Clinical trial NCT02396862: Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment
Official title: A Prospective, International, Longitudinal, Observational Disease Registry of Patient-reported Outcomes (PROs) and the Association With Hemophilia A and Its Treatment in Patients With Moderate to Severe Hemophilia A
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2015-03-24
Enrollment: 272
Sex: MALE
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients aged over 16 years, with documented physician-confirmed diagnosis of moderate or severe Hemophilia A (severity defined as moderate = FVIII activity 1% to 5% and severe = FVIII activity ≤1%)

Brief summary: The purpose of the study is to improve the understanding of key patient reported outcomes such as quality of life as well as clinical outcomes in hemophilia A, in a global real world setting.


Eligibility criteria: Inclusion Criteria:

* Age 16 or over.
* Have documentation of physician-confirmed diagnosis of moderate or severe Hemophilia A (severity defined as moderate = FVIII activity 1% to 5% and severe = FVIII activity ≤1%).
* Signed written informed consent provided by the patient or the patient's parents for patients under the age of 18 (dependent of local regulations).
* Signed written assent is also required for patients under the age 18 years (dependent on local regulations).
* Plan to receive at least half of their Hemophilia care at the registry site.
* Willing and able to enter data as per the data collection schedule.
* Currently receiving prophylactic or on demand treatment (including within last 6months for on demand).
* Expected life expectancy of at least 2 years.

Exclusion Criteria:

* Patients with Hemophilia B
* Patients with von Willebrand disease (vWD)
* Patients with other rare bleeding disorders
* Unable to comply with the study protocol

Primary outcomes:
measure: Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
timeFrame: Baseline and at 12 months
measure: Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
timeFrame: Baseline and at 24 months
measure: Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
timeFrame: Baseline and at 36 months
measure: Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
timeFrame: Baseline and at 48 months
measure: Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
timeFrame: Baseline and at 60 months
measure: Patient Reported Outcome: Change of Quality of Life as measured by EuroQol-5 Dimension questionnaire 5 Level (EQ5D), Short Form Health Survey (SF-12), Hemophilia Quality of Life Measure for Adults (HAEMO-QoL-A)
timeFrame: Baseline and at 12 months
measure: Patient Reported Outcome: Change of Quality of Life as measured by EuroQol-5 Dimension questionnaire 5 Level (EQ5D), Short Form Health Survey (SF-12), Hemophilia Quality of Life Measure for Adults (HAEMO-QoL-A)
timeFrame: Baseline and at 24 months
measure: Patient Reported Outcome: Change of Quality of Life as measured by EuroQol-5 Dimension questionnaire 5 Level (EQ5D), Short Form Health Survey (SF-12), Hemophilia Quality of Life Measure for Adults (HAEMO-QoL-A)
timeFrame: Baseline and at 36 months
measure: Patient Reported Outcome: Change of Quality of Life as measured by EuroQol-5 Dimension questionnaire 5 Level (EQ5D), Short Form Health Survey (SF-12), Hemophilia Quality of Life Measure for Adults (HAEMO-QoL-A)
timeFrame: Baseline and at 48 months
measure: Patient Reported Outcome: Change of Quality of Life as measured by EuroQol-5 Dimension questionnaire 5 Level (EQ5D), Short Form Health Survey (SF-12), Hemophilia Quality of Life Measure for Adults (HAEMO-QoL-A)
timeFrame: Baseline and at 60 months
measure: Patient Reported Outcome: Change of treatment patterns as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 12 months
measure: Patient Reported Outcome: Change of treatment patterns as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 24 months
measure: Patient Reported Outcome: Change of treatment patterns as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 36 months
measure: Patient Reported Outcome: Change of treatment patterns as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 48 months
measure: Patient Reported Outcome: Change of treatment patterns as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 60 months
measure: Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 12 months
measure: Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 24 months
measure: Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 36 months
measure: Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 48 months
measure: Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
timeFrame: Baseline and at 60 months
measure: Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
timeFrame: Baseline and at 12 months
measure: Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
timeFrame: Baseline and at 24 months
measure: Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
timeFrame: Baseline and at 36 months
measure: Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
timeFrame: Baseline and at 48 months
measure: Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
timeFrame: Baseline and at 60 months
measure: Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (Short Form) (BPI-SF)
timeFrame: Baseline and at 12 months
measure: Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
timeFrame: Baseline and at 24 months
measure: Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
timeFrame: Baseline and at 36 months
measure: Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
timeFrame: Baseline and at 48 months
measure: Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
timeFrame: Baseline and at 60 months
measure: Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
timeFrame: Baseline and at 12 months
measure: Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
timeFrame: Baseline and at 24 months
measure: Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
timeFrame: Baseline and at 36 months
measure: Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
timeFrame: Baseline and at 48 months
measure: Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
timeFrame: Baseline and at 60 months
measure: Patient Reported outcome: Change of well-being as measured by Psychological General Well Being Schedule (PGWBI) as well as Smoking and drinking behavior and recreational drug use
timeFrame: Baseline and at 12 months
measure: Patient Reported outcome: Change of well-being as measured by Psychological General Well Being Schedule (PGWBI) as well as Smoking and drinking behavior and recreational drug use
timeFrame: Baseline and at 24 months
measure: Patient Reported outcome: Change of well-being as measured by Psychological General Well Being Schedule (PGWBI) as well as Smoking and drinking behavior and recreational drug use
timeFrame: Baseline and at 36 months
measure: Patient Reported outcome: Change of well-being as measured by Psychological General Well Being Schedule (PGWBI) as well as Smoking and drinking behavior and recreational drug use
timeFrame: Baseline and at 48 months
measure: Patient Reported outcome: Change of well-being as measured by Psychological General Well Being Schedule (PGWBI) as well as Smoking and drinking behavior and recreational drug use
timeFrame: Baseline and at 60 months
Secondary outcomes:
measure: Clinical Outcome: Change of Hemophilia A status
timeFrame: Baseline and every 12 months up to 60 months
measure: Clinical Outcome: Change of Joint Status
timeFrame: Baseline and every 12 months up to 60 months
measure: Clinical Outcome: Baseline disease characteristics
timeFrame: Baseline
measure: Clinical Outcome: Change of treatment patterns including Hemophilia treatments via patient chart
timeFrame: Baseline and every 12 months up to 60 months
Other outcomes:
Locations:
city: Phoenix
country: United States
state: Arizona
city: Aurora
country: United States
state: Colorado
city: Washington
country: United States
state: District of Columbia
city: New Orleans
country: United States
state: Louisiana
city: Detroit
country: United States
state: Michigan
city: Minneapolis
country: United States
state: Minnesota
city: Columbus
country: United States
state: Ohio
city: Multiple Locations
country: Japan
city: Multiple Locations
country: Spain
city: Multiple Locations
country: United Kingdom
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References: