a history of FIX inhibitors.
- Subject is immunocompetent as evidenced by a CD4 count = 200 cells/mm3 at screening.
- Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load
< 200 particles/µL ~ < 400,000 copies/mL
- If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
- Subject is willing and able to comply with the requirements of the protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
FOR SUBJECTS TRANSITIONING FROM BAX STUDIES 250901 and 251101
• The subject received factor IX product(s) other than BAX 326 upon completion of Baxter pivotal study 250901 or Pediatric Study 251101.
• The subject has been diagnosed with an acquired hemostatic defect other than hemophilia B.
• For subjects transferring from Pivotal Study 250901: The subject’s weight is < 35 kg or > 120 kg.
• The subject’s platelet count is < 100,000/mL.
• The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal).
• The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels = 5 times the upper limit of normal.
• The subject 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 a-interferon) other than anti-retroviral chemotherapy.
• The 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.
• The subject is planned to take part in any other clinical study, with the exception of BAX 326 Surgery study as described in this protocol, during the course of the Continuation Study.
• The subject is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (i.e., children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
FOR NEW SUBJECTS
- 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.
- 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.
- Subjects = 12 years of age: Subject’s weight is < 35 kg or > 120 kg
- Subject has a history of allergic reaction, eg, 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 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.
- 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.
- Subject’s platelet count is < 100,000/mL.
- 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.
- Subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agent
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method