check_circleStudy Completed

Hemophilia A

Bayer Identifier:

17285

ClinicalTrials.gov Identifier:

NCT02396862

EudraCT Number:

Not Available

EU CT Number:

Not Available
Patient functioning and well-being, economic, and clinical impact of hemophilia A and its treatment

Trial purpose

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.

Key Participants Requirements

Sex

Male

Age

16 - N/A
  • - 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.


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

Trial summary

Enrollment Goal
272
Trial Dates
December 2015 - March 2018
Phase
Phase 4
Could I Receive a placebo
No
Products
Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Completed
Many Locations, United Kingdom
Withdrawn
Many Locations, Germany
Completed
Many Locations, Japan
Withdrawn
Many Locations, Italy
Withdrawn
Many Locations, Canada
Withdrawn
Many Locations, Netherlands
Withdrawn
Many Locations, China
Completed
Detroit, United States
Completed
Many Locations, Spain
Withdrawn
Many Locations, Mexico
Completed
Columbus, United States
Completed
New Orleans, United States
Completed
Aurora, United States
Completed
Phoenix, United States
Completed
Washington, United States
Completed
Minneapolis, United States
Withdrawn
Palo Alto, United States
Withdrawn
Orange, United States
Withdrawn
Many Locations, Taiwan

Primary Outcome

  • Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
    date_rangeTime Frame:
    Baseline and at 12 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
    date_rangeTime Frame:
    Baseline and at 24 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
    date_rangeTime Frame:
    Baseline and at 36 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
    date_rangeTime Frame:
    Baseline and at 48 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
    date_rangeTime Frame:
    Baseline and at 60 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 12 months
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline and at 24 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 36 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 48 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 60 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 12 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 24 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 36 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 48 months
    enhanced_encryption
    Safety Issue:
    No
  • 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)
    date_rangeTime Frame:
    Baseline and at 60 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
    date_rangeTime Frame:
    Baseline and at 12 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
    date_rangeTime Frame:
    Baseline and at 24 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
    date_rangeTime Frame:
    Baseline and at 36 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
    date_rangeTime Frame:
    Baseline and at 48 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
    date_rangeTime Frame:
    Baseline and at 60 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
    date_rangeTime Frame:
    Baseline and at 12 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
    date_rangeTime Frame:
    Baseline and at 24 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
    date_rangeTime Frame:
    Baseline and at 36 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
    date_rangeTime Frame:
    Baseline and at 48 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
    date_rangeTime Frame:
    Baseline and at 60 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (Short Form) (BPI-SF)
    date_rangeTime Frame:
    Baseline and at 12 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
    date_rangeTime Frame:
    Baseline and at 24 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
    date_rangeTime Frame:
    Baseline and at 36 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
    date_rangeTime Frame:
    Baseline and at 48 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
    date_rangeTime Frame:
    Baseline and at 60 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
    date_rangeTime Frame:
    Baseline and at 12 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
    date_rangeTime Frame:
    Baseline and at 24 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
    date_rangeTime Frame:
    Baseline and at 36 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
    date_rangeTime Frame:
    Baseline and at 48 months
    enhanced_encryption
    Safety Issue:
    No
  • Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
    date_rangeTime Frame:
    Baseline and at 60 months
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline and at 12 months
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline and at 24 months
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline and at 36 months
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline and at 48 months
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline and at 60 months
    enhanced_encryption
    Safety Issue:
    No

Secondary Outcome

  • 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
    date_rangeTime Frame:
    Baseline and every 12 months up to 60 months
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline and every 12 months up to 60 months
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline
    enhanced_encryption
    Safety Issue:
    No
  • 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
    date_rangeTime Frame:
    Baseline and every 12 months up to 60 months
    enhanced_encryption
    Safety Issue:
    No

Trial design

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
Trial Type
Observational
Intervention Type
Drug
Trial Purpose
N/A
Allocation
N/A
Blinding
N/A
Assignment
N/A
Trial Arms
N/A

Additional Information

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