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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Hemophilia; Chronic Disease
• Intervention / Treatment: Other: Quality of life measure (survey)

Detailed Description

BACKGROUND

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.

CURRENT STUDY and FUTURE DIRECTIONS

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

INVESTIGATION TEAM

Principal Investigator:

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

   Co-Investigators:

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.

• Study Type: Observational
• Enrollment (Estimated): 1000

Contacts and Locations

• Study Contact: Name: Mark Skinner; Phone Number: 202.253.8342; Email: mskinnerdc@gmail.com
• Study Contact Backup: Name: Alfonso Iorio; Email: iorioa@mcmaster.ca

Study Locations

• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8S4B2
      • Recruiting
      • McMaster University
      • Contact: Alfonso Iorio, MD; Phone Number: 22421 9055259140; Email: iorioa@mcmaster.ca

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• 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.

Description

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.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Between-countries variability of the PROBE score	Mean difference and standard deviation of the PROBE score between countries.	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Within-countries variability of the PROBE score	Mean difference and standard deviation of the PROBE score in repeated assessments within countries.	2 years
Within-countries comparison of the PROBE score in PWH against national normative data	Mean difference and standard deviation of the PROBE score in PWH against national normative data within countries.	2 years

Collaborators and Investigators

• Sponsor: McMaster University
• Collaborators: National Hemophilia Foundation; Patient Outcomes Research Group (PORG)

Publications and helpful links

General Publications

• Skinner MW, Chai-Adisaksopha C, Curtis R, Frick N, Nichol M, Noone D, O'Mahony B, Page D, Stonebraker JS, Iorio A. The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia. Pilot Feasibility Stud. 2018 Feb 27;4:58. doi: 10.1186/s40814-018-0253-0. eCollection 2018.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2018
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: May 15, 2018
• First Submitted That Met QC Criteria: May 25, 2018
• First Posted (Actual): June 8, 2018

Study Record Updates

• Last Update Posted (Actual): August 22, 2025
• Last Update Submitted That Met QC Criteria: August 18, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Chronic Disease
• Other Study ID Numbers: PROBE-3

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No