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

Hemophilia B Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01662531
• Other study ID #: CSL654_3002
• Secondary ID: 2011-006032-23
• Status: Completed
• Phase: Phase 3
• First received: August 7, 2012
• Last updated: November 5, 2015
• Start date: September 2012

Study information

• Verified date: November 2015
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: Australia: Department of Health and Ageing Therapeutic Goods AdministrationAustria: Federal Office for Safety in Health CareCanada: Health CanadaCzech Republic: State Institute for Drug ControlFrance: Agence Nationale de Sécurité du Médicament et des produits de santéGermany: Paul-Ehrlich-InstitutItaly: The Italian Medicines AgencyRussia: Ministry of Health of the Russian FederationSpain: Agencia Española de Medicamentos y Productos Sanitarios
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. primary completion date: October 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 11 Years

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

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

Locations

Country	Name	City	State
Australia	The Henry Ekert Haemophilia Treatment Centre	Parkville	Victoria
Australia	The Children's Hospital at Westmead	Westmead
Austria	AKH Wien (Paediatrics)	Wien
Canada	McMaster University	Hamilton	Ontario
Czech Republic	Fakultni nemocnice Brno	Brno
Czech Republic	Fakultni nemocnice Ostrava	Ostrava-Poruba
Czech Republic	Fakultni nemocnice Motole	Praha 5
France	C.R.T.H. Hop Bicetre (Hemophilie)	Le Kremlin-Bicetre
France	Hospital Edouard Herriot	Lyon
France	Assistance Publique Hopitaux de Marseille (APHM) - Centre Ho	Marseille
Germany	Heinrich-Heine-Universitaet	Duesseldorf
Germany	CRC Coagulation Research Center GmbH	Duisburg/Altstadt
Israel	Sheba Medical Center	Tel Hashomer
Italy	AOU Careggi	Firenze
Italy	IRCCS Ospedale Maggiore (Centro emofilia e Trombosi)	Milano
Russian Federation	FGU "Kirov Research Institute of Haemotology and Blood Trans)	Kirov
Spain	H.U. La Paz	Madrid

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

Australia,  Austria,  Canada,  Czech Republic,  France,  Germany,  Israel,  Italy,  Russian Federation,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incremental recovery of rIX-FP		30 minutes	No
Primary	Half-life (t1/2) of a single dose of rIX-FP		Approximately 10 to 14 days	No
Primary	Area under the curve (AUC)	AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of rIX-FP	Approximately 10 to 14 days	No
Primary	Clearance of a single dose of rIX-FP		Approximately 10 to 14 days	No
Primary	Number of subjects developing Factor IX (FIX) inhibitors		Approximately 12 months	Yes
Secondary	The frequency of related adverse events		Approximately 12 months	Yes
Secondary	Number of subjects developing antibodies against rIX-FP		Approximately 12 months	Yes
Secondary	Proportion of bleeding episodes requiring one, two or more than two infusions of rIX-FP to achieve hemostasis		Approximately 12 months	No
Secondary	Consumption of rIX-FP	Recombinant IX-FP consumed expressed as: number of infusions per month and per year; IU/kg per month, per year and per event	Approximately 12 months	No