Patient Reported Outcomes Burdens and Experiences - Phase 2 (PROBE)

• ClinicalTrials.gov Identifier: NCT02439710
• Recruitment Status: Completed
• First Posted: May 12, 2015
• Results First Posted: October 31, 2019
• Last Update Posted: October 31, 2019
• Sponsor: McMaster University
• Collaborator: National Hemophilia Foundation
• Information provided by (Responsible Party): McMaster University

• Study Type: Observational
• Study Design: Observational Model: Other; Time Perspective: Prospective
• Condition: Hemophilia
• Intervention: Other: Quality of life measure (survey)
• Enrollment: 656

Participant Flow

Recruitment Details	Participants were recruited through NGOs utilizing their existing membership rosters, social media outlets and meetings or events.
Pre-assignment Details	Participants were required to meet one of two inclusion criteria: (1) PWH (haemophilia A or B with any degree of disease severity) or (2) “Controls” (individuals with no bleeding disorders)

Arm/Group Title	Enrolled Participants
Arm/Group Description	"Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or “Controls” (individuals with no bleeding disorders)
Period Title: Overall Study
Started	656
Completed	656
Not Completed	0

Baseline Characteristics

Arm/Group Title		Enrolled Population
Arm/Group Description		Patients with hemophilia A or hemophilia B with any degree of disease severity or controls
Overall Number of Baseline Participants		656
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	656 participants
		41.9 (17.0)
Sex: Female, Male [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	646 participants
	Female	454 70.3%
	Male	192 29.7%
		[1] Measure Analysis Population Description: Missing data
Race and Ethnicity Not Collected [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	0 participants
		[1] Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Region of Enrollment; Measure Type: Count of Participants; Unit of measure: Participants
Argentina	Number Analyzed	656 participants
		35 5.3%
Australia	Number Analyzed	656 participants
		39 5.9%
Brazil	Number Analyzed	656 participants
		20 3.0%
Canada	Number Analyzed	656 participants
		60 9.1%
France	Number Analyzed	656 participants
		46 7.0%
Germany	Number Analyzed	656 participants
		17 2.6%
Hungary	Number Analyzed	656 participants
		34 5.2%
Ireland	Number Analyzed	656 participants
		38 5.8%
Italy	Number Analyzed	656 participants
		50 7.6%
Japan	Number Analyzed	656 participants
		29 4.4%
Mexico	Number Analyzed	656 participants
		39 5.9%
Netherlands	Number Analyzed	656 participants
		49 7.5%
New Zealand	Number Analyzed	656 participants
		43 6.6%
Spain	Number Analyzed	656 participants
		17 2.6%
United Kingdom	Number Analyzed	656 participants
		22 3.4%
United States	Number Analyzed	656 participants
		84 12.8%
Venezuela	Number Analyzed	656 participants
		34 5.2%
Years of education; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	656 participants
		14.58 (4.67)
Marital status (married); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	656 participants
		421 64.2%

Outcome Measures

1. Primary Outcome

Title	Response Rate
Description	number of filled questionnaires compared to the total number that were sent out
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

patients with hemophilia (hemophilia A or B with any degree of disease severity) or controls

Arm/Group Title	Enrolled Participants
Arm/Group Description:	"Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or “Controls” (individuals with no bleeding disorders)
Overall Number of Participants Analyzed	656
Overall Number of Units Analyzed; Type of Units Analyzed: Questionnaires	1871
Count of Units; Unit of Measure: Questionnaires
	656 35.1%

2. Primary Outcome

Title	Number of Participant Completing the Questionnaire Within 15 Minutes
Description	Number of participants completing the questionnaire within 15 minutes
Time Frame	Up to 15 minutes

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Enrolled Participants
Arm/Group Description:	"Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or “Controls” (individuals with no bleeding disorders)
Overall Number of Participants Analyzed	656
Measure Type: Number; Unit of Measure: participants
	474

3. Primary Outcome

Title	Cost Per Completed Survey
Description	Human and financial resources used by local organization to run the survey - measured as median and full range
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Enrolled Participants
Arm/Group Description:	"Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or “Controls” (individuals with no bleeding disorders)
Overall Number of Participants Analyzed	656
Median (Full Range); Unit of Measure: Dollars (USD) per participants
	8.50; (1.20 to 64.30)

Adverse Events

Time Frame	1 year
Adverse Event Reporting Description	Although it is not anticipated that the PROBE Study Team would receive reports of adverse events within the scope of the Study, if the Investigators are made aware of an adverse event involving a product manufactured by a study sponsor , the Investigators commit to notify sponsor within one (1) business day of actually becoming aware of any significant product complaint, unexpected adverse event, or governmental investigation or inquiry involving a sponsor's product.
Arm/Group Title	Enrolled Participants
Arm/Group Description	"Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or “Controls” (individuals with no bleeding disorders)
All-Cause Mortality
	Enrolled Participants
	Affected / at Risk (%)
Total	0/656 (0.00%)
Serious Adverse Events
	Enrolled Participants
	Affected / at Risk (%)
Total	0/656 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Enrolled Participants
	Affected / at Risk (%)
Total	0/656 (0.00%)

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Mark W Skinner
• Organization: Institute for Policy Advancement Ltd
• Phone: +1-202-253-8342
• EMail: mskinnerdc@gmail.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Chai-Adisaksopha C, Skinner MW, Curtis R, Frick N, Nichol MB, Noone D, O'Mahony B, Page D, Stonebraker J, Thabane L, Crowther M, Iorio A. Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. BMJ Open. 2018 Aug 8;8(8):e021900. doi: 10.1136/bmjopen-2018-021900.

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.

• Responsible Party: McMaster University
• ClinicalTrials.gov Identifier: NCT02439710
• Other Study ID Numbers: HIREB-15-260D
• First Submitted: April 28, 2015
• First Posted: May 12, 2015
• Results First Submitted: May 15, 2018
• Results First Posted: October 31, 2019
• Last Update Posted: October 31, 2019