ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

Alnylam Pharmaceuticals

Status: Enrolling

Conditions: ATTR Amyloidosis; Transthyretin-Mediated Amyloidosis

Study type: Observational

Funder types: Industry

Identifiers

NCT04561518

ALN-TTR02-013

Details and patient eligibility

About

The purpose of this study is to:

• Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients
• Characterize the safety and effectiveness of patisiran and vutrisiran as part of routine clinical practice in the real-world clinical setting
• Describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant

Enrollment: 1,500 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of ATTR amyloidosis or documented known disease-causing TTR variant for the cohort of pre-symptomatic carriers
• Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis

Exclusion criteria

• Current enrollment in a clinical trial for any investigational agent

Trial design

1,500 participants in 2 patient groups

Patients with ATTR amyloidosis

Description:

Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, will be eligible for the study and will follow routine clinical care.

Pre-symptomatic Carriers

Description:

Pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for the study and will follow routine clinical care.