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); MedDRA version: 15.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2011-006032-23-IT
• Lead Sponsor: CSL BEHRING GMBH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-09-27
• Last Update Posted: 6 January 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 24

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 < 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 ofinhibitor 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

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 < 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