Comprehensive Program for Hereditary Transthyretin Amyloidosis

The Comprehensive Program for Hereditary Transthyretin Amyloidosis describes a prospective observational study focused on understanding hereditary transthyretin amyloidosis (ATTR), a progressive and potentially fatal condition marked by amyloid fibril deposits impacting multiple organs. The trial aims to characterize patient phenotypes, investigate factors affecting disease progression, and identify minimum criteria for disease onset. Conducted at Néstor Kirchner Hospital, the trial enrolls participants over 18 years old with confirmed pathogenic TTR variants. It includes thorough evaluations such as genetic testing sponsored by pharmaceutical companies, clinical assessments, and diverse diagnostic tests.

NCT Number	NCT07213297
Lead Sponsor	Hospital de Alta Complejidad en Red
Conditions	Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial
Enrollment	20 participants
Start Date	2025-11-01
Primary Completion	2028-11-01 (estimated)
Study Completion	2028-12-01 (estimated)
Updated on ClinicalTrials.gov	2026-03-24