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

Hemophilia A Clinical Trial

— ECHO  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02396862
• Other study ID #: 17285
• Secondary ID: KG1301
• Status: Completed
• Start date: December 9, 2015
• Est. completion date: March 26, 2018

Study information

• Verified date: March 2019
• Source: Bayer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 272
• Est. completion date: March 26, 2018
• Est. primary completion date: January 17, 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 16 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Recombinant and Human Factor VIII / Used on demand or prophylaxis of bleeds

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Countries where clinical trial is conducted

United States,  Japan,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).		Baseline and at 12 months
Primary	Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).		Baseline and at 24 months
Primary	Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).		Baseline and at 36 months
Primary	Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).		Baseline and at 48 months
Primary	Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).		Baseline and at 60 months
Primary	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)		Baseline and at 12 months
Primary	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)	Resources: Hospital and Healthcare professional visits	Baseline and at 24 months
Primary	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)		Baseline and at 36 months
Primary	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)		Baseline and at 48 months
Primary	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)		Baseline and at 60 months
Primary	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)		Baseline and at 12 months
Primary	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)		Baseline and at 24 months
Primary	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)		Baseline and at 36 months
Primary	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)		Baseline and at 48 months
Primary	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)		Baseline and at 60 months
Primary	Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)		Baseline and at 12 months
Primary	Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)		Baseline and at 24 months
Primary	Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)		Baseline and at 36 months
Primary	Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)		Baseline and at 48 months
Primary	Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)		Baseline and at 60 months
Primary	Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)		Baseline and at 12 months
Primary	Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)		Baseline and at 24 months
Primary	Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)		Baseline and at 36 months
Primary	Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)		Baseline and at 48 months
Primary	Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)		Baseline and at 60 months
Primary	Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (Short Form) (BPI-SF)		Baseline and at 12 months
Primary	Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)		Baseline and at 24 months
Primary	Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)		Baseline and at 36 months
Primary	Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)		Baseline and at 48 months
Primary	Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)		Baseline and at 60 months
Primary	Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective		Baseline and at 12 months
Primary	Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective		Baseline and at 24 months
Primary	Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective		Baseline and at 36 months
Primary	Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective		Baseline and at 48 months
Primary	Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective		Baseline and at 60 months
Primary	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		Baseline and at 12 months
Primary	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		Baseline and at 24 months
Primary	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		Baseline and at 36 months
Primary	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		Baseline and at 48 months
Primary	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		Baseline and at 60 months
Secondary	Clinical Outcome: Change of Hemophilia A status	Hemophilia A status as defined by: - Last measured FVIII level - History of intracranial hemorrhage and age of occurrence - Number of Bleeding episodes/events in the last year - Location of bleed - Confirmed microbleeds on past imaging - PK assessment (if available): Name of FVIII product for most recent PK evaluation - Dose of Factor VIII product administered for most recent PK evaluation - Measured Factor VIII level - Factor VIII half-life and time point of measurement -- Central Venous Access Device (CVAD) or AV (arterial-venous) fistulae	Baseline and every 12 months up to 60 months
Secondary	Clinical Outcome: Change of Joint Status	Joint Status as defined by: - Hemophilia Joint Health Score/ Pettersson Additive Scale - Range of motion - Imaging (where available) - Target joints and past surgical procedures	Baseline and every 12 months up to 60 months
Secondary	Clinical Outcome: Baseline disease characteristics	Baseline disease characteristics and disease course as defined by: - Date of diagnosis of Hemophilia A - Age at first treatment with factor replacement therapy (child/adult) - Type of factor VIII gene mutation (if available) - Factor VIII level at initial diagnosis (if available) - History of inhibitor and assay used for detection - Date of determination of peak level of inhibitor	Baseline
Secondary	Clinical Outcome: Change of treatment patterns including Hemophilia treatments via patient chart	Treatment patterns as measured by: - Treatment choice and dose (Factor VIII use and type) - Treatment Regimen - Immune tolerance therapy - Products for inhibitors - Blood bank products - Non plasma and topical products	Baseline and every 12 months up to 60 months

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