Tato stránka byla automaticky přeložena a přesnost překladu není zaručena. Podívejte se prosím na anglická verze pro zdrojový text.

Neintervenční studie pacientů s transthyretinovou (ATTR) amyloidózou (MaesTTRo)

22. září 2026 aktualizováno: AstraZeneca

Neintervenční, prospektivní studie pro více zemí shromažďující údaje z reálného světa o charakteristikách, vzorcích léčby a výsledcích pacientů s transthyretinovou (ATTR) amyloidózou

The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes. In addition, information on the effectiveness of ATTR amyloidosis treatments, including eplontersen, which is a ligand-conjugated antisense oligonucleotide gene silencing treatment targeting activity against both the mutant and wild-type TTR protein, will be collected.

Přehled studie

Postavení

Nábor

Podmínky

Intervence / Léčba

Detailní popis

MaesTTRo is an international, longitudinal, non-interventional study of adult patients with transthyretin (ATTR) amyloidosis.

The study plans to enroll a minimum of 1850 patients with ATTR amyloidosis, including a minimum of 850 patients with ATTR cardiomyopathy (ATTR-CM), and a minimum of 100 patients with ATTRv-PN hereditary polyneuropathy.

The enrollment period is expected to last approximately 4 years. The duration of follow-up for each patient will be at least 3 years and up to 7 years depending on the date when the patient is enrolled.

This study design will include both primary and secondary data. Primary data will consist of patient-reported outcome (PRO) questionnaires. Patients will be asked to complete electronic PRO questionnaires at enrollment and every 6 months (±3 months) only during routine visits. Secondary data will consist of demographic, clinical, and treatment information, and will be collected as per routine clinical practice. These data will be abstracted directly from the electronic health record or review of paper charts for each patient and entered in the electronic data capture system. No site visits are required for this study, and patients will not be contacted for data collection outside of routine clinic visits.

For patients enrolled in the United States, a tokenization process (creation of a unique, encrypted identifier called a token, in place of personal identifiable information) will be used to collect additional de-identified data (e.g., healthcare resource use, healthcare costs) from other sources that are part of patients' routine medical care (electronic medical, hospital, or pharmacy records). Only de-identified data will be analyzed. Patients will be given a choice within the informed consent form to opt in or opt out of participating in the tokenization process.

Typ studie

Pozorovací

Zápis (Odhadovaný)

1850

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní kontakt

Studijní místa

  • Kanada
    • British Columbia
      • Vancouver, British Columbia, Kanada, V5Z 1M9
        • Nábor
        • Research Site
      • Vancouver, British Columbia, Kanada, V6Z 1Y6
        • Nábor
        • Research Site
    • Nova Scotia
      • Halifax, Nova Scotia, Kanada, B3RIV9
        • Nábor
        • Research Site
    • Ontario
      • London, Ontario, Kanada, N6A 5A5
        • Nábor
        • Research Site
      • Toronto, Ontario, Kanada, M2J 4W8
        • Nábor
        • Research Site
    • Quebec
      • Montreal, Quebec, Kanada, H2X 0A9
        • Zatím nenabíráme
        • Research Site
      • Rimouski, Quebec, Kanada, G5L 5T1
        • Zatím nenabíráme
        • Research Site
  • Německo
      • Berlin, Německo, 10117
        • Nábor
        • Research Site
    • Bavaria
      • Würzburg, Bavaria, Německo, DE-97072
        • Zatím nenabíráme
        • Research Site
    • Hesse
      • Frankfurt am Main, Hesse, Německo, 60590
        • Nábor
        • Research Site
    • Lower Saxony
      • Hanover, Lower Saxony, Německo, 30625
        • Nábor
        • Research Site
    • North Rhine-Westphalia
      • Aachen, North Rhine-Westphalia, Německo, 52074
        • Nábor
        • Research Site
      • Cologne, North Rhine-Westphalia, Německo, 50937
        • Nábor
        • Research Site
    • Rhineland-Palatinate
      • Mainz, Rhineland-Palatinate, Německo, 55131
        • Nábor
        • Research Site
    • Saarland
      • Homburg, Saarland, Německo, 66421
        • Nábor
        • Research Site
  • Spojené království
      • Glasgow, Spojené království, G51 4TF
        • Nábor
        • Research Site
      • London, Spojené království, NW3 2QG
        • Nábor
        • Research Site
    • West Midlands
      • Birmingham, West Midlands, Spojené království, B15 2GW
        • Nábor
        • Research Site
  • Spojené státy
    • California
      • La Jolla, California, Spojené státy, 92037
        • Nábor
        • Research Site
      • Los Angeles, California, Spojené státy, 90095
        • Nábor
        • Research Site
      • San Francisco, California, Spojené státy, 94143
        • Nábor
        • Research Site
      • San Francisco, California, Spojené státy, 94025
        • Nábor
        • Research Site
    • Connecticut
      • New Haven, Connecticut, Spojené státy, 06510
        • Nábor
        • Research Site
    • District of Columbia
      • Washington D.C., District of Columbia, Spojené státy, 20010
        • Nábor
        • Research Site
    • Illinois
      • Chicago, Illinois, Spojené státy, 60637
        • Nábor
        • Research Site
    • Indiana
      • Indianapolis, Indiana, Spojené státy, 06200
        • Nábor
        • Research Site
    • Maryland
      • Baltimore, Maryland, Spojené státy, 21218
        • Nábor
        • Research Site
    • Massachusetts
      • Boston, Massachusetts, Spojené státy, 02111
        • Nábor
        • Research Site
      • Boston, Massachusetts, Spojené státy, 02114
        • Zatím nenabíráme
        • Research Site
      • Boston, Massachusetts, Spojené státy, 02115
        • Nábor
        • Research Site
    • Minnesota
      • Rochester, Minnesota, Spojené státy, 55905
        • Nábor
        • Research Site
    • Missouri
      • Kansas City, Missouri, Spojené státy, 64111
        • Nábor
        • Research Site
      • St Louis, Missouri, Spojené státy, 63110
        • Nábor
        • Research Site
    • New Jersey
      • New Brunswick, New Jersey, Spojené státy, 08901
        • Nábor
        • Research Site
    • New York
      • Manhasset, New York, Spojené státy, 11030
        • Nábor
        • Research Site
      • New York, New York, Spojené státy, 10029
        • Nábor
        • Research Site
      • New York, New York, Spojené státy, 10016
        • Zatím nenabíráme
        • Research Site
      • New York, New York, Spojené státy, 10027
        • Nábor
        • Research Site
    • North Carolina
      • Durham, North Carolina, Spojené státy, 27710
        • Nábor
        • Research Site
    • Oregon
      • Portland, Oregon, Spojené státy, 97239
        • Nábor
        • Research Site
    • Pennsylvania
      • Danville, Pennsylvania, Spojené státy, 17822
        • Nábor
        • Research Site
      • Philadelphia, Pennsylvania, Spojené státy, 19104
        • Nábor
        • Research Site
      • Philadelphia, Pennsylvania, Spojené státy, 19107
        • Nábor
        • Research Site
    • South Carolina
      • Greenville, South Carolina, Spojené státy, 29607
        • Nábor
        • Research Site
    • Tennessee
      • Germantown, Tennessee, Spojené státy, 38138
        • Nábor
        • Research Site
      • Nashville, Tennessee, Spojené státy, 037214
        • Nábor
        • Research Site
    • Texas
      • Dallas, Texas, Spojené státy, 75246
        • Nábor
        • Research Site
      • Houston, Texas, Spojené státy, 77030
        • Zatím nenabíráme
        • Research Site
    • Virginia
      • Richmond, Virginia, Spojené státy, 23298
        • Nábor
        • Research Site
    • Washington
      • Seattle, Washington, Spojené státy, 98915
        • Nábor
        • Research Site
    • Wisconsin
      • Milwaukee, Wisconsin, Spojené státy, 53226
        • Nábor
        • Research Site
  • Čína
    • Anhui
      • Hefei, Anhui, Čína, 230001
        • Zatím nenabíráme
        • Research Site
    • Beijing Municipality
      • Beijing, Beijing Municipality, Čína, 100191
        • Nábor
        • Research Site
      • Beijing, Beijing Municipality, Čína, 100730
        • Nábor
        • Research Site
      • Beijing, Beijing Municipality, Čína, 100034
        • Nábor
        • Research Site
      • Beijing, Beijing Municipality, Čína, 100029
        • Nábor
        • Research Site
    • Chongqing Municipality
      • Chongqing, Chongqing Municipality, Čína, 400000
        • Nábor
        • Research Site
    • Fujian
      • Fuzhou, Fujian, Čína, 350005
        • Zatím nenabíráme
        • Research Site
      • Xiamen, Fujian, Čína, 361016
        • Nábor
        • Research Site
    • Guangdong
      • Guangzhou, Guangdong, Čína, 510515
        • Nábor
        • Research Site
      • Guangzhou, Guangdong, Čína, 510080
        • Nábor
        • Research Site
    • Guizhou
      • Guiyang, Guizhou, Čína, 550004
        • Zatím nenabíráme
        • Research Site
    • Hebei
      • Shijiazhuang, Hebei, Čína, 050000
        • Zatím nenabíráme
        • Research Site
    • Hubei
      • Wuhan, Hubei, Čína, 430000
        • Nábor
        • Research Site
      • Wuhan, Hubei, Čína, 430030
        • Nábor
        • Research Site
    • Hunan
      • Changsha, Hunan, Čína, 410013
        • Nábor
        • Research Site
      • Changsha, Hunan, Čína, 410011
        • Nábor
        • Research Site
    • Jiangsu
      • Nanjing, Jiangsu, Čína, 210029
        • Nábor
        • Research Site
      • Suzhou, Jiangsu, Čína, 215006
        • Zatím nenabíráme
        • Research Site
    • Jiangxi
      • Nanchang, Jiangxi, Čína, 330000
        • Nábor
        • Research Site
    • Liaoning
      • Dalian, Liaoning, Čína, 116011
        • Zatím nenabíráme
        • Research Site
    • Shandong
      • Jinan, Shandong, Čína, 250012
        • Nábor
        • Research Site
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, Čína, 200040
        • Nábor
        • Research Site
      • Shanghai, Shanghai Municipality, Čína, 200025
        • Nábor
        • Research Site
      • Shanghai, Shanghai Municipality, Čína, 200031
        • Nábor
        • Research Site
    • Shanxi
      • Taiyuan, Shanxi, Čína, 030001
        • Zatím nenabíráme
        • Research Site
    • Sichuan
      • Chengdu, Sichuan, Čína, 610041
        • Nábor
        • Research Site
      • Chengdu, Sichuan, Čína, 610072
        • Nábor
        • Research Site
    • Xi'An
      • Shaanxi, Xi'An, Čína, 710061
        • Nábor
        • Research Site
    • Zhejiang
      • Hangzhou, Zhejiang, Čína, 310009
        • Zatím nenabíráme
        • Research Site
  • Španělsko
    • Andalusia
      • Huelva, Andalusia, Španělsko, 21005
        • Nábor
        • Research Site
    • Basque Country
      • Bilbao, Basque Country, Španělsko, 48013
        • Nábor
        • Research Site
    • Canary Islands
      • Las Palmas de Gran Canaria, Canary Islands, Španělsko, 35010
        • Nábor
        • Research Site
    • Castille and León
      • Salamanca, Castille and León, Španělsko, 37007
        • Nábor
        • Research Site
    • Catalu A
      • Barcelona, Catalu A, Španělsko, 08035
        • Nábor
        • Research Site
      • Barcelona, Catalu A, Španělsko, 8036
        • Zatím nenabíráme
        • Research Site
      • Cataluna, Catalu A, Španělsko, 08907
        • Zatím nenabíráme
        • Research Site
    • Madrid
      • Majadahonda, Madrid, Španělsko, 28222
        • Nábor
        • Research Site
    • Palma de Mallorca
      • Islas Baleares, Palma de Mallorca, Španělsko, 07198
        • Nábor
        • Research Site

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

18 let až 130 let (Dospělý, Starší dospělý)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Všechno

Metoda odběru vzorků

Vzorek nepravděpodobnosti

Studijní populace

Patients with a diagnosis of amyloid transthyretin (ATTR) amyloidosis

Popis

Inclusion Criteria:

  • Patient willing and able to provide written informed consent to participate in the study
  • Confirmed diagnosis of amyloid transthyretin (ATTR) amyloidosis
  • Aged ≥18 years at the time of signing the informed consent
  • Patient willing and able to participate in collection of electronic patient reported outcomes (PROs)

Exclusion Criteria:

  • Concurrent participation in any interventional trial for ATTR amyloidosis
  • Involvement in the planning and/or conduct of the current study
  • Patients with evidence of primary or light chain amyloidosis (AL) or serum protein A amyloidosis (AA)
  • Asymptomatic patients with ATTR amyloidosis and asymptomatic ATTR mutation carriers

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Observační modely: COHORT
  • Časové perspektivy: PROSPECTIVE

Kohorty a intervence

Skupina / kohorta
Intervence / Léčba
ATTR cardiomyopathy (ATTR-CM)

Patients with ATTR-CM at enrollment
Lék: Treatment of transthyretin (ATTR) amyloidosis in observational study setting

Data will be collected on patients with ATTR amyloidosis in a real-world setting
Hereditary polyneuropathy (ATTRv-PN)

Patients with ATTRv-PN at enrollment
Lék: Treatment of transthyretin (ATTR) amyloidosis in observational study setting

Data will be collected on patients with ATTR amyloidosis in a real-world setting
ATTR-Mixed

Patients with a mixed ATTR amyloidosis phenotype
Lék: Treatment of transthyretin (ATTR) amyloidosis in observational study setting

Data will be collected on patients with ATTR amyloidosis in a real-world setting

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Demographic characteristics (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years
  • Age
  • Sex as determined by the investigator (male/female)
  • Race and ethnicity, where allowed
From time of enrollment for up to 7 years
Treatment patterns (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following treatments will be assessed:

  • ATTR amyloidosis treatment: Tafamidis, Diflunisal, Acoramidis, Vutrisiran, Patisiran, Inotersen, Eplontersen, Doxycycline and taurodesoxycholic acid, Liver transplant
  • Heart failure/arrhythmia-related treatment: Diuretics, Angiotensin converting enzyme inhibitor, Angiotensin receptor blocker, Angiotensin receptor-neprilysin inhibitor, Anticoagulation, Beta-blockers, Sodium-glucose co-transporter-2 inhibitor, Mineralocorticoid receptor antagonist, Digoxin, Pacemaker use, Implantable cardioverter-defibrillator, Left ventricular assist device, Cardiac transplant, Transcatheter aortic valve replacement, Surgical aortic valve replacement
  • Polyneuropathy-related treatment: Antiepileptics (gabapentin, pregabalin, carbamazepine, phenytoin), Antidepressants, Topical pain treatments, Opioids, Tetrahydrocannabinol
  • Other: Medications for gastrointestinal symptoms, Vitamin A supplementation, Dialysis
From time of enrollment for up to 7 years
Clinical characteristics (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following clinical characteristics will be assessed:

  • Modified body mass index (mBMI)
  • Medical history
  • TTR genetic test results
  • Family history of ATTR
  • Time period between the first symptoms to date of diagnosis of ATTR
  • Time since diagnosis of ATTR
From time of enrollment for up to 7 years
Findings from biopsy (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following biopsy information will be collected:

  • Type of biopsy
  • Amyloid identification result (Positive, Negative, Inconclusive for amyloid)
  • Method of amyloid typing
  • Result of the biopsy (Normal/Abnormal)
  • Reason for considering the biopsy result abnormal
From time of enrollment for up to 7 years
Findings from Cardiovascular magnetic resonance imaging (CMR) (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following information will be collected: extracellular volume (ECV), contrast use, left ventricular (LV) end-diastolic volume, LV end-systolic volume, LV ejection fraction, LV Mass Index, interventricular wall thickness, right ventricular Free Wall Thickness, LV Free Wall Thickness, left Atrial Volume Index, native T1 mapping, CMR result (Normal/Abnormal), reason for considering the result abnormal.
From time of enrollment for up to 7 years
Findings from Bone tracer cardiac scintigraphy (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following information will be collected: Type of tracer, Heart to contralateral lung ratio (H/CL), Perugini grade, scintigraphy result (Normal/Abnormal), reason for considering the result abnormal.
From time of enrollment for up to 7 years
Findings from Echocardiography (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following information will be collected: LV ejection fraction, LV End Diastolic Volume, LV End Systolic Volume LV End Diastolic Dimension, LV End Systolic Dimension, Interventricular Septal Thickness End Diastole, Posterial Wall Thickness End Diastole, Left Ventricular Mass Index, Left Atrial Volume, Left Atrial Volume Index, Mitral valve regurgitation, Aortic valve regurgitation, Tricuspid valve regurgitation, Pulmonic valve regurgitation, LV Outflow Gradient, Stroke Volume, Lateral early diastolic myocardial velocity (e' lateral), Medial early diastolic myocardial velocity (e' medial), Mitral E/e' Ratio, Early diastolic mitral inflow velocity (E), Late diastolic mitral inflow velocity (A), Mitral Peak E/A Ratio, Global LV longitudinal strain, Pulmonary artery systolic pressure, RV Free Wall Thickness Severity of Aortic stenosis, Severity of Mitral stenosis.
From time of enrollment for up to 7 years
ECG variables (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following ECG information will be collected:

  • Interpretation of the ECG (Normal/Abnormal/Borderline)
  • Heart rhythm
  • Presence of extrasystoles
  • Presence of conduction abnormalities
  • Evidence of left ventricular hypertrophy (LVH)
From time of enrollment for up to 7 years
Sural nerve and tibial nerve amplitude (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

Sural nerve and tibial nerve amplitude will be measured in overall and in patients initiating a treatment with eplontersen
From time of enrollment for up to 7 years
Biomarker results (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following biomarker results will be collected:

  • Serum TTR levels
  • Complete blood count
  • Hemoglobin
  • Troponin I
  • Cystatin
  • Creatinine
  • Reported glomerular filtration rate (GFR)
  • Albumin
  • Liver enzymes: alkaline phosphatase (ALP), alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma glutamyl transferase (GGT), total bilirubin
  • N-terminal pro B-type natriuretic peptide (NT-proBNP)
  • Vitamin A level
  • Neurofilament light chain (NfL)
  • International normalized ratio (INR) test
From time of enrollment for up to 7 years
Urine test results (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following test results will be collected:

  • Urine albumin-creatinine ratio (UACR)
  • Urine protein creatinine ratio (UPCR)
From time of enrollment for up to 7 years
Clinical manifestations (signs and symptoms) of ATTR amyloidosis (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following clinical manifestations will be assessed:

ischemic heart disease, acute myocardial infarction, heart failure, atrial fibrillation, arrhythmias, conduction system disease, aortic valve stenosis polyneuropathy, carpal tunnel syndrome, autonomic neuropathy, nephrotic syndrome, subnephrotic proteinuria, gastrointestinal dysfunction, chronic kidney disease / acute kidney injury, spinal stenosis, spinal stenosis surgery, hepatomegaly, ascites, oedema, other amyloidosis related manifestations (e.g., Popeye's sign, tendon rupture)
From time of enrollment for up to 7 years
36-Item Short Form Health Survey Version 2 (SF-36v2) Physical Component Summary score (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

The SF-36v2 is a 36-item, generic health survey that provides scores for eight health domains (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional, and mental health) and two summary scores; the physical component summary (PCS) score and the mental component summary (MCS) score. Higher scores indicate a better health state.
From time of enrollment for up to 7 years
Norfolk Quality of Life-Diabetic Neuropathy total score (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

The Norfolk QOL-DN is a 35-item, disease-specific instrument that provides scores for five domains (symptoms, large fiber neuropathy, small fiber neuropathy, autonomic neuropathy, and activities of daily living) and a total score. Higher scores indicate a worse health state.
From time of enrollment for up to 7 years
Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

The KCCQ is a 23-item, disease-specific questionnaire that assesses seven domains (physical limitations, symptom stability, symptom frequency, symptom burden, self-efficacy, quality of life, and social limitation) and provides three summary scores (total symptom score, clinical summary score, and overall summary score). Higher scores indicate a better health state.
From time of enrollment for up to 7 years
New York Heart Association (NYHA) classification (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

I=No symptoms; II=Symptoms with ordinary physical activity; III=Symptoms with less than ordinary physical activity; IV=Symptoms at rest.
From time of enrollment for up to 7 years
National Amyloidosis Centre (NAC) ATTR staging or Mayo staging (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

For NAC staging, Stage I: N-terminal pro-brain natriuretic peptide (NT-proBNP) ≤3000 ng/L and estimated glomerular filtration rate (eGFR) ≥45 ml/min; Stage III: NT-proBNP >3000 ng/L and eGFR <45 ml/min; Stage IV:NT-proBNP ≥10,000 ng/L; Stage II: remainder of patients

For Mayo staging, Stage I: Both and biomarker values are below the established thresholds; Stage II: Either troponin T or NT-proBNP is above the threshold; Stage III: Both troponin T and NT-proBNP biomarker values are above the threshold.

Biomarker Thresholds: Troponin T: >0.05 mg/mL and NT-proBNP: >3000 pg/mL.
From time of enrollment for up to 7 years
Familial amyloid polyneuropathy (FAP) (Coutinho) staging (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

Stage 0: No symptoms; Stage I: Unimpaired ambulation; mostly mild sensory, motor and autonomic neuropathy in the lower limbs; Stage II: Assistance with ambulation required, mostly moderate impairment progression to the lower limbs, upper limbs, and trunk; Stage III: Wheelchair-bound or bedridden; severe sensory, motor, and autonomic involvement of all limbs.
From time of enrollment for up to 7 years
Polyneuropathy disability (PND) score (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

Stage 0=No symptoms; Stage I=Sensory disturbances but preserved walking capabilities; Stage II=Impaired walking capacity, but ability to walk without a stick or crutches; Stage IIIA=Walking with help of 1 stick or crutch; Stage IIIB=Walking with the help of 2 sticks or crutches; Stage IV=confined to wheel chair or bedridden.
From time of enrollment for up to 7 years
Left Ventricular Ejection Fraction (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

Left Ventricular Ejection Fraction will be measured in overall and in patients initiating a treatment with eplontersen
From time of enrollment for up to 7 years
6-minute walk test (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

To address this objective, the distance walked in 6 minutes will be measured.
From time of enrollment for up to 7 years
Charlson comorbidity index (CCI) and CCI components (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address CCI, most recent score and all CCI componenet will be measured

  • Myocardial infarction
  • Congestive heart failure
  • Peripheral vascular disease
  • Cerebrovascular disease
  • Dementia
  • Chronic pulmonary disease
  • Rheumatic disease
  • Peptic ulcer disease
  • Diabetes (None, Without chronic complications, With chronic complications)
  • Hemiplegia or paraplegia
  • Renal disease
  • Any malignancy, including lymphoma and leukemia, except malignant neoplasm of skin (None, Localized, Metastatic)
  • Liver disease (None, Mild, Moderate, Severe)
  • Metastatic solid tumor
  • Acquired immune deficiency syndrome (AIDS) / Human immunodeficiency virus (HIV)
From time of enrollment for up to 7 years
Other comorbidities of interest (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following information will be collected:

  • Percentage of patients with depression
  • Percentage of patients with fibromyalgia
  • Percentage of patients with paraproteinemia
From time of enrollment for up to 7 years
Healthcare resource utilization (overall and in patients initiating a treatment with eplontersen)
Časové okno: From time of enrollment for up to 7 years

In order to address this objective, the following information will be collected:

  • Number of emergency department visits
  • Number of outpatient visits
  • Inpatient care/hospitalization general ward (non-intensive): number of inpatient stays, number of bed days
  • Inpatient care/hospitalization intensive ward: number of inpatient stays, number of bed days
From time of enrollment for up to 7 years
Mortality (overall and in patients initiating a treatment with eplontersen)
Časové okno: Throughout study follow-up (up to 7 years)

Mortality during study follow-up (all-cause, related to ATTR amyloidosis), overall and by NYHA/NAC or Mayo/FAP stage
Throughout study follow-up (up to 7 years)
Liver Disease and Live Transplant
Časové okno: From time of Enrollment for up to 7 Years
  • Liver disease (for patients with mild, moderate or severe liver disease: Child Pugh score, Child Pugh class, ascites, encephalopathy)
  • Liver transplantation (reason for transplant, type of transplant)
From time of Enrollment for up to 7 Years

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Comparison of demographic and clinical characteristics of patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:

  • Demographics
  • mBMI
  • Medical history
  • Family history of ATTR amyloidosis
  • Time period between the first symptoms to date of diagnosis of ATTR amyloidosis
  • Time since diagnosis of ATTR amyloidosis
Up to 7 years
Comparison of findings from biopsy in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:

  • Type of biopsy
  • Amyloid identification result (Positive, Negative, Inconclusive for amyloid)
  • Method of amyloid typing
  • Result of the biopsy (Normal/Abnormal)
  • Reason for considering the biopsy result abnormal
Up to 7 years
Comparison of findings from Cardiovascular magnetic resonance imaging (CMR) in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments: extracellular volume (ECV), contrast use, left ventricular (LV) end-diastolic volume, LV end-systolic volume, LV ejection fraction, LV Mass Index, interventricular wall thickness, right ventricular Free Wall Thickness, LV Free Wall Thickness, left Atrial Volume Index, native T1 mapping, CMR result (Normal/Abnormal), reason for considering the result abnormal.
Up to 7 years
Comparison of findings from Echocardiography in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments: LV ejection fraction, LV End Diastolic Volume, LV End Systolic Volume LV End Diastolic Dimension, LV End Systolic Dimension, Interventricular Septal, Thickness Diastole, Posterial Wall Thickness Diastole, Left Ventricular Mass Index, Left Atrial Volume, Left Atrial Volume Index, Mitral valve regurgitation, Aortic valve regurgitation, Tricuspid valve regurgitation, Pulmonic valve regurgitation, LV Outflow Gradient, Stroke Volume, Lateral early diastolic myocardial velocity (e' lateral), Medial early diastolic myocardial velocity (e' medial), Mitral E/e' Ratio, Early diastolic mitral inflow velocity (E), Late diastolic mitral inflow velocity (A), Mitral Peak E/A Ratio, Global LV longitudinal strain, Pulmonary artery systolic pressure, RV Free Wall Thickness Severity of Aortic stenosis, Severity of Mitral stenosis.
Up to 7 years
Comparison of ECG variables of patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:

  • Interpretation of the ECG (Normal/Abnormal/Borderline)
  • Heart rhythm
  • Presence of extrasystoles
  • Presence of conduction abnormalities
  • Evidence of left ventricular hypertrophy (LVH)
Up to 7 years
Comparison of findings from Bone tracer cardiac scintigraphyin patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments: Type of tracer, Heart to contralateral lung ratio (H/CL), Perugini grade, scintigraphy result (Normal/Abnormal), reason for considering the result abnormal.
Up to 7 years
Comparison of sural nerve and tibial nerve amplitude in patients prescribed eplontersen at any time during the observation eriod to patients on other ATTR treatments
Časové okno: Up to 7 years

Sural nerve and tibial nerve amplitude will be compared in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Up to 7 years
Comparison of biomarker results in patients prescribed eplontersen at any time during the observation eriod to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:

  • Serum TTR levels
  • Complete blood count
  • Hemoglobin
  • Troponin I
  • Cystatin
  • Creatinine
  • Reported glomerular filtration rate (GFR)
  • Albumin
  • Liver enzymes: alkaline phosphatase (ALP), alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma glutamyl transferase (GGT), total bilirubin
  • N-terminal pro B-type natriuretic peptide (NT-proBNP)
  • Vitamin A level
  • Neurofilament light chain (NfL)
  • International normalized ratio (INR) test
Up to 7 years
Comparison of urine test results in patients prescribed eplontersen at any time during the observation eriod to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:

  • Urine albumin-creatinine ratio (UACR)
  • Urine protein creatinine ratio (UPCR)
Up to 7 years
Comparison of Clinical manifestations (signs and symptoms) of ATTR amyloidosis in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments: ischemic heart disease, acute myocardial infarction, heart failure, atrial fibrillation, arrhythmias, conduction system disease, aortic valve stenosis polyneuropathy, carpal tunnel syndrome, autonomic neuropathy, nephrotic syndrome, subnephrotic proteinuria, gastrointestinal dysfunction, chronic kidney disease / acute kidney injury, spinal stenosis, spinal stenosis surgery, hepatomegaly, ascites, oedema, other amyloidosis related manifestations (e.g., Popeye's sign, tendon rupture)
Up to 7 years
Comparison of 36-Item Short Form Health Survey Version 2 (SF-36v2) Physical Component Summary score in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

SF-36v2, physical component summary scores will be compared between patients treated with eplontersen and patients on other ATTR treatments.
Up to 7 years
Comparison of Norfolk Quality of Life-Diabetic Neuropathy total score in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

Norfolk Quality of Life-Diabetic Neuropathy total scores will be compared between patients treated with eplontersen and patients on other ATTR treatments.
Up to 7 years
Comparison of Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score will be compared in patients prescribed eplontersen to patients on other ATTR treatments
Up to 7 years
Comparison of New York Heart Association (NYHA) classification in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

New York Heart Association (NYHA) classification will be compared in patients prescribed eplontersen to patients on other ATTR treatments
Up to 7 years
Comparison of Familial amyloid polyneuropathy (FAP) (Coutinho) staging in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

FAP staging:

Stage 0: No symptoms; Stage I: Unimpaired ambulation; mostly mild sensory, motor and autonomic neuropathy in the lower limbs; Stage II: Assistance with ambulation required, mostly moderate impairment progression to the lower limbs, upper limbs, and trunk; Stage III: Wheelchair-bound or bedridden; severe sensory, motor, and autonomic involvement of all limbs.
Up to 7 years
Comparison of Left Ventricular Ejection Fraction in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

Left Ventricular Ejection Fraction will be compared in patients prescribed eplontersen to patients on other ATTR treatments
Up to 7 years
Comparison of Charlson comorbidity index (CCI) and CCI components in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

Charlson comorbidity index (CCI) and CCI components will be compared in patients prescribed eplontersen to patients on other ATTR treatments
Up to 7 years
Comparison of other comorbidities of interest in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:

  • Percentage of patients with depression
  • Percentage of patients with fibromyalgia
  • Percentage of patients with paraproteinemia
Up to 7 years
Comparison of healthcare resource utilization in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:

  • Number of emergency department visits
  • Number of outpatient visits
  • Inpatient care/hospitalization general ward (non-intensive): number of inpatient stays, number of bed days
  • Inpatient care/hospitalization intensive ward: number of inpatient stays, number of bed days
Up to 7 years
Comparison of mortality in patients prescribed eplontersen to patients on other ATTR treatments
Časové okno: Up to 7 years

Mortality will be compared in patients prescribed eplontersen to patients on other ATTR treatments
Up to 7 years

Další výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Risk factors for worsening ATTR progression
Časové okno: up to 7 years

Factors associated with changes in clinical manifestations of ATTR amyloidosis, NYHA classification, NAC ATTR staging or Mayo staging, FAP (Coutinho) staging, PND score, LVEF, CCI and CCI components, and other comorbidities of interest will be identified.
up to 7 years
Healthcare costs in patients with ATTR amyloidosis
Časové okno: Up to 7 years

Average annual costs will be calculated based on available data in the different countries.
Up to 7 years
Serious adverse events in patients treated with ATTR amyloidosis treatments
Časové okno: Up to 7 years

Serious adverse events in patients treated with ATTR amyloidosis treatments will be measured
Up to 7 years

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

AstraZeneca

Spolupracovníci

ICON plc

Publikace a užitečné odkazy

Osoba odpovědná za zadávání informací o studiu tyto publikace poskytuje dobrovolně. Mohou se týkat čehokoli, co souvisí se studiem.

Obecné publikace

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Aktuální)

25. června 2024

Primární dokončení (Odhadovaný)

31. prosince 2031

Dokončení studie (Odhadovaný)

31. prosince 2031

Termíny zápisu do studia

První předloženo

13. května 2024

První předloženo, které splnilo kritéria kontroly kvality

13. června 2024

První zveřejněno (Aktuální)

20. června 2024

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

23. září 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

22. září 2026

Naposledy ověřeno

1. září 2026

Více informací

Termíny související s touto studií

Klíčová slova

Další relevantní podmínky MeSH

Další identifikační čísla studie

  • D8450R00003

Plán pro data jednotlivých účastníků (IPD)

Plánujete sdílet data jednotlivých účastníků (IPD)?

ANO

Popis plánu IPD

Plan to share only the redacted CSR synopsis

Časový rámec sdílení IPD

AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA/PhRMA Data-Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Kritéria přístupu pro sdílení IPD

When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. A Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Typ podpůrných informací pro sdílení IPD

  • CSR

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ne

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .

Předplatit