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

May 26, 2017 updated by: Hoffmann-La Roche

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

221

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Royal Prince Alfred Hospital; Haematology
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit
  • China
      • Beijing, China, 100730
        • Peking Union Medical College Hospital
      • Guangzhou, China, 510515
        • Nanfang Hospital, Southern Medical University
      • Tianjin, China, 300020
        • Tianjin Institute of Hematology & Blood Diseases Hospital
  • Costa Rica
      • San Jose, Costa Rica, 1000
        • ICIC
  • Germany
      • Bonn, Germany, 53127
        • Universitätsklinikum Bonn (AöR); Inst. für Experimentelle Hämatologie u. Transfusionsmedizin (IHT)
  • Italy
    • Lombardia
      • Milano, Lombardia, Italy, 20122
        • IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"
    • Toscana
      • Firenze, Toscana, Italy, 50134
        • AOU Careggi; SOD Malattie Emorragiche
  • Japan
      • Aichi, Japan, 466-8560
        • Nagoya University Hospital
      • Hyogo, Japan, 663-8501
        • Hyogo College of Medicine Hospital
      • Kanagawa, Japan, 216-8511
        • St. Marianna University School of Medicine Hospital
      • Kitakyushu-shi, Japan, 807-8556
        • Hospital of the University of Occupational and Environmental Health,Japan
      • Nara, Japan, 634-8521
        • Nara Medical University Hospital
      • Tokyo, Japan, 160-0023
        • Tokyo Medical University Hospital
  • Korea, Republic of
      • Seoul, Korea, Republic of, 03722
        • Severance Hospital
  • Poland
      • Gdansk, Poland, 80-952
        • Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii
      • Lublin, Poland, 20-081
        • SPSK Nr1 Klinika Hematoo&Transpl.Szpiku
      • Poznan, Poland, 61-828
        • ALVAMED Lekarskie Gabinety Specjalistyczne
      • Warsaw, Poland, 02-776
        • Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych
  • South Africa
      • Johannesburg, South Africa, 2193
        • Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center
  • Spain
      • Madrid, Spain, 28046
        • Hospital Universitario la Paz; Servicio de Hematologia
      • Sevilla, Spain, 41013
        • Hospital Universitario Virgen del Rocio; Servicio de Hematologia
      • Valencia, Spain, 46026
        • Hospital Universitario la Fe; Servicio de Hematologia
  • Taiwan
      • Taipei, Taiwan, 100
        • National Taiwan Uni Hospital
  • United States
    • California
      • Santa Monica, California, United States, 90403
        • Santa Monica Oncology Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Denver, Children's Hospital
    • District of Columbia
      • Washington, D.C., District of Columbia, United States, 20007
        • Georgetown Uni Medical Center; Lombardi Cancer Center
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Winship Cancer Institute
    • Louisiana
      • New Orleans, Louisiana, United States, 70112-2699
        • Tulane Uni Health Sciences Center
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Children's Hospital of Michigan; Pediatrics
    • New York
      • New York, New York, United States, 10021
        • Cornell Univ Medical College; Hematology-Oncolog
    • Oregon
      • Portland, Oregon, United States, 97201
        • Oregon Health & Science Uni ; Dept of Pediatrics
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh
    • Washington
      • Seattle, Washington, United States, 98104
        • Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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.
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.
Cohort B: Children with FVIII Inhibitors
Children with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.
Cohort C: Adults and Adolescents without FVIII Inhibitors
Adults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Bleeds
Time Frame: Approximately 6 months (from Baseline until study completion)
Approximately 6 months (from Baseline until study completion)

Secondary Outcome Measures

Outcome Measure
Time Frame
European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients
Time Frame: Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported)
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
Time Frame: Approximately 6 months (every 4 weeks from Baseline until study completion)
Approximately 6 months (every 4 weeks from Baseline until study completion)
Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score
Time Frame: Approximately 6 months (every 4 weeks from Baseline until study completion)
Approximately 6 months (every 4 weeks from Baseline until study completion)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hoffmann-La Roche

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 26, 2015

Primary Completion (Actual)

March 31, 2017

Study Completion (Actual)

March 31, 2017

Study Registration Dates

First Submitted

June 3, 2015

First Submitted That Met QC Criteria

June 19, 2015

First Posted (Estimate)

June 22, 2015

Study Record Updates

Last Update Posted (Actual)

May 30, 2017

Last Update Submitted That Met QC Criteria

May 26, 2017

Last Verified

May 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BH29768

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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