Hemophilia B

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

NCT02053792 | PHASE 3 | INTERVENTIONAL

This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B. The study will include subjects who have not previously been treated with Factor IX products, subjects who previously completed a CSL-sponsored rIX-FP lead-in study and subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study. A surgical prophylaxis substudy will examine the efficacy of rIX-FP in subjects with hemophilia B who are undergoing non-emergency major or minor surgery. An additional substudy will examine the safety and PK of subcutaneous (SC) administration of rIX-FP.

42 Sites

97 Participants

Recruiting

18 Years to 70 Years

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SUBMIT

University of Colorado

Aurora,Colorado,United States,80045

Indiana Hemophilia & Thrombosis Center Inc.

Indianapolis,Indiana,United States,46260

Hospital of the University of Pennsylvania

Philadelphia,Pennsylvania,United States,19104

University of Utah

Salt Lake City,Utah,United States,84103

Royal Children's Hospital

Parkville,Victoria,Australia,3052

The Children's Hospital Westmead

Westmead,Victoria,Australia,2145

Department of Pediatrics, Medical University of Vienna

Vienna,Austria,1090

Medical University of Vienna, Vienna General Hospital

Vienna,Austria,1090

SHAT "Joan Pavel" ODD Hemorrhagic Diathesis & Anemia

Sofia,Bulgaria,1233

McMaster University

Hamilton,Ontario,Canada,L8L2X2

Fakultni nemocnice Brno

Brno,Czechia,62500

Fakultni Nemocrice Ostrava

Ostrava-Poruba,Czechia,70852

Fakultni nemocnice v Motole

Praha,Czechia,15006

CHRU Hopital Morvan

Brest,France,29609

Hopital Louis Pradel

Bron Cedex,France,69677

Hopital Bicetre Centre de Traitement del'Hemophilia

Le Kremlin-Bicetre,France,94275

Hopital d'Enfants La Timonepital

Marseille Cedex,France,13385

Hopital NeckerEnfants Malades

Paris,France,75015

Institut fur experimentelle Hamatologie

Bonn,Germany,53127

Prof. Hess Kinderklinik

Bremen,Germany,28177

CRC Coagulation Research Centre GmbH

Duisburg,Germany,47051

Heinrich Heine University Dusseldorf

Dusseldorf,Germany,40225

Universtatsklinikum HamburgEppendorf

Hamburg,Germany,20246

WerlhofInstitute for Haemostasis and Thrombosis

Hannover,Germany,30159

Kurpfalzkrankenhaus Heidlerberg GmbH

Heidelberg,Germany

Chaim Sheba Medical Center

Tel Aviv,Israel

IRCCS Ospedale MaggioreCentro emofilia e Trobosi

Milano,Italy,20122

UOS Gestione e Organizzazione Funzlone Hub Emofilia

Parma,Italy,43126

Centro Malattie Emorragiche e Trombotiche Ospedale

Vicenza,Italy,36100

Nara Medical University Hospital

Kashihara,Japan,634-8522

University of Occupational and Environmental Health

Kitakyushu,Japan

Nagoya University Hospital

Nagoya,Japan,466-8550

The Hospital of Hyogo College of Medicine

Nishinomiya,Japan

Tokyo Medical University Hospital

Tokyo,Japan,160-0023

Ogikubo Hospital

Tokyo,Japan,167-0035

St. Marianna University, School of Medicine, Seibu Hospital

Yokohama,Japan,241-0811

National Blood Center

Kuala Lumpur,Malaysia,50400

Perpetual Succour Hospital

Cebu,Philippines,6000

Haemophilia Comprehensive Care Centre

Parktown,South Africa,2193

C.H.U. A Coruna

A Coruna,Spain

Hospital Vall Hebron

Barcelona,Spain,08035

H.U. La Paz

Madrid,Spain,28046

Study Eligibility Criteria

Inclusion Criteria

Main study inclusion criteria:
For previously treated subjects, either:
* Completed a CSL-sponsored rIX-FP (CSL654) study, including study CSL654_3001 \[NCT01496274\] or study CSL654_3002 \[NCT01662531\].
Or:
* Scheduled to have a major non-emergency surgery within approximately 8 weeks from the anticipated date of receiving the first rIX-FP injection.
* Not previously completed a CSL-sponsored rIX-FP lead-in study.
* Male, 12 to 70 years of age.
* Documented severe hemophilia B (FIX activity of ≤ 2%), or confirmed at screening by the central laboratory.
* Subjects who have received FIX products (plasma-derived and / or recombinant FIX) for \> 150 exposure days (EDs), confirmed by their treating physician.
* No confirmed history of FIX inhibitor formation at screening by the central laboratory
For previously untreated subjects:
* Male, up to 18 years of age.
* Documented severe hemophilia B (FIX activity of ≤ 2%), or confirmed at screening by the central laboratory.
* Never previously been treated with FIX clotting factor products (except previous exposure to blood components).
* No confirmed history of FIX inhibitor formation
Surgery substudy inclusion criterion:
* Must require non-emergency surgery
Subcutaneous substudy inclusion criteria:
* Male, at least 18 years of age.
* Subjects currently enrolled in Study CSL654_3003
* Subjects who have received rIX-FP for ≥ 100 EDs (single-dose cohorts) or for ≥ 50 EDs (repeated-dose cohort)

Exclusion Criteria

Main study exclusion criteria:
* Currently receiving a therapy not permitted during the study.
* Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.
For subjects who have previously completed a CSL-sponsored rIX-FP study:
* Unwilling to participate in the study for a total of 100 exposure days.
For subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study:
* Known hypersensitivity (ie, allergic reaction or anaphylaxis) to any FIX product or hamster protein.
* Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
* Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
* Low platelet count, kidney or liver disease.
* Human immunodeficiency virus positive with a CD4 count \< 200/mm3.
For previously untreated subjects:
* Known congenital or acquired coagulation disorder other than congenital FIX deficiency (except for vitamin K deficiency of the newborn).
* Known kidney or liver dysfunction or any condition which, in the investigator's opinion, place the patient at unjustifiable risk.
The surgical substudy does not have any additional exclusion criteria, although subject(s) in France will not be eligible for the surgery sub-study.
Subcutaneous substudy exclusion criteria:
* Intravenous use of rIX-FP within 14 days of subcutaneous administration of rIX-FP.
* Life-threatening bleeding episode or major surgery during the 3 months prior to substudy entry

Additional Studies

Additional studies can be found at ClinicalTrials.gov