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Completed

NCT Number: NCT02396862

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

16 year and older

Sex eligibility

Male

Study type

Observational

Primary location

Multiple Locations, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)

Drug

Recombinant and Human Factor VIII / Used on demand or prophylaxis of bleeds

Primary outcomes

  1. Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).

    Time frame: Baseline and at 12 months

  2. Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).

    Time frame: Baseline and at 24 months

  3. Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).

    Time frame: Baseline and at 36 months

  4. Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).

    Time frame: Baseline and at 48 months

  5. Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).

    Time frame: Baseline and at 60 months

  6. 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)

    Time frame: Baseline and at 12 months

  7. 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)

    Time frame: Baseline and at 24 months

    Resources: Hospital and Healthcare professional visits

  8. 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)

    Time frame: Baseline and at 36 months

  9. 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)

    Time frame: Baseline and at 48 months

  10. 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)

    Time frame: Baseline and at 60 months

  11. 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)

    Time frame: Baseline and at 12 months

  12. 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)

    Time frame: Baseline and at 24 months

  13. 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)

    Time frame: Baseline and at 36 months

  14. 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)

    Time frame: Baseline and at 48 months

  15. 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)

    Time frame: Baseline and at 60 months

  16. Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)

    Time frame: Baseline and at 12 months

  17. Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)

    Time frame: Baseline and at 24 months

  18. Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)

    Time frame: Baseline and at 36 months

  19. Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)

    Time frame: Baseline and at 48 months

  20. Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)

    Time frame: Baseline and at 60 months

  21. Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)

    Time frame: Baseline and at 12 months

  22. Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)

    Time frame: Baseline and at 24 months

  23. Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)

    Time frame: Baseline and at 36 months

  24. Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)

    Time frame: Baseline and at 48 months

  25. Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)

    Time frame: Baseline and at 60 months

  26. Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (Short Form) (BPI-SF)

    Time frame: Baseline and at 12 months

  27. Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)

    Time frame: Baseline and at 24 months

  28. Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)

    Time frame: Baseline and at 36 months

  29. Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)

    Time frame: Baseline and at 48 months

  30. Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)

    Time frame: Baseline and at 60 months

  31. Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective

    Time frame: Baseline and at 12 months

  32. Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective

    Time frame: Baseline and at 24 months

  33. Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective

    Time frame: Baseline and at 36 months

  34. Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective

    Time frame: Baseline and at 48 months

  35. Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective

    Time frame: Baseline and at 60 months

  36. 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

    Time frame: Baseline and at 12 months

  37. 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

    Time frame: Baseline and at 24 months

  38. 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

    Time frame: Baseline and at 36 months

  39. 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

    Time frame: Baseline and at 48 months

  40. 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

    Time frame: Baseline and at 60 months

Secondary outcomes

  1. Clinical Outcome: Change of Hemophilia A status

    Time frame: Baseline and every 12 months up to 60 months

    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

  2. Clinical Outcome: Change of Joint Status

    Time frame: Baseline and every 12 months up to 60 months

    Joint Status as defined by: - Hemophilia Joint Health Score/ Pettersson Additive Scale - Range of motion - Imaging (where available) - Target joints and past surgical procedures

  3. Clinical Outcome: Baseline disease characteristics

    Time frame: Baseline

    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

  4. Clinical Outcome: Change of treatment patterns including Hemophilia treatments via patient chart

    Time frame: Baseline and every 12 months up to 60 months

    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

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study 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

Acronym: ECHO

Important dates

Study start
2015
Primary completion
2018
Study completion
2018
First posted
Mar 24, 2015
Registry last updated
Mar 27, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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