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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/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
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
Secondary ID [1] 0 0
2011-006032-23
Secondary ID [2] 0 0
CSL654_3002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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)

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product - 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.
Timepoint [1] 0 0
30 minutes after infusion
Primary outcome [2] 0 0
Half-life (t1/2) 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.
Timepoint [2] 0 0
Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
Primary outcome [3] 0 0
Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast) - 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.
Timepoint [3] 0 0
Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
Primary outcome [4] 0 0
Clearance for FIX Activity 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 is normalized for body weight.
Timepoint [4] 0 0
Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose
Primary outcome [5] 0 0
Number of Subjects Developing Inhibitors to Factor IX (FIX) - Inhibitor formation was defined as any inhibitor (=0.6 BU [Bethesda Units]/mL) identified and confirmed by retesting.
Timepoint [5] 0 0
12 months
Secondary outcome [1] 0 0
Number of Subjects With Treatment-related Adverse Events
Timepoint [1] 0 0
12 months
Secondary outcome [2] 0 0
Number of Subjects Developing Antibodies Against rIX-FP - Antibodies to rIX-FP were measured using a direct-binding enzyme-linked immunosorbent assay (ELISA).
Timepoint [2] 0 0
12 months
Secondary outcome [3] 0 0
Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis - 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
Timepoint [3] 0 0
Approximately 12 months
Secondary outcome [4] 0 0
Consumption of rIX-FP During Routine Prophylaxis - Consumption of rIX-FP during routine prophylaxis is expressed as the total prophylaxis dose per month.
Timepoint [4] 0 0
12 months

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.
Minimum age
No limit
Maximum age
11 Years
Gender
Males
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Royal Children's Hospital, Melbourne - Parkville
Recruitment hospital [2] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
Austria
State/province [1] 0 0
Wien
Country [2] 0 0
Canada
State/province [2] 0 0
Ontario
Country [3] 0 0
Czech Republic
State/province [3] 0 0
Brno
Country [4] 0 0
Czech Republic
State/province [4] 0 0
Ostrava
Country [5] 0 0
Czech Republic
State/province [5] 0 0
Praha 5
Country [6] 0 0
France
State/province [6] 0 0
Le Kremlin-Bicentre
Country [7] 0 0
France
State/province [7] 0 0
Lyon
Country [8] 0 0
France
State/province [8] 0 0
Marseille
Country [9] 0 0
Germany
State/province [9] 0 0
Duisburg/Altstadt
Country [10] 0 0
Germany
State/province [10] 0 0
Düsseldorf
Country [11] 0 0
Israel
State/province [11] 0 0
Tel Hashomer
Country [12] 0 0
Italy
State/province [12] 0 0
Firenze
Country [13] 0 0
Italy
State/province [13] 0 0
Milano
Country [14] 0 0
Russian Federation
State/province [14] 0 0
Kirov
Country [15] 0 0
Spain
State/province [15] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
CSL Behring
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/show/NCT01662531
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Program Director
Address 0 0
CSL Behring
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications