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

Hemophilia B Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01496274
• Other study ID #: CSL654_3001
• Secondary ID: 2011-002415-28
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: December 19, 2011
• Last updated: September 29, 2015
• Start date: February 2012

Study information

• Verified date: October 2014
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: Austria: Federal Office for Safety in Health CareBulgaria: Bulgarian Drug AgencyFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: Paul-Ehrlich-InstitutIsrael: Ministry of HealthItaly: Ministry of HealthJapan: Pharmaceuticals and Medical Devices AgencyRussia: Ministry of Health of the Russian FederationSpain: Spanish Agency of MedicinesUnited States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 63
• Est. primary completion date: July 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 65 Years

Eligibility

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

• On-demand subjects only, who have experienced a minimum average of 2 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months

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, kidney or liver dysfunction

• Recent life-threatening bleeding episode

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Biological:
• rIX-FP
Recombinant IX-FP (rIX-FP) is a fusion protein linking coagulation factor IX with albumin, and will be administered by intravenous administration

Locations

Country	Name	City	State
Austria	AKH Wien [Hämatologie, Hämostaseol	Wien
Bulgaria	SHAT "Joan Pavel" OOD [Hemorrhagic Diathesis and Anemia]	Sophia
France	Centre Hospitalier Universitaire de Brest/CHU Morvan	Brest
France	C.R.T.H. Hôp. Bicêtre-Hémophilie	Le Kremlin-Bicêtre
France	CHU de Lyon - Hôpital Edouard Herriot [Hemophilie]	Lyon
France	Hôpital Necker-CRTH	Paris
Germany	Instit. für Experimentelle - Hämato & Transfusionsmedizin	Bonn
Germany	Zentralkrankenhaus Prof. Hess-Kinderklinik	Bremen
Germany	Unikinderklinik Frankfurt/Main [Kinderheilkunde]	Frankfurt
Germany	Universitätsklinikum Hamburg-Eppendorf, Abt für Pädiatr. Hämatologie	Hamburg
Germany	Werlhof-Inst. Hannover	Hannover
Israel	Chaim Sheba Medical Center	Tel Aviv
Italy	IRCCS Ospedale Maggiore[Centro emofilia e Trombosi]	Milano
Italy	A.O.U. di Parma [Centro di Rif. Reg. per la cura dell'Emofil	Parma
Italy	Osp. S.Bortolo ULSS N.6 [Terapie Cell. ed Ematologia]	Vicenza
Japan	Nara Medical University Hospital [PEDIATRICS]	Kashihara
Japan	University of Occupational and Environmental Health	Kitakyushu
Japan	Nagoya University Hospital	Nagoya
Japan	The Hospital of Hyogo College of Medicine	Nishinomiya
Japan	Ogikubo Hospital	Tokyo
Japan	Tokyo Medical University Hospital	Tokyo
Japan	St. Marianna University, School of Medicine, Yokohama Seibu	Yokohama
Russian Federation	FGU "Kirov Research Institute of Haemotology and Blood Trans	Kirov
Spain	C.H.U. A Coruña [Hematología]	A Coruna
Spain	H.U.Vall d'Hebrón [Hemofillia]	Barcelona
Spain	H.U. La Paz [Coagulopatias Congénitas]	Madrid
United States	Rush University Medical Center	Chicago	Illinois
United States	Indiana Hemophilia and Thrombosis Center, Inc.	Indianapolis	Indiana
United States	BloodCenter of Wisconsin	Milwaukee	Wisconsin
United States	Hospital of the University of Pennsylvania	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

United States,  Austria,  Bulgaria,  France,  Germany,  Israel,  Italy,  Japan,  Russian Federation,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in frequency of spontaneous bleeding events between on-demand and prophylaxis treatments (annualized)		Approximately 14 months	No
Primary	Number of subjects developing inhibitors against factor IX (FIX)		Approximately 14 months	Yes
Secondary	The frequency of related Adverse Events		Approximately 14 months	Yes
Secondary	Number of subjects developing antibodies against rIX-FP		Approximately 14 months	Yes
Secondary	Proportion of bleeding episodes requiring one or = two infusions of rIX-FP to achieve hemostasis		Approximately 14 months	No
Secondary	Investigator's overall clinical assessment of hemostatic efficacy for treatment of bleeding episodes, based on a four point ordinal scales (excellent, good, moderate, poor/ none)		Approximately 14 months	No
Secondary	rIX-FP consumed per month while maintaining assigned prophylactic treatment interval during routine prophylaxis.		Approximately 14 months	No
Secondary	Incremental recovery of rIX-FP		30 minutes	No
Secondary	Half-life (t1/2) of a single dose of rIX-FP		240 hours	No
Secondary	Area under the curve (AUC)	AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of rIX-FP	240 hours	No
Secondary	Clearance of a single dose of rIX-FP		240 hours	No
Secondary	Investigator's (or surgeon's) overall clinical assessment of hemostatic efficacy for surgical prophylaxis, based on a four point ordinal scale (excellent, good, moderate, poor/ none)		Approximately 14 days	No
Secondary	Annualized spontaneous bleeding events during the 7-day prophylactic regimen compared to those during prophylactic regimens longer than 7-days.		Approximately 14 months	No