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| Name | Class |
|---|---|
| ICON plc | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ATTR cardiomyopathy (ATTR-CM) | Patients with ATTR-CM at enrollment |
| |
| Hereditary polyneuropathy (ATTRv-PN) | Patients with ATTRv-PN at enrollment |
| |
| ATTR-Mixed | Patients with a mixed ATTR amyloidosis phenotype |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Treatment of transthyretin (ATTR) amyloidosis in observational study setting | Drug | Data will be collected on patients with ATTR amyloidosis in a real-world setting |
|
| Measure | Description | Time Frame |
|---|---|---|
| Demographic characteristics (overall and in patients initiating a treatment with eplontersen) |
| From time of enrollment for up to 7 years |
| Treatment patterns (overall and in patients initiating a treatment with eplontersen) | In order to address this objective, the following treatments will be assessed:
| From time of enrollment for up to 7 years |
| Clinical characteristics (overall and in patients initiating a treatment with eplontersen) | In order to address this objective, the following clinical characteristics will be assessed:
| From time of enrollment for up to 7 years |
| Findings from biopsy (overall and in patients initiating a treatment with eplontersen) | In order to address this objective, the following biopsy information will be collected:
|
| Measure | Description | Time Frame |
|---|---|---|
| Comparison of demographic and clinical characteristics of patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments | The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:
|
| Measure | Description | Time Frame |
|---|---|---|
| Risk factors for worsening ATTR progression | 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 |
Inclusion Criteria:
Exclusion Criteria:
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Patients with a diagnosis of amyloid transthyretin (ATTR) amyloidosis
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| AstraZeneca Clinical Study Information Center | Contact | 1-877-240-9479 | information.center@astrazeneca.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Recruiting | La Jolla | California | 92037 | United States | |
| Research Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40108078 | Derived | Gillmore JD, Hahn K, Smith JG, Conceicao I, Tian Z, Grogan M, Pao C, Wittbrodt E, Jarbrink K, Papas MA, Davis MK. Rationale and Design of ANTHOLOGY: An ATTR Amyloidosis Real-World Evidence Program Aiming to Address Gaps in Amyloidosis Care. Cardiol Ther. 2025 Sep;14(3):477-490. doi: 10.1007/s40119-025-00402-y. Epub 2025 Mar 19. |
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Plan to share only the redacted CSR synopsis
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.
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.
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| 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) | 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) | 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) | 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) | In order to address this objective, the following ECG information will be collected:
| From time of enrollment for up to 7 years |
| Sural nerve and tibial nerve amplitude (overall and in patients initiating a treatment with eplontersen) | 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) | In order to address this objective, the following biomarker results will be collected:
| From time of enrollment for up to 7 years |
| Urine test results (overall and in patients initiating a treatment with eplontersen) | In order to address this objective, the following test results will be collected:
| 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) | 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) | 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) | 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) | 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) | 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) | 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) | 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) | 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) | 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) | 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) | In order to address CCI, most recent score and all CCI componenet will be measured
| From time of enrollment for up to 7 years |
| Other comorbidities of interest (overall and in patients initiating a treatment with eplontersen) | In order to address this objective, the following information will be collected:
| From time of enrollment for up to 7 years |
| Healthcare resource utilization (overall and in patients initiating a treatment with eplontersen) | In order to address this objective, the following information will be collected:
| From time of enrollment for up to 7 years |
| Mortality (overall and in patients initiating a treatment with eplontersen) | 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 |
| From time of Enrollment for up to 7 Years |
| 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 | The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:
| 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 | 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 | 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 | The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:
| 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 | 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 | 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 | The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:
| 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 | The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:
| Up to 7 years |
| Comparison of Clinical manifestations (signs and symptoms) of ATTR amyloidosis in patients prescribed eplontersen to patients on other ATTR treatments | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:
| Up to 7 years |
| Comparison of healthcare resource utilization in patients prescribed eplontersen to patients on other ATTR treatments | The following outcomes will be compared between patients treated with eplontersen and patients on other ATTR treatments:
| Up to 7 years |
| Comparison of mortality in patients prescribed eplontersen to patients on other ATTR treatments | Mortality will be compared in patients prescribed eplontersen to patients on other ATTR treatments | Up to 7 years |
| Healthcare costs in patients with ATTR amyloidosis |
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 | Serious adverse events in patients treated with ATTR amyloidosis treatments will be measured | Up to 7 years |
| Recruiting |
| Los Angeles |
| California |
| 90095 |
| United States |
| Research Site | Recruiting | San Francisco | California | 94025 | United States |
| Research Site | Recruiting | San Francisco | California | 94143 | United States |
| Research Site | Recruiting | New Haven | Connecticut | 06510 | United States |
| Research Site | Recruiting | Washington D.C. | District of Columbia | 20010 | United States |
| Research Site | Recruiting | Chicago | Illinois | 60637 | United States |
| Research Site | Recruiting | Indianapolis | Indiana | 06200 | United States |
| Research Site | Recruiting | Boston | Massachusetts | 02111 | United States |
| Research Site | Not yet recruiting | Boston | Massachusetts | 02114 | United States |
| Research Site | Recruiting | Boston | Massachusetts | 02115 | United States |
| Research Site | Recruiting | Rochester | Minnesota | 55905 | United States |
| Research Site | Recruiting | Kansas City | Missouri | 64111 | United States |
| Research Site | Recruiting | St Louis | Missouri | 63110 | United States |
| Research Site | Recruiting | New Brunswick | New Jersey | 08901 | United States |
| Research Site | Recruiting | Manhasset | New York | 11030 | United States |
| Research Site | Recruiting | New York | New York | 10027 | United States |
| Research Site | Recruiting | New York | New York | 10029 | United States |
| Research Site | Recruiting | Durham | North Carolina | 27710 | United States |
| Research Site | Not yet recruiting | Portland | Oregon | 97239 | United States |
| Research Site | Recruiting | Danville | Pennsylvania | 17822 | United States |
| Research Site | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| Research Site | Recruiting | Philadelphia | Pennsylvania | 19107 | United States |
| Research Site | Recruiting | Greenville | South Carolina | 29607 | United States |
| Research Site | Recruiting | Germantown | Tennessee | 38138 | United States |
| Research Site | Recruiting | Nashville | Tennessee | 037214 | United States |
| Research Site | Recruiting | Dallas | Texas | 75246 | United States |
| Research Site | Recruiting | Richmond | Virginia | 23298 | United States |
| Research Site | Recruiting | Seattle | Washington | 98915 | United States |
| Research Site | Recruiting | Milwaukee | Wisconsin | 53226 | United States |
| Research Site | Recruiting | Vancouver | British Columbia | V5Z 1M9 | Canada |
| Research Site | Recruiting | Vancouver | British Columbia | V6Z 1Y6 | Canada |
| Research Site | Recruiting | Halifax | Nova Scotia | B3RIV9 | Canada |
| Research Site | Recruiting | London | Ontario | N6A 5A5 | Canada |
| Research Site | Recruiting | Toronto | Ontario | M2J 4W8 | Canada |
| Research Site | Not yet recruiting | Rimouski | Quebec | G5L 5T1 | Canada |
| Research Site | Not yet recruiting | Beijing | Beijing Municipality | 100029 | China |
| Research Site | Recruiting | Beijing | Beijing Municipality | 100034 | China |
| Research Site | Recruiting | Beijing | Beijing Municipality | 100191 | China |
| Research Site | Recruiting | Beijing | Beijing Municipality | 100730 | China |
| Research Site | Not yet recruiting | Fuzhou | Fujian | 350005 | China |
| Research Site | Not yet recruiting | Guangzhou | Guangdong | 510080 | China |
| Research Site | Not yet recruiting | Guangzhou | Guangdong | 510515 | China |
| Research Site | Not yet recruiting | Wuhan | Hubei | 430030 | China |
| Research Site | Recruiting | Changsha | Hunan | 410013 | China |
| Research Site | Recruiting | Nanjing | Jiangsu | 210029 | China |
| Research Site | Not yet recruiting | Suzhou | Jiangsu | 215006 | China |
| Research Site | Recruiting | Nanchang | Jiangxi | 330000 | China |
| Research Site | Not yet recruiting | Jinan | Shandong | 250012 | China |
| Research Site | Recruiting | Shanghai | Shanghai Municipality | 200040 | China |
| Research Site | Not yet recruiting | Taiyuan | Shanxi | 30001 | China |
| Research Site | Not yet recruiting | Hebei Sheng | Shijiazhuang | 050000 | China |
| Research Site | Recruiting | Chengdu | Sichuan | 610072 | China |
| Research Site | Recruiting | Shaanxi | Xi'An | 710061 | China |
| Research Site | Not yet recruiting | Würzburg | Bavaria | DE-97072 | Germany |
| Research Site | Recruiting | Frankfurt am Main | Hesse | 60590 | Germany |
| Research Site | Recruiting | Hanover | Lower Saxony | 30625 | Germany |
| Research Site | Recruiting | Aachen | North Rhine-Westphalia | 52074 | Germany |
| Research Site | Recruiting | Cologne | North Rhine-Westphalia | 50937 | Germany |
| Research Site | Recruiting | Mainz | Rhineland-Palatinate | 55131 | Germany |
| Research Site | Recruiting | Homburg | Saarland | 66421 | Germany |
| Research Site | Recruiting | Berlin | 10117 | Germany |
| Research Site | Recruiting | Huelva | Andalusia | 21005 | Spain |
| Research Site | Recruiting | Bilbao | Basque Country | 48013 | Spain |
| Research Site | Recruiting | Las Palmas de Gran Canaria | Canary Islands | 35010 | Spain |
| Research Site | Recruiting | Salamanca | Castille and León | 37007 | Spain |
| Research Site | Recruiting | Barcelona | Catalu A | 08035 | Spain |
| Research Site | Not yet recruiting | Barcelona | Catalu A | 8036 | Spain |
| Research Site | Not yet recruiting | Cataluna | Catalu A | 08907 | Spain |
| Research Site | Recruiting | Majadahonda | Madrid | 28222 | Spain |
| Research Site | Recruiting | Islas Baleares | Palma de Mallorca | 07198 | Spain |
| Research Site | Recruiting | Birmingham | West Midlands | B15 2GW | United Kingdom |
| Research Site | Recruiting | Glasgow | G51 4TF | United Kingdom |
| Research Site | Recruiting | London | NW3 2QG | United Kingdom |
| ID | Term |
|---|---|
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| D000686 | Amyloidosis |
| D028227 | Amyloid Neuropathies, Familial |
| D011115 | Polyneuropathies |
| D017772 | Amyloid Neuropathies |
| D028226 | Amyloidosis, Familial |
| ID | Term |
|---|---|
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
Not provided
Not provided
| ID | Term |
|---|---|
| D000682 | Amyloid |
| ID | Term |
|---|---|
| D046912 | Multiprotein Complexes |
| D046911 | Macromolecular Substances |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
Not provided
Not provided