Prophylaxis and treatment of bleeding episodes in previously treated children with congenital FIX deficiency (hemophilia B) MedDRA version: 17.1 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850
Conditions
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: •Male subjects, aged younger than 12 years (Prior to their 12th birthday at Day 1), and body weight = 10 kg at the time of screening visit •Documented severe hemophilia B (FIX activity of = 2%) •Subjects who are currently receiving routine FIX replacement therapy and have received FIX products for > 150 EDs (6 to 50 EDs (=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: •Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or hamster protein. •Known congenital or acquired coagulation disorder other than congenital FIX deficiency. •Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment. •Platelet count 5 x upper limit of normal (ULN) at Screening. •Serum creatinine concentration > 2 x ULN at Screening. •Experienced a life-threatening bleeding episode or had major surgical intervention within 4 months prior to dosing on Day 1. •Evidence of thrombosis, including deep vein thrombosis, stroke, myocardial infarction or arterial embolus within 4 months prior to dosing on Day 1. •Planning to have major surgical procedure during the study period. •Use of any Investigational Medicinal Product (IMP) other than rIX-FP within 4 weeks prior to the first day of rIX-FP administration. •Participated in any extended half-life Investigational FIX product clinical study other than for rIX-FP. •Concurrent inflammatory joint disease or other medical condition that could confound study results. •Suspected inability or unwillingness of study subjects and/or caregiver to comply with study procedures or history of noncompliance.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To evaluate the PK of a single dose of rIX-FP. •To evaluate the safety of rIX-FP with respect to the development of inhibitors to FIX in patients with severe hemophilia B (FIX: = 2%). ;Secondary Objective: •To evaluate the safety of rIX-FP, based on AEs and the development of antibodies to rIX-FP. •To evaluate the clinical response of rIX-FP for the prevention of bleeding episodes. •To evaluate the clinical response of rIX-FP in treatment bleeding episodes. ;Primary end point(s): •Incremental recovery (IU/ml/IU/kg) of 50 IU/kg rIX-FP. •Half-life (t1/2) of a single dose of 50 IU/kg rIX-FP. •AUC of the last sample with quantifiable drug concentration (AUC0-t) of a single dose of 50 IU/kg rIX-FP. •Clearance of a single dose of 50 IU/kg rIX-FP. •The number of subjects with FIX inhibitors. ;Timepoint(s) of evaluation of this end point: 10-14 days for the PK assessment. approximately 12 months for the inhibitor assessment. (throughout the study) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •The frequency of related AEs to rIX-FP over the course of the study. •The number of subjects who developed antibodies against rIX-FP. •The consumption of rIX-FP, as expressed as number of infusion and IU/kg per month and per year, as well as IU/kg per event. •Proportion of bleeding episodes requiring one, two or more than two infusions of rIX-FP to achieve hemostasis. ;Timepoint(s) of evaluation of this end point: approximately 12 months (throughout the study) | — |
Countries
Australia, Austria, Canada, Czech Republic, France, Germany, Italy, Russian Federation, Spain
Contacts
Public ContactProject Leader
PAREXEL International
Outcome results
None listed