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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 26, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 31, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 31, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 3, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 19, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 22, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 30, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 26, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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