An observational study in Amyloidosis, sponsored by Fondazione Policlinico Universitario Agostino Gemelli IRCCS. Status unknown at 1 site in Italy. Per ClinicalTrials.gov, last updated 2023-03-31.
Sponsored by Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Observational
The objective of this prospective observational study is to periodically monitor pre-symptomatic subjects carrying a mutation of Transthyretin (TTR), identified in the context of a family screening of affected proband, through instrumental methods and clinical scales in order to identify the first signs of clinically significant organ involvement by the disease. Healthy asymptomatic carriers will be subjected to regular monitoring through clinical evaluations and instrumental investigations defined by the consensus group (Conceicao et al.) in order to validate the criteria defined by this group to define the onset of the disease. A subgroup of carriers with scales and instrumental tests negative for damage to the peripheral nervous system or cardiac, but with subjective symptoms compatible with the disease, will be subjected to further instrumental tests not indicated by consent.
491 studies on the registry are indexed under Amyloidosis; 135 are open to participants now.
This study's planned enrollment of 20 is below the median of 141 across 156 observational studies indexed under Amyloidosis.
Browse Amyloidosis studies →Fondazione Policlinico Universitario Agostino Gemelli IRCCS is the lead sponsor of 920 studies on the registry; 529 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Data from pre-symptomatic carriers who have signed the written informed consent, in regular follow-up at the Centres, will be included in the study. Carriers recruited should be no less than 10 years of age compared to the age of onset of the affected relative with the youngest age of onset or should have a history of bilateral carpal tunnel syndrome undergoing surgery.
Exclusion criteria
Evaluation of a cohort of pre-symptomatic subjects carrying a TTR mutation
To evaluate in a cohort of pre-symptomatic subjects carrying a TTR mutation the disease onset according to the European consensus criteria (Conceicao et al.).
Time frame: 3 years
This study is status unknown, as verified in Feb 2023. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Fondazione Policlinico Universitario Agostino Gemelli IRCCS