A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
- Conditions
- Hemophilia B
- Interventions
- Biological: rIX-FP
- Registration Number
- NCT02053792
- Lead Sponsor
- CSL Behring
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 97
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description rIX-FP rIX-FP Subjects will administer rIX-FP by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 3 years. The routine prophylaxis treatment interval for previously treated patients may be changed at each scheduled 6-month follow-up assessment. On-demand treatment with rIX-FP will be used for all bleeding episodes requiring treatment. Subjects (other than those in France) may participate in a surgical 'substudy' in which rIX-FP may be administered before, during and after surgery. An additional substudy will examine the safety and PK of subcutaneous administration of rIX-FP. For previously untreated patients, subjects will administer rIX-FP intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
- Primary Outcome Measures
Name Time Method Mean Incremental Recovery of a 50 IU/kg Dose of CSL654 in Previously Untreated Patients (PUPs) Approximately 30 minutes after infusion of CSL654 Incremental Recovery: The increase in plasma concentration per IU/kg of factor administered.
Total Number of Participants Who Developed Inhibitors Against Factor IX (FIX) For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.
- Secondary Outcome Measures
Name Time Method Total ABR for On-demand Regimen vs. 14-Day Regimen in PTPs For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). Spontaneous ABR for On-demand Regimen vs. 14-Day Regimen in PTPs For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). Total Annualized Bleeding Rate (ABR) by Prophylaxis Regimen in Previously Treated Patients (PTPs) For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). Spontaneous ABR by Prophylaxis Regimen in PTPs For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). Number of Participants With Investigator's Overall Clinical Assessment of Hemostatic Efficacy for the Treatment of Major Bleeding Events With CSL654 in PUPs Up to 3 years or the time it takes to achieve 50 EDs The investigator will rate the efficacy of the rIX-FP treatment based on a hemostatic efficacy four point rating scale of excellent, good, moderate, or poor/no response.
Total ABR for Subjects >=12 Years: 7-Day Regimen vs. 14-Day Regimen in PTPs For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). Average Amount of CSL654 (rIX-FP) Consumed Per Month Per Subject During Routine Prophylaxis Treatment. For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs. Spontaneous ABR for Subjects >=12 Years: 7-Day Regimen vs. (10 or 14)-Day Regimen in PTPs For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). Percentage of Participants With at Least One Treatment Emergent Adverse Event (TEAE) and the Percentage of Participants With at Least One CSL654-related TEAE For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs. Spontaneous ABR for Subjects >=12 Years: 7-Day Regimen vs. 14-Day Regimen in PTPs For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). Total ABR for Subjects >=12 Years: 7-Day Regimen vs. (10 or 14)-Day Regimen in PTPs For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Trial Locations
- Locations (42)
University of Colorado
🇺🇸Aurora, Colorado, United States
Royal Children's Hospital
🇦🇺Parkville, Victoria, Australia
The Hospital of Hyogo College of Medicine
🇯🇵Nishinomiya, Japan
Tokyo Medical University Hospital
🇯🇵Tokyo, Japan
Ogikubo Hospital
🇯🇵Tokyo, Japan
National Blood Center
🇲🇾Kuala Lumpur, Malaysia
Department of Pediatrics, Medical University of Vienna
🇦🇹Vienna, Austria
Hospital Vall Hebron
🇪🇸Barcelona, Spain
The Children's Hospital Westmead
🇦🇺Westmead, Victoria, Australia
University of Utah
🇺🇸Salt Lake City, Utah, United States
Indiana Hemophilia & Thrombosis Center Inc.
🇺🇸Indianapolis, Indiana, United States
Hospital of the University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Medical University of Vienna, Vienna General Hospital
🇦🇹Vienna, Austria
SHAT "Joan Pavel" ODD [Hemorrhagic Diathesis & Anemia]
🇧🇬Sofia, Bulgaria
Fakultni nemocnice Brno
🇨🇿Brno, Czechia
McMaster University
🇨🇦Hamilton, Ontario, Canada
Fakultni Nemocrice Ostrava
🇨🇿Ostrava-Poruba, Czechia
CHRU Hopital Morvan
🇫🇷Brest, France
Hopital Louis Pradel
🇫🇷Bron Cedex, France
Hopital Bicetre - Centre de Traitement del'Hemophilia
🇫🇷Le Kremlin-Bicetre, France
Fakultni nemocnice v Motole
🇨🇿Praha, Czechia
Prof. Hess Kinderklinik
🇩🇪Bremen, Germany
Hopital Necker-Enfants Malades
🇫🇷Paris, France
Hopital d'Enfants La Timonepital
🇫🇷Marseille Cedex, France
CRC Coagulation Research Centre GmbH
🇩🇪Duisburg, Germany
Institut fur experimentelle Hamatologie
🇩🇪Bonn, Germany
Universtatsklinikum Hamburg-Eppendorf
🇩🇪Hamburg, Germany
Heinrich Heine University Dusseldorf
🇩🇪Dusseldorf, Germany
Chaim Sheba Medical Center
🇮🇱Tel Aviv, Israel
Werlhof-Institute for Haemostasis and Thrombosis
🇩🇪Hannover, Germany
UOS Gestione e Organizzazione Funzlone Hub Emofilia
🇮🇹Parma, Italy
Kurpfalzkrankenhaus Heidlerberg GmbH
🇩🇪Heidelberg, Germany
IRCCS Ospedale Maggiore[Centro emofilia e Trobosi]
🇮🇹Milano, Italy
Nara Medical University Hospital
🇯🇵Kashihara, Japan
Centro Malattie Emorragiche e Trombotiche Ospedale
🇮🇹Vicenza, Italy
University of Occupational and Environmental Health
🇯🇵Kitakyushu, Japan
Nagoya University Hospital
🇯🇵Nagoya, Japan
Haemophilia Comprehensive Care Centre
🇿🇦Parktown, South Africa
St. Marianna University, School of Medicine, Seibu Hospital
🇯🇵Yokohama, Japan
Perpetual Succour Hospital
🇵🇭Cebu, Philippines
C.H.U. A Coruna
🇪🇸A Coruna, Spain
H.U. La Paz
🇪🇸Madrid, Spain