Clinical trial NCT02476942: 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
Official title: A Multicenter, Non-Interventional Study Evaluating Bleeding Incidence, Health-Related Quality of Life, and Safety in Patients With Hemophilia A Under Standard-of-Care Treatment
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2015-06-22
Enrollment: 221
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with hemophilia A with or without inhibitors against FVIII under standard-of-care treatment are to be enrolled.

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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)

Primary outcomes:
measure: Number of Bleeds
timeFrame: Approximately 6 months (from Baseline until study completion)
Secondary outcomes:
measure: European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients
timeFrame: Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported)
measure: Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients
timeFrame: Approximately 6 months (every 4 weeks from Baseline until study completion)
measure: Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score
timeFrame: Approximately 6 months (every 4 weeks from Baseline until study completion)
Other outcomes:
Locations:
city: Santa Monica
country: United States
facility: Santa Monica Oncology Center
state: California
city: Aurora
country: United States
facility: University of Colorado Denver, Children's Hospital
state: Colorado
city: Washington, D.C.
country: United States
facility: Georgetown Uni Medical Center; Lombardi Cancer Center
state: District of Columbia
city: Atlanta
country: United States
facility: Winship Cancer Institute
state: Georgia
city: New Orleans
country: United States
facility: Tulane Uni Health Sciences Center
state: Louisiana
city: Detroit
country: United States
facility: Children's Hospital of Michigan; Pediatrics
state: Michigan
city: New York
country: United States
facility: Cornell Univ Medical College; Hematology-Oncolog
state: New York
city: Portland
country: United States
facility: Oregon Health & Science Uni ; Dept of Pediatrics
state: Oregon
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Seattle
country: United States
facility: Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
state: Washington
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital; Haematology
state: New South Wales
city: Melbourne
country: Australia
facility: The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit
state: Victoria
city: Beijing
country: China
facility: Peking Union Medical College Hospital
city: Guangzhou
country: China
facility: Nanfang Hospital, Southern Medical University
city: Tianjin
country: China
facility: Tianjin Institute of Hematology & Blood Diseases Hospital
city: San Jose
country: Costa Rica
facility: ICIC
city: Bonn
country: Germany
facility: Universitätsklinikum Bonn (AöR); Inst. für Experimentelle Hämatologie u. Transfusionsmedizin (IHT)
city: Milano
country: Italy
facility: IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"
state: Lombardia
city: Firenze
country: Italy
facility: AOU Careggi; SOD Malattie Emorragiche
state: Toscana
city: Aichi
country: Japan
facility: Nagoya University Hospital
city: Hyogo
country: Japan
facility: Hyogo College of Medicine Hospital
city: Kanagawa
country: Japan
facility: St. Marianna University School of Medicine Hospital
city: Kitakyushu-shi
country: Japan
facility: Hospital of the University of Occupational and Environmental Health,Japan
city: Nara
country: Japan
facility: Nara Medical University Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii
city: Lublin
country: Poland
facility: SPSK Nr1 Klinika Hematoo&Transpl.Szpiku
city: Poznan
country: Poland
facility: ALVAMED Lekarskie Gabinety Specjalistyczne
city: Warsaw
country: Poland
facility: Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych
city: Johannesburg
country: South Africa
facility: Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center
city: Madrid
country: Spain
facility: Hospital Universitario la Paz; Servicio de Hematologia
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio; Servicio de Hematologia
city: Valencia
country: Spain
facility: Hospital Universitario la Fe; Servicio de Hematologia
city: Taipei
country: Taiwan
facility: National Taiwan Uni Hospital
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References:
citation: Oldenburg J, Shima M, Kruse-Jarres R, Santagostino E, Mahlangu J, Lehle M, Selak Bienz N, Chebon S, Asikanius E, Trask P, Mancuso ME, Jimenez-Yuste V, von Mackensen S, Levy GG. Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional study. Pediatr Blood Cancer. 2020 Oct;67(10):e28474. doi: 10.1002/pbc.28474. Epub 2020 Aug 9.