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 is currently recruiting participants.
Verified April 2014 by CSL Behring
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01662531
First received: August 7, 2012
Last updated: April 7, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention Phase
Hemophilia B
Biological: rIX-FP
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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


Estimated Enrollment: 24
Study Start Date: September 2012
Estimated Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Biological: rIX-FP

  Eligibility

Ages Eligible for Study:   up to 11 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01662531

Contacts
Contact: Clinical Trials Registration Coordinator clinicaltrials@cslbehring.com

Locations
Australia, Victoria
The Henry Ekert Haemophilia Treatment Centre Recruiting
Parkville, Victoria, Australia, 3052
Contact: Use Central Contact         
Australia
The Children's Hospital at Westmead Not yet recruiting
Westmead, Australia, 2145
Contact: Use Central Contact         
Austria
AKH Wien (Paediatrics) Recruiting
Wien, Austria
Contact: Use Central Contact         
Canada, Ontario
McMaster University Recruiting
Hamilton, Ontario, Canada, L8L 2X2
Contact: Use Central Contact         
Czech Republic
Fakultni nemocnice Brno Recruiting
Brno, Czech Republic, 625 00
Contact: Use Central Contact         
Fakultni nemocnice Ostrava Recruiting
Ostrava-Poruba, Czech Republic, 708 52
Contact: Use Central Contact         
Fakultni nemocnice Motole Recruiting
Praha 5, Czech Republic, 150 06
Contact: Use Central Contact         
France
C.R.T.H. Hop Bicetre (Hemophilie) Recruiting
Le Kremlin-Bicetre, France, 94270
Contact: Use Central Contact         
Assistance Publique Hopitaux de Marseille (APHM) - Centre Ho Recruiting
Marseille, France, 13005
Contact: Use Central Contact         
Hopital Necker Enfants Malades Not yet recruiting
Paris, France, 75015
Contact: Use Central Contact         
Place du Docteur Baylac Recruiting
Toulouse, France, 31000
Contact: Use Central Contact         
Germany
CRC Coagulation Research Center GmbH Recruiting
Duisburg/Altstadt, Germany, 47051
Contact: Use Central Contact         
Unikinderklinik Frankfurt/Main Recruiting
Frankfurt/Main, Germany, 60596
Contact: Use Central Contact         
Universitatsklinikum Hamburg-Eppendorf (Padiatr Hamat) Recruiting
Hamburg, Germany, 20246
Contact: Use Central Contact         
Israel
Sheba Medical Center Recruiting
Tel Hashomer, Israel, 52621
Contact: Use Central Contact         
Italy
AOU Careggi Recruiting
Firenze, Italy, 50134
Contact: Use Central Contact         
IRCCS Ospedale Maggiore (Centro emofilia e Trombosi) Recruiting
Milano, Italy, 20122
Contact: Use Central Contact         
Ospedale S. Bortolo, AULSS n.6 Vicenza Recruiting
Vicenza, Italy, 36100
Contact: Use Central Contact         
Russian Federation
FGU "Kirov Research Institute of Haemotology and Blood Trans) Recruiting
Kirov, Russian Federation, 610027
Contact: Use Central Contact         
Spain
C.H.U. A Coruna Not yet recruiting
A Coruna, Spain, 15006
Contact: Use Central Contact         
H.U. La Paz Recruiting
Madrid, Spain, 28046
Contact: Use Central Contact         
Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Program Director CSL Behring
  More Information

No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01662531     History of Changes
Other Study ID Numbers: CSL654_3002, 2011-006032-23
Study First Received: August 7, 2012
Last Updated: April 7, 2014
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Austria: Federal Office for Safety in Health Care
Canada: Health Canada
Czech Republic: State Institute for Drug Control
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Germany: Paul-Ehrlich-Institut
Italy: The Italian Medicines Agency
Russia: Ministry of Health of the Russian Federation
Spain: Agencia Española de Medicamentos y Productos Sanitarios

Additional relevant MeSH terms:
Hemophilia B
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on April 22, 2014