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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01662531
Registration number
NCT01662531
Ethics application status
Date submitted
7/08/2012
Date registered
10/08/2012
Date last updated
9/05/2016
Titles & IDs
Public title
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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Scientific 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
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Secondary ID [1]
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2011-006032-23
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Secondary ID [2]
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CSL654_3002
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia B
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - rIX-FP
Experimental: rIX-FP - 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.
Other interventions: rIX-FP
Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product
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Assessment method [1]
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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.
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Timepoint [1]
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30 minutes after infusion
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Primary outcome [2]
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Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product
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Assessment method [2]
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FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values.
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Timepoint [2]
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Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
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Primary outcome [3]
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Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast)
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Assessment method [3]
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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.
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Timepoint [3]
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Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
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Primary outcome [4]
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Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product
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Assessment method [4]
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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.
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Timepoint [4]
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Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
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Primary outcome [5]
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Number of Subjects Developing Inhibitors to Factor IX (FIX)
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Assessment method [5]
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Inhibitor formation was defined as any inhibitor (=0.6 BU [Bethesda Units]/mL) identified and confirmed by retesting.
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Timepoint [5]
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12 months
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Secondary outcome [1]
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Number of Subjects With Treatment-related Adverse Events
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Assessment method [1]
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Timepoint [1]
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12 months
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Secondary outcome [2]
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Number of Subjects Developing Antibodies Against rIX-FP
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Assessment method [2]
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Antibodies to rIX-FP were measured using a direct-binding enzyme-linked immunosorbent assay (ELISA).
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Timepoint [2]
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12 months
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Secondary outcome [3]
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Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis
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Assessment method [3]
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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
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Timepoint [3]
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Approximately 12 months
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Secondary outcome [4]
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Consumption of rIX-FP During Routine Prophylaxis
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Assessment method [4]
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Consumption of rIX-FP during routine prophylaxis is expressed as the total prophylaxis dose per month.
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Timepoint [4]
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12 months
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Eligibility
Key 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.
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Minimum age
No limit
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Maximum age
11
Years
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Sex
Males
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/01/2013
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
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Sample size
Target
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Accrual to date
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Final
27
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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The Royal Children's Hospital, Melbourne - Parkville
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Recruitment hospital [2]
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The Children's Hospital at Westmead - Westmead
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment outside Australia
Country [1]
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Austria
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State/province [1]
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Wien
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Canada
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State/province [2]
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Ontario
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Country [3]
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Czech Republic
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State/province [3]
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Brno
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Country [4]
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Czech Republic
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State/province [4]
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Ostrava
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Country [5]
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Czech Republic
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State/province [5]
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Praha 5
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Country [6]
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France
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State/province [6]
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Le Kremlin-Bicentre
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Country [7]
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France
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Lyon
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France
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Marseille
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Germany
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Duisburg/Altstadt
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Germany
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State/province [10]
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Düsseldorf
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Israel
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State/province [11]
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Tel Hashomer
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Italy
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State/province [12]
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Firenze
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Country [13]
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Italy
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State/province [13]
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Milano
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Country [14]
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Russian Federation
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State/province [14]
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Kirov
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Country [15]
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Spain
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State/province [15]
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Madrid
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
CSL Behring
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01662531
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Program Director
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Address
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CSL Behring
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01662531
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