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

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02476942
• Other study ID #: BH29768
• Status: Completed
• Phase: N/A
• First received: June 3, 2015
• Last updated: May 26, 2017
• Start date: May 26, 2015
• Est. completion date: March 31, 2017

Study information

• Verified date: May 2017
• Source: Hoffmann-La Roche
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 221
• Est. completion date: March 31, 2017
• Est. primary completion date: March 31, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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)

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Bypassing Agents
Episodic or prophylactic treatment with the use of bypassing agents must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.
• FVIII Replacement
Episodic or prophylactic treatment with the use of FVIII replacement must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.

Locations

Country	Name	City	State
Australia	Royal Prince Alfred Hospital; Haematology	Camperdown	New South Wales
Australia	The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit	Melbourne	Victoria
China	Peking Union Medical College Hospital	Beijing
China	Nanfang Hospital, Southern Medical University	Guangzhou
China	Tianjin Institute of Hematology & Blood Diseases Hospital	Tianjin
Costa Rica	ICIC	San Jose
Germany	Universitätsklinikum Bonn (AöR); Inst. für Experimentelle Hämatologie u. Transfusionsmedizin (IHT)	Bonn
Italy	AOU Careggi; SOD Malattie Emorragiche	Firenze	Toscana
Italy	IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"	Milano	Lombardia
Japan	Nagoya University Hospital	Aichi
Japan	Hyogo College of Medicine Hospital	Hyogo
Japan	St. Marianna University School of Medicine Hospital	Kanagawa
Japan	Hospital of the University of Occupational and Environmental Health,Japan	Kitakyushu-shi
Japan	Nara Medical University Hospital	Nara
Japan	Tokyo Medical University Hospital	Tokyo
Korea, Republic of	Severance Hospital	Seoul
Poland	Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii	Gdansk
Poland	SPSK Nr1 Klinika Hematoo&Transpl.Szpiku	Lublin
Poland	ALVAMED Lekarskie Gabinety Specjalistyczne	Poznan
Poland	Instytut Hematologii i Transfuzjologii; Klinika Zaburzen Hemostazy i Chorób Wewnetrznych	Warsaw
South Africa	Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center	Johannesburg
Spain	Hospital Universitario la Paz; Servicio de Hematologia	Madrid
Spain	Hospital Universitario Virgen del Rocio; Servicio de Hematologia	Sevilla
Spain	Hospital Universitario la Fe; Servicio de Hematologia	Valencia
Taiwan	National Taiwan Uni Hospital	Taipei
United States	Winship Cancer Institute	Atlanta	Georgia
United States	University of Colorado Denver, Children's Hospital	Aurora	Colorado
United States	Children's Hospital of Michigan; Pediatrics	Detroit	Michigan
United States	Tulane Uni Health Sciences Center	New Orleans	Louisiana
United States	Cornell Univ Medical College; Hematology-Oncolog	New York	New York
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health & Science Uni ; Dept of Pediatrics	Portland	Oregon
United States	Santa Monica Oncology Center	Santa Monica	California
United States	Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia	Seattle	Washington
United States	Georgetown Uni Medical Center; Lombardi Cancer Center	Washington, D.C.	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Countries where clinical trial is conducted

United States,  Australia,  China,  Costa Rica,  Germany,  Italy,  Japan,  Korea, Republic of,  Poland,  South Africa,  Spain,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Bleeds		Approximately 6 months (from Baseline until study completion)
Secondary	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)
Secondary	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)
Secondary	Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score		Approximately 6 months (every 4 weeks from Baseline until study completion)