Amyloidosis, Hereditary

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Information
Disease name
Amyloidosis, Hereditary
Disease ID
Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
Genes Mutation Description Source Links
NCT ID Status Phase Summary Start date Completion date
NCT03759379 Active, not recruiting Phase 3 HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) February 14, 2019 October 2026
NCT03400098 Approved for marketing ATTR Expanded Access Program (EAP) by Ionis
NCT02939820 Approved for marketing Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
NCT01960348 Completed Phase 3 APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis November 2013 August 2017
NCT02319005 Completed Phase 3 ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) December 2014 March 30, 2017
NCT05974644 Not yet recruiting Southeastern ATTR Amyloidosis Consortium: SEATTRAC Family Registry September 1, 2024 December 1, 2030
NCT05489549 Recruiting Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers November 21, 2022 June 30, 2027
NCT05873868 Recruiting Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran April 12, 2024 July 2026
NCT05879874 Recruiting Prospective Evaluation of NfL as a Biomarker in ATTRv September 1, 2023 June 2026