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Patient Reported Outcomes Burdens and Experiences - Phase 3 (PROBE-3)

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ClinicalTrials.gov Identifier: NCT03549858
Recruitment Status : Recruiting
First Posted : June 8, 2018
Last Update Posted : August 12, 2022
Patient Outcomes Research Group (PORG)
National Hemophilia Foundation
Information provided by (Responsible Party):
McMaster University

Brief Summary:
The PROBE Phase-3 study will collect data on patient reported outcomes, burdens, and experiences in patients living with hemophilia. The investigators will perform comparisons among countries, within country over time, within country against national normative data.

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

Detailed Description:


The PROBE questionnaire is a tool for the assessment of patient-reported outcome, burdens and experiences. Until now, it has been used in persons living with hemophilia (PWH) and healthy controls. In phase 1 of the study, the investigators developed the questionnaire. In phase 2 of the study, the investigators evaluated the reliability, reproducibility, responsiveness, and costs of PROBE. The tool has been tested across 21 countries and it has been shown that PROBE is a valid questionnaire for the evaluation of patient reported outcomes (PROs) in PWH and control populations. The questionnaire is available in 11 languages (with 20 localized versions worldwide), and another 17 languages and 45 more localizations are in the process of development.


Phase 3 is the natural prosecution of the project. In this phase, the investigators aim at maintaining the capacity to collect data on PROBE, and the infrastructure will also be modified in order to allow for longitudinal collection of PROBE data (in previous phases, the investigators only collected anonymized data).

The objective of the study is to compare data on PROBE between countries, within-countries over time, and within-countries against national normative data. Reports will be produced every 2 years. Data collected during phase 2 of the study will also be used.

At the same time, the research framework will have the potential for:

  • Expanding country participation within existing and new regions.
  • Integrating the PROBE questionnaire in clinical trials, longitudinal studies, health technology assessment studies, routine clinical care, and registries.
  • Using PROBE in new countries
  • Testing the performance of the PROBE questionnaire in new patient populations


Principal Investigator:

  1. Mark Skinner JD, Institute for Policy Advancement Ltd. (US)


  2. Alfonso Iorio MD Ph.D. FRCPC, McMaster University, Department of Clinical Epidemiology and Biostatistics (Canada)
  3. Randall Curtis MBA, Factor VIII Computing (US)
  4. Neil Frick MS, National Hemophilia Foundation (US)
  5. Michael Nichol Ph.D., University of Southern California, School of Policy and Planning Development (US)
  6. Declan Noone, Irish Hemophilia Society (Ireland)
  7. David Page, Canadian Hemophilia Society (Canada)
  8. Jeff Stonebraker Ph.D., North Carolina State University Poole College of Management (US)
  9. Brian O'Mahoney, Irish Hemophilia Society (Ireland)
  10. Chatree Chai-Adisaksopha, MD MSc.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 1000 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: Patient Reported Outcomes Burdens and Experiences (PROBE) - Phase 3 - Longitudinal Data Collection
Actual Study Start Date : January 1, 2018
Estimated Primary Completion Date : December 31, 2025
Estimated Study Completion Date : December 31, 2026

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. Between-countries variability of the PROBE score [ Time Frame: 2 years ]
    Mean difference and standard deviation of the PROBE score between countries.

Secondary Outcome Measures :
  1. Within-countries variability of the PROBE score [ Time Frame: 2 years ]
    Mean difference and standard deviation of the PROBE score in repeated assessments within countries.

  2. Within-countries comparison of the PROBE score in PWH against national normative data [ Time Frame: 2 years ]
    Mean difference and standard deviation of the PROBE score in PWH against national normative data within countries.

Information from the National Library of Medicine

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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.

In the future, the questionnaire might also be administered to patients with other chronic conditions.

Exclusion Criteria:

  • Disease severity and Age bands or age limits (e.g. ≥ Age 18) may be utilized to narrow the study population.

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 ClinicalTrials.gov identifier (NCT number): NCT03549858

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Contact: Mark Skinner 202.253.8342 mskinnerdc@gmail.com
Contact: Alfonso Iorio iorioa@mcmaster.ca

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Canada, Ontario
McMaster University Recruiting
Hamilton, Ontario, Canada, L8S4B2
Contact: Alfonso Iorio, MD    9055259140 ext 22421    iorioa@mcmaster.ca   
Sponsors and Collaborators
McMaster University
Patient Outcomes Research Group (PORG)
National Hemophilia Foundation
Additional Information:

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Responsible Party: McMaster University
ClinicalTrials.gov Identifier: NCT03549858    
Other Study ID Numbers: PROBE-3
First Posted: June 8, 2018    Key Record Dates
Last Update Posted: August 12, 2022
Last Verified: August 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Chronic Disease
Disease Attributes
Pathologic Processes