Can a new questionnaire capture life with hereditary amyloidosis?
NCT ID NCT07080268
First seen Sep 17, 2026 · Last updated Sep 18, 2026 · Updated 1 time
Summary
Researchers in Argentina are developing and testing a self-administered questionnaire to measure how hereditary transthyretin amyloidosis affects patients' physical, emotional, and social quality of life. The study includes input from neurologists, a cardiologist, a psychologist, a social worker, and patients with confirmed hereditary amyloidosis. Adults over 18 with confirmed ATTR and varying levels of disability from polyneuropathy can take part. The goal is to create a validated tool that reflects the specific experiences of patients living in Argentina.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If validated, the questionnaire could give doctors in Argentina a standard way to track how hereditary amyloidosis affects patients' daily lives and to compare treatments or care strategies.
- What could go wrong
- This is a small, early validation study limited to Argentina, so the questionnaire may not work well in other countries or languages. It measures quality of life, not whether a treatment works.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Started
-
Jun 2025
- Expected to finish
-
Dec 2025
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Patients with confirmed ATTR (genetic test), older than 18 years in stage I, II and III of the PND (polyneuropathy disability) scale
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * patients with confirmed ATTR (genetic test), older than 18 years old in stage I, II and III of the PND scale (polyneuropathy disability: I Sensory neuropathy ; II Motor symptoms, but with preserved ambulation; IIIa Requires 1 cane to ambulate; IIIb requires two canes/walker). Exclusion Criteria: * patients who participated in the validation process of the questionnaire: patients who participated in the questionnaire construction process or adjustments to the questionnaire.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Amyloidosis in transthyretin (TTR) are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
HGNPE
Buenos Aires, Buenos Aires, 1752, Argentina
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a daily pill keep a rare heart disease in check for the long haul?
- Rare gene variant under the microscope: new study aims to map disease patterns
- Blood marker may predict nerve damage onset in rare genetic disease
- Blood test and imaging aim to spot nerve damage before symptoms worsen
- New study aims to unravel mysteries of rare genetic disease