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

NCT ID: NCT01496274

Last Updated: 2016-05-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 63 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-02-29

Brief 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.

Conditions

Hemophilia B

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Prophylaxis

Routine weekly prophylaxis and episodic treatment for bleeding episodes. An individualized dosing interval may be tested in sub-group subjects during the 2nd part of the trial.

Subjects may participate in a surgical 'sub-study' in which rIX-FP may be administered prior to, during and after surgical intervention.

Group Type: EXPERIMENTAL

rIX-FP

Intervention Type: BIOLOGICAL

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

On-demand

Episodic treatment for bleeding episodes during the first 6 months then switch to routine weekly prophylaxis for a further 6 months Subjects may participate in a surgical 'sub-study' in which rIX-FP may be administered prior to, during and after surgical intervention.

Group Type: EXPERIMENTAL

rIX-FP

Intervention Type: BIOLOGICAL

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

Interventions

rIX-FP

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

Intervention Type: BIOLOGICAL

Eligibility Criteria

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

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Program Director

Role: STUDY_DIRECTOR

CSL Behring

Locations

Rush University Medical Center

Chicago, Illinois, United States

Indiana Hemophilia and Thrombosis Center, Inc.

Indianapolis, Indiana, United States

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

BloodCenter of Wisconsin

Milwaukee, Wisconsin, United States

AKH Wien [Hämatologie, Hämostaseol

Vienna, , Austria

SHAT "Joan Pavel" OOD [Hemorrhagic Diathesis and Anemia]

Sofia, , Bulgaria

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, , France

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

Hanover, , Germany

Chaim Sheba Medical Center

Tel Aviv, , Israel

IRCCS Ospedale Maggiore[Centro emofilia e Trombosi]

Milan, , 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, , Italy

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, , Japan

FGU "Kirov Research Institute of Haemotology and Blood Trans

Kirov, , Russia

C.H.U. A Coruña [Hematología]

A Coruña, , Spain

H.U.Vall d'Hebrón [Hemofillia]

Barcelona, , Spain

H.U. La Paz [Coagulopatias Congénitas]

Madrid, , Spain

Countries

United States; Austria; Bulgaria; France; Germany; Israel; Italy; Japan; Russia; Spain

References

Santagostino E, Martinowitz U, Lissitchkov T, Pan-Petesch B, Hanabusa H, Oldenburg J, Boggio L, Negrier C, Pabinger I, von Depka Prondzinski M, Altisent C, Castaman G, Yamamoto K, Alvarez-Roman MT, Voigt C, Blackman N, Jacobs I; PROLONG-9FP Investigators Study Group. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood. 2016 Apr 7;127(14):1761-9. doi: 10.1182/blood-2015-09-669234. Epub 2016 Jan 11.

Reference Type: DERIVED

PMID: 26755710 (View on PubMed)

Other Identifiers

2011-002415-28

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSL654_3001

Identifier Type: -

Identifier Source: org_study_id