Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis.

Prospective study of development and validation of a questionnaire on quality of life in patients with Hereditary Amyloidosis The questionnaire development process will include 4 medical specialists experts in Transthyretin Amyloidosis (2 neurologists, 1 cardiologist, 1 physician clinical/hepathologist), 1 psychologist; 1 social worker; and 3 patients (with confirmed Hereditary amyloidosis) members of ALAPA. (Local PAG)

Population and sample:

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Key information

Conditions

Amyloidosis in Transthyretin (TTR) Amyloidosis, Hereditary, Transthyretin-Related Hereditary Amyloidosis, Transthyretin-Related

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

HGNPE

Buenos Aires, 1752, Argentina

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

To develop and validate a structured and self-administered quality of life questionnaire in patients with hereditary amyloidosis

Time frame: 6 months

To develop and validate a structured and self-administered questionnaire specific to covering physical, emotional and social aspects, to assess the impact of hereditary transthyretin amyloidosis on the quality of life of patients residing in Argentina.

Sponsors and collaborators

Lead sponsor

Hospital General de Niños Pedro de Elizalde

Other

Registry information

Official study title

Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis in Argentina.

Important dates

Study start: 2025
Primary completion: 2025
Study completion: 2025
First posted: Jul 23, 2025
Registry last updated: Jul 23, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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