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

Active, not recruiting

• Conditions: Amyloidosis in Transthyretin (TTR); Hereditary Amyloidosis, Transthyretin-Related

• Registration Number: NCT07080268
• Lead Sponsor: Hospital General de Niños Pedro de Elizalde

Brief Summary

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:

Detailed Description

Not available

Study Timeline

• Study Start: 2025-06-20
• Status Verified: 2025-07
• Study First Submitted: 2025-07-15
• Study First Submitted QC: 2025-07-15
• Last Update Submitted: 2025-07-15
• Study First Posted: 2025-07-23
• Last Update Posted: 2025-07-23
• Primary Completion: 2025-09-01
• Study Completion: 2025-12-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 70

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

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

Trial Locations

Locations (1)

HGNPE; 🇦🇷 Buenos Aires, Argentina