NCT02396862

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
272

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Dec 2015

Typical duration for all trials

Geographic Reach
4 countries

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 19, 2015

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 24, 2015

Completed
9 months until next milestone

Study Start

First participant enrolled

December 9, 2015

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 17, 2018

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 26, 2018

Completed
Last Updated

March 27, 2019

Status Verified

March 1, 2019

Enrollment Period

2.1 years

First QC Date

March 19, 2015

Last Update Submit

March 26, 2019

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (40)

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

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

    Baseline and at 12 months

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

    Baseline and at 24 months

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

    Baseline and at 36 months

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

    Baseline and at 48 months

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

    Baseline and at 60 months

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

    Baseline and at 12 months

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

    Baseline and at 24 months

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

    Baseline and at 36 months

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

    Baseline and at 48 months

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

    Baseline and at 60 months

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

    Baseline and at 12 months

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

    Baseline and at 24 months

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

    Baseline and at 36 months

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

    Baseline and at 48 months

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

    Baseline and at 60 months

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

    Baseline and at 12 months

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

    Baseline and at 24 months

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

    Baseline and at 36 months

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

    Baseline and at 48 months

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

    Baseline and at 60 months

  • 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

  • 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

  • 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

  • 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

  • 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 Outcomes (4)

  • Clinical Outcome: Change of Hemophilia A status

    Baseline and every 12 months up to 60 months

  • Clinical Outcome: Change of Joint Status

    Baseline and every 12 months up to 60 months

  • Clinical Outcome: Baseline disease characteristics

    Baseline

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

    Baseline and every 12 months up to 60 months

Study Arms (1)

Patients with moderate to severe Hemophilia A / Cohort 1

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%)

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

Interventions

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

Patients with moderate to severe Hemophilia A / Cohort 1

Eligibility Criteria

Age16 Years+
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability 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%)

You may qualify if:

  • 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.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Unknown Facility

Phoenix, Arizona, United States

Location

Unknown Facility

Aurora, Colorado, United States

Location

Unknown Facility

Washington D.C., District of Columbia, United States

Location

Unknown Facility

New Orleans, Louisiana, United States

Location

Unknown Facility

Detroit, Michigan, United States

Location

Unknown Facility

Minneapolis, Minnesota, United States

Location

Unknown Facility

Columbus, Ohio, United States

Location

Unknown Facility

Multiple Locations, Japan

Location

Unknown Facility

Multiple Locations, Spain

Location

Unknown Facility

Multiple Locations, United Kingdom

Location

Related Links

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIIIBAY 14-2222

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Study Officials

  • Bayer Study Director

    Bayer

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 19, 2015

First Posted

March 24, 2015

Study Start

December 9, 2015

Primary Completion

January 17, 2018

Study Completion

March 26, 2018

Last Updated

March 27, 2019

Record last verified: 2019-03

Locations

US(7)GB(1)JP(1)ES(1)