A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
Information source: CSL Behring
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hemophilia B
Intervention: rIX-FP (Biological)
Phase: Phase 3
Status: Completed
Sponsored by: CSL Behring Official(s) and/or principal investigator(s): Program Director, Study Director, Affiliation: CSL Behring
Summary
This study will examine the pharmacokinetics, safety and efficacy of rIX-FP for the control
and prevention of bleeding episodes in children who have previously received factor
replacement therapy for hemophilia B.
Clinical Details
Official title: A Phase III Open-label, Multicenter, Pharmacokinetic, Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Previously Treated Children With Hemophilia B
Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Incremental recovery of rIX-FPHalf-life (t1/2) of a single dose of rIX-FP Area under the curve (AUC) Clearance of a single dose of rIX-FP Number of subjects developing Factor IX (FIX) inhibitors
Secondary outcome: The frequency of related adverse eventsNumber of subjects developing antibodies against rIX-FP Proportion of bleeding episodes requiring one, two or more than two infusions of rIX-FP to achieve hemostasis Consumption of rIX-FP
Eligibility
Minimum age: N/A.
Maximum age: 11 Years.
Gender(s): Male.
Criteria:
Inclusion Criteria:
- Male subjects, younger than 12 years old.
- Severe hemophilia B (Factor IX [FIX] activity of ≤ 2%).
- Body weight ≥ 10 kg.
- Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for >
150 exposure days (EDs) (6 to < 12 years), and > 50 EDs (< 6 years).
- No history of FIX inhibitor formation, no detectable inhibitors at Screening and no
family history of inhibitors against FIX.
- Written informed consent for study participation.
Exclusion Criteria:
- Known hypersensitivity to any FIX product or hamster protein.
- Known congenital or acquired coagulation disorder other than congenital FIX
deficiency.
- Kidney or liver disease.
- Recent life-threatening bleeding episode.
Locations and Contacts
The Children's Hospital at Westmead, Westmead 2145, Australia
AKH Wien (Paediatrics), Wien, Austria
Fakultni nemocnice Brno, Brno 625 00, Czech Republic
Fakultni nemocnice Ostrava, Ostrava-Poruba 708 52, Czech Republic
Fakultni nemocnice Motole, Praha 5 150 06, Czech Republic
C.R.T.H. Hop Bicetre (Hemophilie), Le Kremlin-Bicetre 94270, France
Hospital Edouard Herriot, Lyon 69003, France
Assistance Publique Hopitaux de Marseille (APHM) - Centre Ho, Marseille 13005, France
Heinrich-Heine-Universitaet, Duesseldorf 40225, Germany
CRC Coagulation Research Center GmbH, Duisburg/Altstadt 47051, Germany
Sheba Medical Center, Tel Hashomer 52621, Israel
AOU Careggi, Firenze 50134, Italy
IRCCS Ospedale Maggiore (Centro emofilia e Trombosi), Milano 20122, Italy
FGU "Kirov Research Institute of Haemotology and Blood Trans), Kirov 610027, Russian Federation
H.U. La Paz, Madrid 28046, Spain
McMaster University, Hamilton, Ontario L8L 2X2, Canada
The Henry Ekert Haemophilia Treatment Centre, Parkville, Victoria 3052, Australia
Additional Information
Starting date: September 2012
Last updated: December 15, 2014
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