Amyloidosis in Transthyretin (TTR)NCT07080268
Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis.
An observational study of Amyloidosis in Transthyretin (TTR) and Hereditary Amyloidosis, Transthyretin-Related, run by Hospital General de Niños Pedro de Elizalde at 1 site in Argentina.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis in Transthyretin (TTR), Hereditary Amyloidosis, Transthyretin-Related
- How many people
- 0 participants
- Where
- 1 siteArgentina
- Study dates
- 20 Jun 2025to 30 Dec 2025
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with confirmed ATTR (genetic test), older than 18 years in stage I, II and III of the PND (polyneuropathy disability) scale
Who may be able to join
- 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).
Who cannot join
- patients who participated in the validation process of the questionnaire: patients who participated in the questionnaire construction process or adjustments to the questionnaire.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis in Argentina.
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:
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Symptomatic patients with hereditary amyloidosis | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
To develop and validate a structured and self-administered quality of life questionnaire in patients with hereditary amyloidosis
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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2025
- Study start20 Jun 2025
- Primary completion1 Sep 2025 (estimated)
- Study completion30 Dec 2025 (estimated)
Why it stopped: Study withdrawn before enrollment due to protocol and consent-related issues identified during the study preparation phase.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Amyloidosis in Transthyretin (TTR)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital General de Niños Pedro de Elizalde
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fernando Torres, Hospital General de Niños Pedro de Elizalde
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07080268
- Sponsor's protocol ID
- HGNPE
- Status last verified
- Sep 2026
- First submitted
- 15 Jul 2025
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Conceicao I, Coelho T, Rapezzi C, Parman Y, Obici L, Galan L, Rousseau A. Assessment of patients with hereditary transthyretin amyloidosis - understanding the impact of management and disease progression. Amyloid. 2019 Sep;26(3):103-111. doi: 10.1080/13506129.2019.1627312. Epub 2019 Jul 24.
PubMed 31339362Aimo A, Rapezzi C, Perfetto F, Cappelli F, Palladini G, Obici L, Merlini G, Di Bella G, Serenelli M, Zampieri M, Milani P, Licordari R, Teresi L, Ribarich N, Castiglione V, Quattrone F, De Rosis S, Vergaro G, Panichella G, Emdin M, Passino C. Quality of life assessment in amyloid transthyretin (ATTR) amyloidosis. Eur J Clin Invest. 2021 Nov;51(11):e13598. doi: 10.1111/eci.13598. Epub 2021 May 22.
PubMed 33982288