A safety and Efficacy Study of a recombinant Factor IX in pediatric patients with severe Hemophilia B
- Conditions
- Prophylaxis and treatment of bleeding episodes in previously treated children with congenital FIX deficiency (hemophilia B).Therapeutic area: Body processes [G] - Genetic Phenomena [G05]MedDRA version: 14.1Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2011-006032-23-ES
- Lead Sponsor
- CSL Behring GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 24
-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 -Subjects who are currently receiving routine FIX replacement therapy and have received FIX products for > 150 EDs (6 to < 12 years of age) or > 50 EDs (< 6 years of age), confirmed by their treating physician.
-No confirmed prior history of FIX inhibitor formation (defined as two consecutive positive tests-requiring a confirmatory test on a second separately drawn blood sample shortly after the previous positive test), no confirmed detectable inhibitors (defined as < 0.6 Bethesda Units [BU]) at Screening by the central laboratory, and no family history of inhibitor formation against FIX.
-Written informed consent for study participation obtained before undergoing any study specific procedures.
-Ability of subject/caregiver to assess a bleeding episode, and document treatment with an electronic diary.
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
-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 < 100,000/µL at Screening.
-Documented HIV positive subjects with a CD4 count < 200/mm3
-Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) concentration > 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: -To evaluate the PK of a single dose of rIX-FP.<br>-To evaluate the safety of rIX-FP with respect to the development of inhibitors to FIX in patients with severe hemophilia B (FIX: <or=2%).;Secondary Objective: -To evaluate the safety of rIX-FP, based on AEs and the development of antibodies to rIX-FP.<br>-To evaluate the clinical response of rIX-FP for the prevention of bleeding episodes.<br>-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.<br>-Half-life (t1/2) of a single dose of 50 IU/kg rIX-FP. <br>-AUC of the last sample with quantifiable drug concentration (AUC0-t) of a single dose of 50 IU/kg rIX-FP.<br>-Clearance of a single dose of 50 IU/kg rIX-FP.<br>-The number of subjects with FIX inhibitors.;Timepoint(s) of evaluation of this end point: 10-14 days for the PK assessment.<br>approximately 12 months for the inhibitor assessment. (throughout the study)
- Secondary Outcome Measures
Name Time Method Secondary end point(s): -The frequency of related AEs to rIX-FP over the course of the study.<br>-The number of subjects who developed antibodies against rIX-FP.<br>-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. <br>-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)