A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01662531
First received: August 7, 2012
Last updated: April 3, 2016
Last verified: April 2016

August 7, 2012
April 3, 2016
January 2013
October 2014   (final data collection date for primary outcome measure)
  • Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product [ Time Frame: 30 minutes after infusion ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]

    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
    Inhibitor formation was defined as any inhibitor (≥0.6 BU [Bethesda Units]/mL) identified and confirmed by retesting.
  • Incremental recovery of rIX-FP [ Time Frame: 30 minutes ] [ Designated as safety issue: No ]
  • Half-life (t1/2) of a single dose of rIX-FP [ Time Frame: Approximately 10 to 14 days ] [ Designated as safety issue: No ]
  • Area under the curve (AUC) [ Time Frame: Approximately 10 to 14 days ] [ Designated as safety issue: No ]
    AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of rIX-FP
  • Clearance of a single dose of rIX-FP [ Time Frame: Approximately 10 to 14 days ] [ Designated as safety issue: No ]
  • Number of subjects developing Factor IX (FIX) inhibitors [ Time Frame: Approximately 12 months ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT01662531 on ClinicalTrials.gov Archive Site
  • Number of Subjects With Treatment-related Adverse Events [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Number of Subjects Developing Antibodies Against rIX-FP [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Consumption of rIX-FP during routine prophylaxis is expressed as the total prophylaxis dose per month.
  • The frequency of related adverse events [ Time Frame: Approximately 12 months ] [ Designated as safety issue: Yes ]
  • Number of Subjects Developing Antibodies Against rIX-FP [ Time Frame: Approximately 12 months ] [ Designated as safety issue: Yes ]
  • Proportion of bleeding episodes requiring one, two or more than two infusions of rIX-FP to achieve hemostasis [ Time Frame: Approximately 12 months ] [ Designated as safety issue: No ]
  • Consumption of rIX-FP [ Time Frame: Approximately 12 months ] [ Designated as safety issue: No ]

    Recombinant IX-FP consumed expressed as:

    • number of infusions per month and per year
    • IU/kg per month, per year and per event
Not Provided
Not Provided
 
A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
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
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.
Not Provided
Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hemophilia B
Biological: rIX-FP
Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (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.
Intervention: Biological: rIX-FP
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
27
Not Provided
October 2014   (final data collection date for primary outcome measure)

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.
Male
up to 11 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Austria,   Canada,   Czech Republic,   France,   Germany,   Israel,   Italy,   Russian Federation,   Spain
 
NCT01662531
CSL654_3002, 2011-006032-23
Not Provided
Not Provided
Not Provided
CSL Behring
CSL Behring
Not Provided
Study Director: Program Director CSL Behring
CSL Behring
April 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP