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

NCT ID: NCT02476942

Last Updated: 2017-05-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

221 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-05-26

Study Completion Date

2017-03-31

Brief Summary

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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

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Conditions

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Hemophilia A

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Cohort A: Adults and Adolescents with FVIII Inhibitors

Adults and adolescents with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.

Bypassing Agents

Intervention Type DRUG

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.

Cohort B: Children with FVIII Inhibitors

Children with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.

No interventions assigned to this group

Cohort C: Adults and Adolescents without FVIII Inhibitors

Adults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed.

FVIII Replacement

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type DRUG

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.

Intervention Type DRUG

Eligibility Criteria

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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)
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Santa Monica Oncology Center

Santa Monica, California, United States

Site Status

University of Colorado Denver, Children's Hospital

Aurora, Colorado, United States

Site Status

Georgetown Uni Medical Center; Lombardi Cancer Center

Washington D.C., District of Columbia, United States

Site Status

Winship Cancer Institute

Atlanta, Georgia, United States

Site Status

Tulane Uni Health Sciences Center

New Orleans, Louisiana, United States

Site Status

Children's Hospital of Michigan; Pediatrics

Detroit, Michigan, United States

Site Status

Cornell Univ Medical College; Hematology-Oncolog

New York, New York, United States

Site Status

Oregon Health & Science Uni ; Dept of Pediatrics

Portland, Oregon, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia

Seattle, Washington, United States

Site Status

Royal Prince Alfred Hospital; Haematology

Camperdown, New South Wales, Australia

Site Status

The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit

Melbourne, Victoria, Australia

Site Status

Peking Union Medical College Hospital

Beijing, , China

Site Status

Nanfang Hospital, Southern Medical University

Guangzhou, , China

Site Status

Tianjin Institute of Hematology & Blood Diseases Hospital

Tianjin, , China

Site Status

ICIC

San José, , Costa Rica

Site Status

Universitätsklinikum Bonn (AöR); Inst. für Experimentelle Hämatologie u. Transfusionsmedizin (IHT)

Bonn, , Germany

Site Status

IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"

Milan, Lombardy, Italy

Site Status

AOU Careggi; SOD Malattie Emorragiche

Florence, Tuscany, Italy

Site Status

Nagoya University Hospital

Aichi, , Japan

Site Status

Hyogo College of Medicine Hospital

Hyōgo, , Japan

Site Status

St. Marianna University School of Medicine Hospital

Kanagawa, , Japan

Site Status

Hospital of the University of Occupational and Environmental Health,Japan

Kitakyushu-shi, , Japan

Site Status

Nara Medical University Hospital

Nara, , Japan

Site Status

Tokyo Medical University Hospital

Tokyo, , Japan

Site Status

Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii

Gdansk, , Poland

Site Status

SPSK Nr1 Klinika Hematoo&Transpl.Szpiku

Lublin, , Poland

Site Status

ALVAMED Lekarskie Gabinety Specjalistyczne

Poznan, , Poland

Site Status

Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych

Warsaw, , Poland

Site Status

Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center

Johannesburg, , South Africa

Site Status

Severance Hospital

Seoul, , South Korea

Site Status

Hospital Universitario la Paz; Servicio de Hematologia

Madrid, , Spain

Site Status

Hospital Universitario Virgen del Rocio; Servicio de Hematologia

Seville, , Spain

Site Status

Hospital Universitario la Fe; Servicio de Hematologia

Valencia, , Spain

Site Status

National Taiwan Uni Hospital

Taipei, , Taiwan

Site Status

Countries

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

References

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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.

Reference Type DERIVED
PMID: 32776489 (View on PubMed)

Other Identifiers

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BH29768

Identifier Type: -

Identifier Source: org_study_id

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