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

Completed · Phase 3

Conditions studied: Hemophilia B

In brief

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.

Key facts

Study ID: NCT02053792
Run by: CSL Behring
People needed: 97
Starts: 2014-02-06
Expected to finish: 2021-06-02
Last updated by the study team: 2022-07-14

Who can join

Age: any, up to 70. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

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)

You may not qualify if…

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

Where it is running

University of Colorado — Aurora, Colorado, United States
Indiana Hemophilia & Thrombosis Center Inc. — Indianapolis, Indiana, United States
Hospital of the University of Pennsylvania — Philadelphia, Pennsylvania, United States
University of Utah — Salt Lake City, Utah, United States
Royal Children's Hospital — Parkville, Victoria, Australia
The Children's Hospital Westmead — Westmead, Victoria, Australia
Department of Pediatrics, Medical University of Vienna — Vienna, Austria
Medical University of Vienna, Vienna General Hospital — Vienna, Austria
SHAT "Joan Pavel" ODD [Hemorrhagic Diathesis & Anemia] — Sofia, Bulgaria
McMaster University — Hamilton, Ontario, Canada
Fakultni nemocnice Brno — Brno, Czechia
Fakultni Nemocrice Ostrava — Ostrava-Poruba, Czechia
Fakultni nemocnice v Motole — Prague, Czechia
CHRU Hopital Morvan — Brest, France
Hopital Louis Pradel — Bron, France
Hopital Bicetre - Centre de Traitement del'Hemophilia — Le Kremlin-Bicêtre, France
Hopital d'Enfants La Timonepital — Marseille, France
Hopital Necker-Enfants Malades — Paris, France
Institut fur experimentelle Hamatologie — Bonn, Germany
Prof. Hess Kinderklinik — Bremen, Germany
CRC Coagulation Research Centre GmbH — Duisburg, Germany
Heinrich Heine University Dusseldorf — Düsseldorf, Germany
Universtatsklinikum Hamburg-Eppendorf — Hamburg, Germany
Werlhof-Institute for Haemostasis and Thrombosis — Hanover, Germany
Kurpfalzkrankenhaus Heidlerberg GmbH — Heidelberg, Germany

Full record on ClinicalTrials.gov

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