Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
- Conditions
- Severe Hemophilia A
- Interventions
- Biological: Human cl rhFVIII
- Registration Number
- NCT01712438
- Lead Sponsor
- Octapharma
- Brief Summary
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 110
- Male patients
- Severe Hemophilia A (FVIII:C <1%)
- No previous treatment with FVIII concentrates or other blood products containing FVIII
- Diagnosis with a coagulation disorder other than Hemophilia A
- Severe liver or kidney disease
- Concomitant treatment with any systemic immunosuppressive drug
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Human cl rhFVIII Human cl rhFVIII -
- Primary Outcome Measures
Name Time Method Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors maximum 5 years (100 exposure days) The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development using the modified Bethesda assay (Nijmegen modification). The definitions for thresholds were ≥0.6 to \<5 BU/mL for a "low titre" inhibitor and ≥5 BU/mL for a "high-titre" inhibitor.
- Secondary Outcome Measures
Name Time Method Efficacy of Human-cl rhFVIII for Surgical Prophylaxis Maximum 5 years (100 exposure days) An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none).
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds Maximum 5 years (100 exposure days) A personal efficacy assessment to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes. Efficacy was assessed using a four-point scale (excellent, good, moderate, none).
Frequency of Spontaneous Break-through Bleeds Maximum 5 years (100 exposure days) The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII
Trial Locations
- Locations (37)
Republican Scientific Practical Center for Pediatric Oncology and Hematology
🇧🇾Minsk, Belarus
Hopital Ste-Justine
🇨🇦Montreal, Quebec, Canada
Kasturba Medical College, Dr. TMA Pai Hospital
🇮🇳Manipala, Karnataka, India
University of Alberta
🇨🇦Edmonton, Alberta, Canada
Sahyadri Speciality Hospital, Haematology & BMT Unit
🇮🇳Pune, India
Morozovsky Children's Hospital
🇷🇺Moscow, Russian Federation
Christian Medical College & Hospital, Dept of Haematology
🇮🇳Vellore, India
All Children's Hospital
🇺🇸Saint Petersburg, Florida, United States
UC Davis
🇺🇸Sacramento, California, United States
Harvard Children's Hospital Boston
🇺🇸Boston, Massachusetts, United States
BC Children's Hospital
🇨🇦Vancouver, British Columbia, Canada
Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Mc Master Children's Hospital
🇨🇦Hamilton, Ontario, Canada
L'hôpital Côte de Nacre - CHU de Caen
🇫🇷Caen, France
Centre de traitement de l'hemophilie, Hôpital Bicêtre
🇫🇷Le Kremlin-Bicêtre, France
Hopital de la Timone
🇫🇷Marseille, France
Hopital Trousseau - CHU Tours
🇫🇷Tours, France
Hôpital Necker
🇫🇷Paris, France
CHU de Rennes - Hôpital Pontchaillou
🇫🇷Rennes, France
University Hospital Frankfurt/M
🇩🇪Frankfurt, Germany
Institute of Haematology and Transfusiology
🇬🇪Tbilisi, Georgia
Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie
🇫🇷Nantes, France
Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT)
🇩🇪Bonn, Germany
Universitätsmedizin der Johannes-Gutenberg-Universität Mainz
🇩🇪Mainz, Germany
Univ. Di Perugia
🇮🇹Perugia, Italy
Scientific Research Institute of Mother and Child Health Care
🇲🇩Chisinau, Moldova, Republic of
Centre Hospitalier Ibn Sina
🇲🇦Rabat, Morocco
University Medical School Warsaw
🇵🇱Warsaw, Poland
Centro di Referimento per le Malattie Emorragiche e Trombotiche
🇮🇹Torino, Italy
HSJ - Hospital de São João, EPE
🇵🇹Porto, Portugal
Haemophilia Centre, University Clinical Centre
🇸🇮Ljubljana, Slovenia
Unitat d'hemofilia, Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
National Children's Specialized Hospital "OHMATDET"
🇺🇦Kiev, Ukraine
Institute of Blood Pathology and Transfusion Medicine
🇺🇦Lviv, Ukraine
Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
Cambridge University Hospital
🇬🇧Cambridge, United Kingdom
Hospital Universitario La Paz
🇪🇸Madrid, Spain