Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers

Pfizer

Status: Completed

Conditions: Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP); Senile Systemic Amyloidosis (SSA); Transthyretin Cardiomyopathy (TTR-CM); Familial Amyloid Cardiomyopathy

Treatments: Other: No drug

Study type: Observational

Funder types: Industry

Identifiers

NCT01604122

B3461036

Details and patient eligibility

About

This study is an online (web-based) or paper-based survey for patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) and caregivers. The results will be used to describe the emotional, physical, and financial impact of having TTR-FAP or caring for someone who has the disease.

Full description

Convenience sample of patients and caregivers recruited through patient advocacy groups. Convenience sample.

Enrollment: 1 patient

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Adults (ages 18-85 years inclusive) diagnosed with TTR-FAP or TTR-CM and experiencing symptoms or currently providing care for a patient with TTR-FAP or TTR-FAP

Exclusion criteria

• Caregivers who formally care for a TTR-FAP or TTR-CM patient as part of their job description and receive payment for their services.

Trial design

1 participants in 2 patient groups

Patients

Description:

Patients with TTR-FAP or TTR-CM. No drug will be administered; this is a non-interventional observational study.

Treatment:

Other: No drug

Other: No drug

Caregivers

Description:

Caregivers who are taking care of patients with TTR-FAP or TTR-CM. No drug will be administered; this is a non-interventional observational study.

Treatment:

Other: No drug

Other: No drug