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Completed

NCT Number: NCT01361126

A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B

This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B. The study consists of a screening period, a pharmacokinetic (PK) period, followed by approximately a 5 month treatment period. Subjects will receive weekly routine prophylactic therapy and on-demand treatment for bleeding episodes. In addition, subjects who are not on routine factor replacement therapy prior to the study will receive only on-demand treatment for bleeding episodes.

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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

12 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Study Site, Sofia, Bulgaria

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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, 12 to 65 years old
  • Severe hemophilia B (FIX activity of ≤ 2%)
  • Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs)
  • 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 obtained before undergoing any study specific procedures

Exclusion criteria

  • Known hypersensitivity to any FIX product or hamster protein
  • Known congenital or acquired coagulation disorder other than congenital FIX deficiency
  • HIV positive subjects with a CD4 count < 200/mm3
  • Low platelet count, abnormal kidney function, or liver disease
  • On-demand subjects experiencing less than 12 or 6 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months, respectively
  • Planned major surgical intervention during the study period

Treatment and study plan

Recombinant Coagulation Factor IX Albumin Fusion Protein

Biological

Study subjects will receive a single dose of 25IU/kg of rIX_FP for pharmacokinetic analysis. Subjects will then be treated for approximately 5 months. The treatment dose will be based on the subject's PK profile and the subject's bleeding phenotype.

Other names: CSL654

Primary outcomes

  1. Number of Subjects With Treatment-related Adverse Events

    Time frame: Approximately 20 weeks

    The causal relationship of each adverse event to rIX-FP was assessed by the Investigator.

  2. Number of Subjects With Inhibitors Against Factor IX (FIX)

    Time frame: Baseline, Day 10 and Weeks 4, 12 and 20

    The presence of inhibitors against FIX was assessed by the central laboratory by a FIX potency assay. To quantify anti-FIX neutralizing antibodies, the Bethesda assay with the Nijmegen modification was used, and the results expressed as Bethesda Units per mL (BU/mL). A positive inhibitor test is >=0.6 BU/mL.

  3. Number of Subjects Who Developed Antibodies to rIX-FP

    Time frame: Pre-dose, Day 10 and Weeks 4, 12, and 20

    Antibodies against rIX-FP were detected using a direct binding enzyme-linked immunosorbent assay (ELISA).

Secondary outcomes

  1. Area Under the Curve to the Last Sample With Quantifiable Drug Concentration (AUC0-t) After a Single Dose of rIX-FP

    Time frame: Pre-dose and up to 14 days after rIX-FP infusion.

    The plasma concentrations of rIX-FP were measured as FIX activity using a validated, 1-stage assay in a central laboratory for a quantification range from 0.25 to 150% (or 0.25 IU/dL to 150 IU/dL). The PK population comprised all subjects who received at least 1 dose of rIX-FP and for whom a sufficient number of analyzable PK samples had been obtained in order to permit the evaluation of the PK profile of rIX-FP, and who did not receive a dose of rIX-FP or any other FIX product for the treatment of a bleed during the PK sampling period.

  2. Half-life (t1/2) of a Single Dose of rIX-FP

    Time frame: Pre-dose and up to 14 days after infusion

  3. Incremental Recovery of rIX-FP at 30 Minutes Following Infusion of rIX-FP

    Time frame: 30 minutes after infusion

    Incremental recovery (IU/mL/IU/kg) is defined as FIX activity (IU/mL) 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.

  4. Clearance of a Single Dose of rIX-FP

    Time frame: Pre-dose and up to 14 days after rIX-FP infusion

  5. Breakthrough Bleeding Events

    Time frame: Week 9 to approximately Week 20

    Number of breakthrough bleeding events (spontaneous bleeding events) requiring treatment per subject in subjects receiving prophylactic treatment regimen with rIX-FP

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

A Phase I/II Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B

Important dates

Study start
2011
Primary completion
2012
Study completion
2012
First posted
May 26, 2011
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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