Summary

Location
at UCLA
Dates
study started
estimated completion

Description

Summary

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 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) mutation

Official Title

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

Keywords

Transthyretin-Mediated Amyloidosis ATTR Amyloidosis RNAi therapeutic Transthyretin TTR Amyloidosis Hereditary Transthyretin-mediated (hATTR) Amyloidosis hATTR amyloidosis Hereditary ATTR amyloidosis Wild-type amyloidosis wtATTR amyloidosis ATTRv amyloidosis ATTRwt amyloidosis Polyneuropathy Familial amyloid polyneuropathies ATTR Transthyretin amyloidosis TTR-mediated amyloidosis Polyneuropathies Amyloid neuropathies Amyloid neuropathies, familial Amyloidosis, familial Pre-symptomatic Carriers

Eligibility

You can join if…

  • Diagnosis of ATTR amyloidosis or documented known disease-causing TTR mutation 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

You CAN'T join if...

  • Current enrollment in a clinical trial for any investigational agent

Locations

  • Clinical Trial Site accepting new patients
    Los Angeles California 90095 United States
  • Clinical Trial Site accepting new patients
    La Jolla California 92037 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alnylam Pharmaceuticals
ID
NCT04561518
Study Type
Observational
Last Updated