Trial Outcomes & Findings for Patient Reported Outcomes Burdens and Experiences - Phase 2 (NCT NCT02439710)

NCT ID: NCT02439710

Last Updated: 2019-10-31

Results Overview

number of filled questionnaires compared to the total number that were sent out

Recruitment status

COMPLETED

Target enrollment

656 participants

Primary outcome timeframe

1 year

Results posted on

2019-10-31

Participant Flow

Participants were recruited through NGOs utilizing their existing membership rosters, social media outlets and meetings or events.

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)

Participant milestones

Participant milestones
Measure	Enrolled Participants "Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or "Controls" (individuals with no bleeding disorders)
Overall Study STARTED	656
Overall Study COMPLETED	656
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Missing data

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Enrolled Population n=656 Participants Patients with hemophilia A or hemophilia B with any degree of disease severity or controls
Age, Continuous	41.9 years STANDARD_DEVIATION 17.0 • n=656 Participants
Sex: Female, Male Female	454 Participants n=646 Participants • Missing data
Sex: Female, Male Male	192 Participants n=646 Participants • Missing data
Region of Enrollment Argentina	35 Participants n=656 Participants
Region of Enrollment Australia	39 Participants n=656 Participants
Region of Enrollment Brazil	20 Participants n=656 Participants
Region of Enrollment Canada	60 Participants n=656 Participants
Region of Enrollment France	46 Participants n=656 Participants
Region of Enrollment Germany	17 Participants n=656 Participants
Region of Enrollment Hungary	34 Participants n=656 Participants
Region of Enrollment Ireland	38 Participants n=656 Participants
Region of Enrollment Italy	50 Participants n=656 Participants
Region of Enrollment Japan	29 Participants n=656 Participants
Region of Enrollment Mexico	39 Participants n=656 Participants
Region of Enrollment Netherlands	49 Participants n=656 Participants
Region of Enrollment New Zealand	43 Participants n=656 Participants
Region of Enrollment Spain	17 Participants n=656 Participants
Region of Enrollment United Kingdom	22 Participants n=656 Participants
Region of Enrollment United States	84 Participants n=656 Participants
Region of Enrollment Venezuela	34 Participants n=656 Participants
Years of education	14.58 years STANDARD_DEVIATION 4.67 • n=656 Participants
Marital status (married)	421 Participants n=656 Participants

PRIMARY outcome

Timeframe: 1 year

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

number of filled questionnaires compared to the total number that were sent out

Outcome measures

Outcome measures
Measure	Enrolled Participants n=1871 Questionnaires "Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or "Controls" (individuals with no bleeding disorders)
Response Rate	656 Questionnaires

PRIMARY outcome

Timeframe: Up to 15 minutes

Number of participants completing the questionnaire within 15 minutes

Outcome measures

Outcome measures
Measure	Enrolled Participants n=656 Participants "Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or "Controls" (individuals with no bleeding disorders)
Number of Participant Completing the Questionnaire Within 15 Minutes	474 participants

PRIMARY outcome

Timeframe: 1 year

Human and financial resources used by local organization to run the survey - measured as median and full range

Outcome measures

Outcome measures
Measure	Enrolled Participants n=656 Participants "Patients with hemophilia" (haemophilia A or B with any degree of disease severity) or "Controls" (individuals with no bleeding disorders)
Cost Per Completed Survey	8.50 Dollars (USD) per participants Interval 1.2 to 64.3

Adverse Events

Enrolled Participants

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Mark W Skinner

Institute for Policy Advancement Ltd

Phone: +1-202-253-8342

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place