Clinical trial NCT01662531: A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia B
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2012-08-10
Enrollment: 27
Sex: MALE
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility 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.

Primary outcomes:
measure: Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product
timeFrame: 30 minutes after infusion
measure: Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product
timeFrame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
measure: Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast)
timeFrame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
measure: Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product
timeFrame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
measure: Number of Subjects Developing Inhibitors to Factor IX (FIX)
timeFrame: 12 months
Secondary outcomes:
measure: Number of Subjects With Treatment-related Adverse Events
timeFrame: 12 months
measure: Number of Subjects Developing Antibodies Against rIX-FP
timeFrame: 12 months
measure: Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis
timeFrame: Approximately 12 months
measure: Consumption of rIX-FP During Routine Prophylaxis
timeFrame: 12 months
Other outcomes:
Locations:
city: Parkville
country: Australia
facility: The Royal Children's Hospital, Melbourne
state: Victoria
city: Westmead
country: Australia
facility: The Children's Hospital at Westmead
city: Wien
country: Austria
facility: AKH Wien (Paediatrics)
city: Hamilton
country: Canada
facility: McMaster Children's Hospital
state: Ontario
city: Brno
country: Czech Republic
facility: Fakultni nemocnice Brno
city: Ostrava
country: Czech Republic
facility: Fakultni nemocnice Ostrava
city: Praha 5
country: Czech Republic
facility: Fakultni nemocnice Motole
city: Le Kremlin-Bicentre
country: France
facility: C.R.T.H. Hopital de Bicentre (Hemophilie)
city: Lyon
country: France
facility: Hospital Edouard Herriot
city: Marseille
country: France
facility: Hôpital d'enfants La Timone
city: Duisburg/Altstadt
country: Germany
facility: CRC Coagulation Research Center GmbH
city: Düsseldorf
country: Germany
facility: Universitätsklinikum Düsseldorf
city: Tel Hashomer
country: Israel
facility: Sheba Medical Center
city: Firenze
country: Italy
facility: AOU Careggi
city: Milano
country: Italy
facility: IRCCS Ospendale Maggiore (Centro emofilia e Trombosi)
city: Kirov
country: Russian Federation
facility: FGU "Kirov Research Institute of Haemotology and Blood Trans)
city: Madrid
country: Spain
facility: H.U. La Paz
Study officials:
affiliation: CSL Behring
name: Program Director
role: STUDY_DIRECTOR
References:
citation: Kenet G, Chambost H, Male C, Lambert T, Halimeh S, Chernova T, Mancuso ME, Curtin J, Voigt C, Li Y, Jacobs I, Santagostino E; PROLONG-9FP Investigator Study Group. Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children. Results of a phase 3 trial. Thromb Haemost. 2016 Sep 27;116(4):659-68. doi: 10.1160/TH16-03-0179. Epub 2016 Sep 1.