A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
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|ClinicalTrials.gov Identifier: NCT01662531|
Recruitment Status : Completed
First Posted : August 10, 2012
Results First Posted : May 9, 2016
Last Update Posted : May 9, 2016
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia B||Biological: rIX-FP||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||27 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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 Start Date :||January 2013|
|Actual Primary Completion Date :||October 2014|
Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) will be administered by IV infusion as routine weekly prophylaxis and episodic treatment for bleeding episodes.
Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)
- Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product [ Time Frame: 30 minutes after infusion ]Incremental recovery (IU/dL/IU/kg) is defined as the FIX activity (IU/dL) obtained 30 minutes following infusion, per dose of (IU/kg) infusion. FIX activity was measured at a central laboratory using validated one-stage clotting method. Recovery values were baseline-corrected for pre-infusion plasma FIX activity. Incremental recovery was measured following a single intravenous dose of 50 IU/kg rIX-FP on Day 1. Analysis of previous FIX product was conducted at the beginning of the study in a subset of subjects who had no historical pharmacokinetic (PK) data of their previous FIX product. For the PK assessment, the previous FIX product was administered by IV infusion after approximately 4 days following the last FIX treatment, prior to any dosing of rIX-FP. The formal PK population consisted of subjects who received at least 1 dose of rIX-FP for PK assessment and for whom a sufficient number of analyzable PK samples had been obtained to permit the evaluation of the PK profile of rIX-FP.
- Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values.
- Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast) [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]
AUClast following a single intravenous dose of 50 IU/kg rIX-FP or previous FIX product.
FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values.
- Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values. Clearance is normalized for body weight.
- Number of Subjects Developing Inhibitors to Factor IX (FIX) [ Time Frame: 12 months ]Inhibitor formation was defined as any inhibitor (≥0.6 BU [Bethesda Units]/mL) identified and confirmed by retesting.
- Number of Subjects With Treatment-related Adverse Events [ Time Frame: 12 months ]
- Number of Subjects Developing Antibodies Against rIX-FP [ Time Frame: 12 months ]Antibodies to rIX-FP were measured using a direct-binding enzyme-linked immunosorbent assay (ELISA).
- Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis [ Time Frame: Approximately 12 months ]For each bleeding episode that required treatment, the number of episodes that required one, two or more than two infusions of rIX-FP to achieve hemostasis
- Consumption of rIX-FP During Routine Prophylaxis [ Time Frame: 12 months ]Consumption of rIX-FP during routine prophylaxis is expressed as the total prophylaxis dose per month.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01662531
|The Royal Children's Hospital, Melbourne|
|Parkville, Victoria, Australia, 3052|
|The Children's Hospital at Westmead|
|Westmead, Australia, 2145|
|AKH Wien (Paediatrics)|
|McMaster Children's Hospital|
|Hamilton, Ontario, Canada, L8N3Z5|
|Fakultni nemocnice Brno|
|Brno, Czech Republic, 625 00|
|Fakultni nemocnice Ostrava|
|Ostrava, Czech Republic, 708 52|
|Fakultni nemocnice Motole|
|Praha 5, Czech Republic, 150 06|
|C.R.T.H. Hopital de Bicentre (Hemophilie)|
|Le Kremlin-Bicentre, France, 94275|
|Hospital Edouard Herriot|
|Lyon, France, 69437|
|Hôpital d'enfants La Timone|
|Marseille, France, 13385|
|CRC Coagulation Research Center GmbH|
|Duisburg/Altstadt, Germany, 47051|
|Düsseldorf, Germany, 40225|
|Sheba Medical Center|
|Tel Hashomer, Israel, 52621|
|Firenze, Italy, 50134|
|IRCCS Ospendale Maggiore (Centro emofilia e Trombosi)|
|Milano, Italy, 20122|
|FGU "Kirov Research Institute of Haemotology and Blood Trans)|
|Kirov, Russian Federation, 610027|
|H.U. La Paz|
|Madrid, Spain, 28046|
|Study Director:||Program Director||CSL Behring|