A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment
- Conditions
- Hemophilia A
- Interventions
- Drug: FVIII ReplacementDrug: Bypassing Agents
- Registration Number
- NCT02476942
- Lead Sponsor
- Hoffmann-La Roche
- Brief Summary
This non-interventional study will prospectively collect detailed, high-quality documentation of bleeds, HRQoL, and safety in patients with hemophilia A with or without FVIII inhibitors treated according to local routine clinical practice (receiving FVIII replacement or bypassing agents as either episodic or prophylactic treatment). Actual patients will be enrolled from routine clinical practice in this observational study.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 221
- Cohort A: Patients greater than or equal to (>/=) 12 years of age at time of informed consent
- Cohort A: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, >/= 5 Bethesda units [BU])
- Cohort B: Pediatric patients less than (<) 12 years of age
- Cohort B: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, >/=5 BU)
- Cohort C: Patients >/=12 years of age
- Cohort C: Diagnosis of congenital hemophilia A and FVIII activity <1 percent (%)
- Cohort C: No prior history of a positive inhibitor against FVIII
- Prior RO5534262 (not applicable if patient agrees that prior RO5534262 will preclude participation in a future investigational RO5534262 study)
- Bleeding disorder other than congenital hemophilia A
- Ongoing (or planned during the study) immune tolerance induction therapy with FVIII or FVIII prophylaxis if currently/previously exposed to an inhibitor
- Previous or concomitant thromboembolic disease
- Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count <200 cells per microliter (cells/mcL)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Cohort C: Adults and Adolescents without FVIII Inhibitors FVIII Replacement Adults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed. Cohort A: Adults and Adolescents with FVIII Inhibitors Bypassing Agents Adults and adolescents with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.
- Primary Outcome Measures
Name Time Method Number of Bleeds Approximately 6 months (from Baseline until study completion)
- Secondary Outcome Measures
Name Time Method European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported) Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients Approximately 6 months (every 4 weeks from Baseline until study completion) Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score Approximately 6 months (every 4 weeks from Baseline until study completion)
Trial Locations
- Locations (35)
University of Colorado Denver, Children's Hospital
🇺🇸Aurora, Colorado, United States
Santa Monica Oncology Center
🇺🇸Santa Monica, California, United States
Georgetown Uni Medical Center; Lombardi Cancer Center
🇺🇸Washington, D.C., District of Columbia, United States
Children's Hospital of Michigan; Pediatrics
🇺🇸Detroit, Michigan, United States
Winship Cancer Institute
🇺🇸Atlanta, Georgia, United States
Cornell Univ Medical College; Hematology-Oncolog
🇺🇸New York, New York, United States
Royal Prince Alfred Hospital; Haematology
🇦🇺Camperdown, New South Wales, Australia
Oregon Health & Science Uni ; Dept of Pediatrics
🇺🇸Portland, Oregon, United States
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
🇺🇸Seattle, Washington, United States
ICIC
🇨🇷San Jose, Costa Rica
AOU Careggi; SOD Malattie Emorragiche
🇮🇹Firenze, Toscana, Italy
Universitätsklinikum Bonn (AöR); Inst. für Experimentelle Hämatologie u. Transfusionsmedizin (IHT)
🇩🇪Bonn, Germany
Hyogo College of Medicine Hospital
🇯🇵Hyogo, Japan
Tokyo Medical University Hospital
🇯🇵Tokyo, Japan
Severance Hospital
🇰🇷Seoul, Korea, Republic of
SPSK Nr1 Klinika Hematoo&Transpl.Szpiku
🇵🇱Lublin, Poland
Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center
🇿🇦Johannesburg, South Africa
Hospital Universitario la Paz; Servicio de Hematologia
🇪🇸Madrid, Spain
Hospital Universitario Virgen del Rocio; Servicio de Hematologia
🇪🇸Sevilla, Spain
Nanfang Hospital, Southern Medical University
🇨🇳Guangzhou, China
Tianjin Institute of Hematology & Blood Diseases Hospital
🇨🇳Tianjin, China
Peking Union Medical College Hospital
🇨🇳Beijing, China
Hospital of the University of Occupational and Environmental Health,Japan
🇯🇵Kitakyushu-shi, Japan
IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"
🇮🇹Milano, Lombardia, Italy
Hospital Universitario la Fe; Servicio de Hematologia
🇪🇸Valencia, Spain
The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit
🇦🇺Melbourne, Victoria, Australia
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Nagoya University Hospital
🇯🇵Aichi, Japan
ALVAMED Lekarskie Gabinety Specjalistyczne
🇵🇱Poznan, Poland
National Taiwan Uni Hospital
🇨🇳Taipei, Taiwan
St. Marianna University School of Medicine Hospital
🇯🇵Kanagawa, Japan
Nara Medical University Hospital
🇯🇵Nara, Japan
Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii
🇵🇱Gdansk, Poland
Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych
🇵🇱Warsaw, Poland
Tulane Uni Health Sciences Center
🇺🇸New Orleans, Louisiana, United States