- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03549858
Patient Reported Outcomes Burdens and Experiences - Phase 3 (PROBE-3)
Patient Reported Outcomes Burdens and Experiences (PROBE) - Phase 3 - Longitudinal Data Collection
Study Overview
Status
Conditions
Intervention / Treatment
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:
Mark Skinner JD, Institute for Policy Advancement Ltd. (US)
Co-Investigators:
- Alfonso Iorio MD Ph.D. FRCPC, McMaster University, Department of Clinical Epidemiology and Biostatistics (Canada)
- Randall Curtis MBA, Factor VIII Computing (US)
- Neil Frick MS, National Hemophilia Foundation (US)
- Michael Nichol Ph.D., University of Southern California, School of Policy and Planning Development (US)
- Declan Noone, Irish Hemophilia Society (Ireland)
- David Page, Canadian Hemophilia Society (Canada)
- Jeff Stonebraker Ph.D., North Carolina State University Poole College of Management (US)
- Brian O'Mahoney, Irish Hemophilia Society (Ireland)
- Chatree Chai-Adisaksopha, MD MSc.
Study Type
Enrollment (Estimated)
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
-
-
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
Sampling Method
Study Population
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?
Design Details
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Between-countries variability of the PROBE score
Time Frame: 2 years
|
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
Time Frame: 2 years
|
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
Time Frame: 2 years
|
Mean difference and standard deviation of the PROBE score in PWH against national normative data within countries.
|
2 years
|
Collaborators and Investigators
Sponsor
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- PROBE-3
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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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