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Patient Reported Outcomes Burdens and Experiences - Phase 2 (PROBE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02439710
Recruitment Status : Completed
First Posted : May 12, 2015
Results First Posted : October 31, 2019
Last Update Posted : October 31, 2019
National Hemophilia Foundation
Information provided by (Responsible Party):
McMaster University

Brief Summary:
The Patient Reported Outcomes, Burdens, and Experiences (PROBE) Study aims to develop a new global tool to enhance the direct patient-voice in health care decision-making. Government and private payers increasingly value data based on patient-centered outcomes research as part of the overall cost-benefit evaluation of high-cost care and treatment of diseases such as hemophilia. This emerging dimension of the healthcare environment presents a significant opportunity and urgent need to improve patient organizations' ability to collect and interpret relevant outcomes data. More robust patient reported data will improve advocacy efforts to build comprehensive care programs, promote home treatment and implement preventative treatment regimens thus allowing advocacy arguments to move beyond emotion and anecdote to those grounded in real-world patient experiences and evidence. With the support of the National Hemophilia Foundation, a global team of investigators will lead a patient focused research project to investigate and directly probe patient perspectives on outcomes they deem relevant to their care. Through PROBE, the investigators will develop and seek to validate the reliability, reproducibility and responsiveness of a low cost, easily administrable inventory for collecting patient self-reported outcomes, burdens and experiences in living with hemophilia. The investigators anticipate that the metrics established through PROBE will allow for comparison of patient outcomes within a country over time and cross-sectionally between countries (regionally and globally).

Condition or disease Intervention/treatment
Hemophilia Other: Quality of life measure (survey)

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Study Type : Observational [Patient Registry]
Actual Enrollment : 656 participants
Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration: 1 Year
Official Title: Patient Reported Outcomes Burdens and Experiences - Phase 2 - Pilot Feasibility Study
Actual Study Start Date : June 2015
Actual Primary Completion Date : December 31, 2018
Actual Study Completion Date : December 31, 2018

Resource links provided by the National Library of Medicine

Intervention Details:
  • Other: Quality of life measure (survey)
    The QoL tool is a questionnaire

Primary Outcome Measures :
  1. Response Rate [ Time Frame: 1 year ]
    number of filled questionnaires compared to the total number that were sent out

  2. Number of Participant Completing the Questionnaire Within 15 Minutes [ Time Frame: Up to 15 minutes ]
    Number of participants completing the questionnaire within 15 minutes

  3. Cost Per Completed Survey [ Time Frame: 1 year ]
    Human and financial resources used by local organization to run the survey - measured as median and full range

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Moderate / Severe PWH (Age bands or limits may be utilized to narrow the study population). Patients will be recruited according to the final inclusion criteria. No randomization will be required. Two moderate to large test runs of the inventory will be conducted in each country three months apart to demonstrate reproducibility. PWH participating in each of the two test runs may, but would not necessarily be the same.

Inclusion Criteria:

  • PWH will be recruited through national hemophilia patient organizations utilizing their existing membership rosters, social media outlets and meetings / events. The investigators are not proposing a pre-determined method of PWH recruitment. They will utilize the information acquired in the workshop and take-home project to inform best practice in recruitment methodology for the study. The investigators may consider requesting different countries test different PWH recruitment strategies to test reproducibility.

Exclusion Criteria:

  • Disease severity and Age bands or age limits (e.g. ≥ Age 18) may be utilized to narrow the study population. During the January 2015 meeting of the investigators the inclusion / exclusion criteria will be finalized based on feedback learned during the workshop. Disease severity will be based on patient self-reported severity level.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02439710

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Canada, Ontario
McMaster University
Hamilton, Ontario, Canada, L8S4B2
Sponsors and Collaborators
McMaster University
National Hemophilia Foundation
  Study Documents (Full-Text)

Documents provided by McMaster University:
Additional Information:

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: McMaster University Identifier: NCT02439710    
Other Study ID Numbers: HIREB-15-260D
First Posted: May 12, 2015    Key Record Dates
Results First Posted: October 31, 2019
Last Update Posted: October 31, 2019
Last Verified: October 2019