HemophiliaPROBE-3NCT03549858
Patient Reported Outcomes Burdens and Experiences - Phase 3
An observational study of Hemophilia and Chronic Disease, run by McMaster University at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia, Chronic Disease
- What is being tested?
- Quality of life measure (survey)
- How many people
- 1,000 participants
- Where
- 1 siteCanada
- Study dates
- 1 Jan 2018to 31 Dec 2027
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- Disease severity and Age bands or age limits (e.g. ≥ Age 18) may be utilized to narrow the study population.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Alfonso Iorio contact
Canada1 site, 1 recruiting
- Recruiting
McMaster University
Hamilton, Ontario, L8S4B2
Alfonso Iorio, MD, 9055259140 ext. 22421, iorioa@mcmaster.ca
About this study
Official title: 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.
Read the 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.
- Conditions
- Hemophilia, Chronic Disease
- Being tested
- Quality of life measure (survey)
- Medical subject headings
- Hemophilia A, Chronic Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Quality of life measure (survey)Other
The QoL tool is a questionnaire
What is being measured
The primary outcome is the main question the study is designed to answer.
Between-countries variability of the PROBE score
Mean difference and standard deviation of the PROBE score between countries.
Measured: 2 years
Secondary outcomes (2)
Within-countries variability of the PROBE score
Mean difference and standard deviation of the PROBE score in repeated assessments within countries.
Measured: 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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2018
- Study start1 Jan 2018
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- More trials run by McMaster UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- McMaster University
- Sponsor type
- University, hospital or other
- Collaborators
- Patient Outcomes Research Group (PORG), National Hemophilia Foundation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03549858
- Sponsor's protocol ID
- PROBE-3
- Acronym
- PROBE-3
- Status last verified
- Aug 2025
- First submitted
- 15 May 2018
- Last updated
- 22 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
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.
PubMed 29497561
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Patient Reported Outcomes Burdens and Experiences (PROBE) - Phase 3 - Longitudinal Data Collection
- ClinicalTrials.gov ID
- NCT03549858
- Acronym / sponsor ID
- PROBE-3 / PROBE-3
- Conditions
- Hemophilia, Chronic Disease
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- McMaster UniversityUniversity, hospital or other
- Collaborators
- Patient Outcomes Research Group (PORG), National Hemophilia Foundation
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 8 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Disease severity and Age bands or age limits (e.g. ≥ Age 18) may be utilized to narrow the study population.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Quality of life measure (survey)The QoL tool is a questionnaire | Other | – |
Primary outcome
Between-countries variability of the PROBE score
Time frame: 2 years
Mean difference and standard deviation of the PROBE score between countries.
2 secondary outcomes
Within-countries variability of the PROBE score
Time frame: 2 years
Within-countries comparison of the PROBE score in PWH against national normative data
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2025.
- Background: PubMed 29497561Skinner 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Alfonso Iorio contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia and Chronic Disease, run by McMaster University at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mark Skinner (202.253.8342, mskinnerdc@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Between-countries variability of the PROBE score (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03549858, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Aug 2025; AzTrials last refreshed its copy on 1 Nov 2026.