Patient Reported Outcomes Burdens and Experiences

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-01-01

Study Completion Date

2027-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Health Related Quality of Life of Youth and Young Adults With Hemophilia A

NCT01034904

Hemophilia A

COMPLETED

Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

NCT02256917

Severe Haemophilia A

COMPLETED PHASE3

Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

NCT03876301

Blood Coagulation Disorder Blood Coagulation Disorders, Inherited Coagulation Protein Disorders +6 more

COMPLETED

Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment

NCT02396862

Hemophilia A

COMPLETED

A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment

NCT02476942

Hemophilia A

COMPLETED

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hemophilia Chronic Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Quality of life measure (survey)

The QoL tool is a questionnaire

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.