Collecting patient experiences and outcomes in hemophilia
Patient Reported Outcomes Burdens and Experiences (PROBE) - Phase 3 - Longitudinal Data Collection
This study is trying to collect and compare experiences and outcomes from people with hemophilia across different countries to better understand their needs and challenges over time.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1000 (estimated) |
| Sex | All |
| Sponsor | McMaster University Academic / other |
| Locations | 1 site (Hamilton, Ontario) |
| Trial ID | NCT03549858 on ClinicalTrials.gov |
What this trial studies
The PROBE Phase-3 study aims to gather data on patient-reported outcomes, burdens, and experiences from individuals living with hemophilia. This observational study will compare data across different countries and within countries over time, utilizing a validated questionnaire developed in previous phases. The study will also adapt its infrastructure to allow for longitudinal data collection, enhancing the understanding of patient experiences. Reports will be generated every two years to track changes and trends in patient-reported outcomes.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals living with hemophilia, particularly those aged 18 and older.
Not a fit: Patients with severe disease conditions unrelated to hemophilia or those under the age of 18 may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the quality of life and challenges faced by patients with hemophilia, potentially guiding better treatment and support strategies.
How similar studies have performed: Previous phases of the PROBE study have shown success in validating the questionnaire across multiple countries, indicating a strong foundation for this phase.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Hamilton, Ontario
- McMaster University — Hamilton, Ontario, Canada (Recruiting)
Study contacts
- Study coordinator: Mark Skinner
- Email: mskinnerdc@gmail.com
- Phone: 202.253.8342
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.