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

Bayer

Status: Completed

Conditions: Hemophilia A

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

Study type: Observational

Funder types: Industry

Identifiers

NCT02396862

KG1301 (Other Identifier)

17285

Details and patient eligibility

About

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.

Enrollment: 272 patients

Sex: Male

Ages: 16+ years old

Volunteers: No Healthy Volunteers

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

Trial design

272 participants in 1 patient group

Patients with moderate to severe Hemophilia A / Cohort 1

Description:

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%)

Treatment:

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