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Completed

NCT Number: 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

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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Key information

Conditions

Hemophilia B Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

Up to 11 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

The Royal Children's Hospital, Melbourne, Parkville, Victoria, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

rIX-FP

Biological

Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

Secondary outcomes

  1. Number of Subjects With Treatment-related Adverse Events

    Time frame: 12 months

  2. 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).

  3. 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

  4. 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.

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2014
First posted
Aug 10, 2012
Registry last updated
May 9, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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