Pediatric previously treated patients (PTPs) with severe (FIX level < 1%) or moderately severe (FIX level = 2%) hemophilia B. MedDRA version: 14.1 Level: LLT Classification code 10018939 Term: Haemophilia B (Factor IX) System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Recombinant Coagulation Factor IX
Product Code: BAX326
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: NONACOG ALFA
CAS Number: 181054-95-5
Curren
Sponsors
Baxter Innovations GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Subject and/or legal representative has/have voluntarily provided signed informed consent • Subject has severe (FIX level 50 EDs (based on the subject’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 subject’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 (=65 years) no F.1.3.1 Number of subjects for this age range
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 • Subject’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 subject’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 a-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 |
|---|---|
| Secondary Objective: • To evaluate the PK parameters of BAX326 in pediatric PTPs < 12 years of age • To monitor incremental recovery (IR) of BAX326 over time • To evaluate the hemostatic efficacy of BAX326 in the management and prevention of acute bleeding episodes for a period of 6 months • To evaluate safety in terms of immunogenicity for a minimum of 50 exposure days (EDs), the occurrence of thrombotic events, as well as clinically significant changes in routine laboratory parameters (hematology/clinical chemistry) and vital signs. • To evaluate changes in HR QoL and health resource use ;Primary end point(s): • All AEs possibly or probably related to BAX326;Timepoint(s) of evaluation of this end point: Ongoing (see protocol);Main Objective: To assess BAX326 pharmacokinetic (PK) parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life (HR QoL) in pediatric patients. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • PK: o Area under the plasma concentration versus time curve from 0 to 72 hours post-infusion (AUC0-72 h/dose), total AUC/dose, mean residence time (MRT), clearance (CL), incremental recovery (IR), elimination phase half-life (T1/2), volume of distribution at steady state (Vss) o IR over time • Hemostatic efficacy: o Treatment of bleeding episodes: number of infusions per bleeding episode, overall hemostatic efficacy rating at resolution of bleed o Prophylaxis: annualized bleeding rate o Prophylaxis: number of bleeding episodes beginning within 24 and 48 hours of an infusion as exploratory outcome measures o Consumption of BAX326: number of infusions and weight-adjusted consumption per month and per year; weight-adjusted consumption per event • Safety and immunogenicity: o Development of inhibitory and total binding antibodies to FIX o Occurrence of severe allergic reactions, e.g. anaphylaxis o Occurrence of thrombotic events o Clinically significant changes in routine laboratory parameters (hematology and clinical chemistry) and vital signs o Development of antibodies to Chinese hamster ovary (CHO) proteins and recombinant furin (rFurin) • Changes in the following HR QoL parameters and health resource use For subjects who are between 2 to 7 years of age: o Generic: PedsQL™ (Parent-proxy versions: age group 2-4 years and age group 5-7 years) o Health resource use (hospitalizations, emergency room visits, doctor office visits, etc.) For subjects who are between 8 to 11 years of age: o Disease-specific: Haemo-QoL, short version o Generic: PedsQL™ Child version o Health resource use (hospitalizations, emergency room visits, doctor office visits, etc.) ;Timepoint(s) of evaluation of this end point: Ongoing (see protocol) | — |
Countries
Bulgaria, Czech Republic, Germany, India, Poland, Romania, Russian Federation, Ukraine, United Kingdom
Contacts
Public ContactGuido Wuerth, Clinical Operations
Baxter Innovations GmbH
Outcome results
None listed