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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 ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01662531
First received: August 7, 2012
Last updated: September 16, 2014
Last verified: September 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Locations
Australia, Victoria
The Henry Ekert Haemophilia Treatment Centre
Parkville, Victoria, Australia, 3052
Australia
The Children's Hospital at Westmead
Westmead, Australia, 2145
Austria
AKH Wien (Paediatrics)
Wien, Austria
Canada, Ontario
McMaster University
Hamilton, Ontario, Canada, L8L 2X2
Czech Republic
Fakultni nemocnice Brno
Brno, Czech Republic, 625 00
Fakultni nemocnice Ostrava
Ostrava-Poruba, Czech Republic, 708 52
Fakultni nemocnice Motole
Praha 5, Czech Republic, 150 06
France
C.R.T.H. Hop Bicetre (Hemophilie)
Le Kremlin-Bicetre, France, 94270
Assistance Publique Hopitaux de Marseille (APHM) - Centre Ho
Marseille, France, 13005
Hopital Necker Enfants Malades
Paris, France, 75015
Place du Docteur Baylac
Toulouse, France, 31000
Germany
CRC Coagulation Research Center GmbH
Duisburg/Altstadt, Germany, 47051
Unikinderklinik Frankfurt/Main
Frankfurt/Main, Germany, 60596
Universitatsklinikum Hamburg-Eppendorf (Padiatr Hamat)
Hamburg, Germany, 20246
Israel
Sheba Medical Center
Tel Hashomer, Israel, 52621
Italy
AOU Careggi
Firenze, Italy, 50134
IRCCS Ospedale Maggiore (Centro emofilia e Trombosi)
Milano, Italy, 20122
Ospedale S. Bortolo, AULSS n.6 Vicenza
Vicenza, Italy, 36100
Russian Federation
FGU "Kirov Research Institute of Haemotology and Blood Trans)
Kirov, Russian Federation, 610027
Spain
C.H.U. A Coruna
A Coruna, Spain, 15006
H.U. La Paz
Madrid, Spain, 28046
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: September 16, 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
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on November 23, 2014