The Regional Scintigraphic DPD Uptake in Cardiac Transthyretin Amyloidosis.

ID studio #: NCT05814380

condizione: Amiloidosi da transtiretina

stato: reclutamento

Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis. The disease is caused by misfolding of the liver-derived precursor protein transthyretin as a result of an acquired wild-type variant (ATTRwt) or as a hereditary mutant variant (ATTRm). Application of single-photon emission computed tomography (SPECT) provides greater anatomic resolution, enabling the assessment of amyloid burden within individual left ventricle segments.This study aims to describe the pattern of regional myocardial distribution of 3,3-diphosphono-1,2-propanedicarboxylic acid (DPD) SPECT uptake among patients with ATTRwt and ATTRm. It will investigate the clinical, biochemical and echocardiographic, including left ventricle longitudinal strain profile in ATTRwt and ATTRm. Moreover, we will evaluate the presence and extent of DPD cardiac uptake among asymptomatic ATTRm variants carriers.This is a prospective multi-center observational study. The study, after obtaining prior written informed consent, will include consecutive patients who have Grade 1-3 cardiac DPD retention in scintigraphy. In addition, first-degree relatives of patients with ATTRm are going to be enrolled. Patients are going to undergo TTR gene sequencing to assess the presence of pathogenic variants associated with ATTRm. Both planar scintigraphy, SPECT and speckle-tracking echocardiography will be reviewed and interpreted using visual and quantitative approaches.

intervento: 99mTc-DPD scintigraphy, Collection of blood samples and echocardiography

risultati: https://clinicaltrials.gov/ct2/show/results/NCT05814380

ultimo aggiornamento: Dicembre 28, 2023

data d'inizio: 4 Maggio 2020

completamento previsto: 1 Marzo 2024

ultimo aggiornamento: 19 aprile 2023

taglia / iscrizione: 100

descrizione dello studio: Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis. The disease is caused by misfolding of the liver-derived precursor protein transthyretin as a result of an acquired wild-type variant (ATTRwt) or as a hereditary mutant variant (ATTRm). Application of single-photon emission computed tomography (SPECT) provides greater anatomic resolution, enabling the assessment of amyloid burden within individual left ventricle segments.

This study aims to describe the pattern of regional myocardial distribution of 3,3-diphosphono-1,2-propanedicarboxylic acid (DPD) SPECT uptake among patients with ATTRwt and ATTRm. It will investigate the clinical, biochemical and echocardiographic, including left ventricle longitudinal strain profile in ATTRwt and ATTRm. Moreover, we will evaluate the presence and extent of DPD cardiac uptake among asymptomatic ATTRm variants carriers.

This is a prospective multi-center observational study. The study, after obtaining prior written informed consent, will include consecutive patients who have Grade 1-3 cardiac DPD retention in scintigraphy. In addition, first-degree relatives of patients with ATTRm are going to be enrolled. Patients are going to undergo TTR gene sequencing to assess the presence of pathogenic variants associated with ATTRm. Both planar scintigraphy, SPECT and speckle-tracking echocardiography will be reviewed and interpreted using visual and quantitative approaches.

The collected data will be analyzed statistically to verify research hypotheses. Approval from the local Bioethical Committee will be obtained before carrying out the study. All procedures performed are going to be in accordance with the ethical standards of the 1964 Helsinki declaration and its later amendments, or comparable ethical standards.

risultati primari:

• regional left ventricle 99mTc-DPD uptake
  To compare the regional left ventricle 99mTc-DPD uptake among patients with hereditary and wild-type cardiac transthyretin amyloidosis.
• giorno 1
  

risultati secondari:

• right ventricular 99mTc-DPD accumulation
  giorno 1
• 99mTc-DPD cardiac uptake among asymptomatic hereditary transthyretin amyloidosis variants carriers
  giorno 1

criterio di inclusione:

criteri di esclusione: criteri:

-

sponsor: Katarzyna Holcman

contatti: Katarzyna Holcman, MD, PhD, +48 12 614 22 87, [email protected]

sedi dei centri di prova:

• Poland, Lesser Poland
  Dipartimento di Malattie Cardiache e Vascolari, Ospedale Giovanni Paolo II
  Katarzyna Holcman, MD, PhD, [email protected]