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

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:

Genetics Home Reference related topics: hemophilia

Drug Information available for: Factor IX

U.S. FDA Resources

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	Not yet 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	Not yet 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:	May 2, 2013
Health Authority:	Germany: Paul-Ehrlich-Institut Austria: Federal Office for Safety in Health Care Canada: Health Canada 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 May 21, 2013