트랜스티레틴(ATTR) 아밀로이드증 환자에 대한 비중재 연구 (MaesTTRo)
트랜스티레틴(ATTR) 아밀로이드증 환자의 특성, 치료 패턴 및 결과에 대한 실제 데이터를 수집하는 비개입, 전향적, 다국가 연구
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.
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
상세 설명
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.
연구 유형
연구 유형
등록 (추정된)
등록
연락처 및 위치
연구 연락처
연구 연락처
- 이름: AstraZeneca Clinical Study Information Center
- 전화번호: 1-877-240-9479
- 이메일: information.center@astrazeneca.com
연구 장소
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Berlin, 독일, 10117
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Bavaria
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Würzburg, Bavaria, 독일, DE-97072
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Hesse
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Frankfurt am Main, Hesse, 독일, 60590
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Lower Saxony
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Hanover, Lower Saxony, 독일, 30625
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North Rhine-Westphalia
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Aachen, North Rhine-Westphalia, 독일, 52074
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Cologne, North Rhine-Westphalia, 독일, 50937
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Rhineland-Palatinate
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Mainz, Rhineland-Palatinate, 독일, 55131
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Saarland
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Homburg, Saarland, 독일, 66421
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California
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La Jolla, California, 미국, 92037
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Los Angeles, California, 미국, 90095
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San Francisco, California, 미국, 94143
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San Francisco, California, 미국, 94025
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Connecticut
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New Haven, Connecticut, 미국, 06510
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District of Columbia
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Washington D.C., District of Columbia, 미국, 20010
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Illinois
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Chicago, Illinois, 미국, 60637
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Indiana
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Indianapolis, Indiana, 미국, 06200
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Maryland
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Baltimore, Maryland, 미국, 21218
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Massachusetts
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Boston, Massachusetts, 미국, 02111
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Boston, Massachusetts, 미국, 02114
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Boston, Massachusetts, 미국, 02115
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Minnesota
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Rochester, Minnesota, 미국, 55905
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Missouri
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Kansas City, Missouri, 미국, 64111
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St Louis, Missouri, 미국, 63110
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New Jersey
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New Brunswick, New Jersey, 미국, 08901
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New York
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Manhasset, New York, 미국, 11030
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New York, New York, 미국, 10029
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New York, New York, 미국, 10016
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New York, New York, 미국, 10027
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North Carolina
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Durham, North Carolina, 미국, 27710
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Oregon
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Portland, Oregon, 미국, 97239
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Pennsylvania
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Danville, Pennsylvania, 미국, 17822
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Philadelphia, Pennsylvania, 미국, 19104
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Philadelphia, Pennsylvania, 미국, 19107
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South Carolina
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Greenville, South Carolina, 미국, 29607
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Tennessee
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Germantown, Tennessee, 미국, 38138
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Nashville, Tennessee, 미국, 037214
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Texas
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Dallas, Texas, 미국, 75246
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Houston, Texas, 미국, 77030
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Virginia
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Richmond, Virginia, 미국, 23298
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Washington
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Seattle, Washington, 미국, 98915
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Wisconsin
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Milwaukee, Wisconsin, 미국, 53226
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Andalusia
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Huelva, Andalusia, 스페인, 21005
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Basque Country
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Bilbao, Basque Country, 스페인, 48013
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Canary Islands
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Las Palmas de Gran Canaria, Canary Islands, 스페인, 35010
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Castille and León
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Salamanca, Castille and León, 스페인, 37007
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Catalu A
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Barcelona, Catalu A, 스페인, 08035
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Barcelona, Catalu A, 스페인, 8036
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Cataluna, Catalu A, 스페인, 08907
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Madrid
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Majadahonda, Madrid, 스페인, 28222
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Palma de Mallorca
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Islas Baleares, Palma de Mallorca, 스페인, 07198
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Glasgow, 영국, G51 4TF
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London, 영국, NW3 2QG
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West Midlands
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Birmingham, West Midlands, 영국, B15 2GW
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Anhui
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Hefei, Anhui, 중국, 230001
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Beijing Municipality
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Beijing, Beijing Municipality, 중국, 100191
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Beijing, Beijing Municipality, 중국, 100730
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Beijing, Beijing Municipality, 중국, 100034
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Beijing, Beijing Municipality, 중국, 100029
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Chongqing Municipality
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Chongqing, Chongqing Municipality, 중국, 400000
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Fujian
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Fuzhou, Fujian, 중국, 350005
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Xiamen, Fujian, 중국, 361016
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Guangdong
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Guangzhou, Guangdong, 중국, 510515
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Guangzhou, Guangdong, 중국, 510080
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Guizhou
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Guiyang, Guizhou, 중국, 550004
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Hebei
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Shijiazhuang, Hebei, 중국, 050000
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Hubei
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Wuhan, Hubei, 중국, 430000
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Wuhan, Hubei, 중국, 430030
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Hunan
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Changsha, Hunan, 중국, 410013
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Changsha, Hunan, 중국, 410011
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Jiangsu
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Nanjing, Jiangsu, 중국, 210029
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Suzhou, Jiangsu, 중국, 215006
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Jiangxi
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Nanchang, Jiangxi, 중국, 330000
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Liaoning
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Dalian, Liaoning, 중국, 116011
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Shandong
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Jinan, Shandong, 중국, 250012
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Shanghai Municipality
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Shanghai, Shanghai Municipality, 중국, 200040
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Shanghai, Shanghai Municipality, 중국, 200025
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Shanghai, Shanghai Municipality, 중국, 200031
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Shanxi
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Taiyuan, Shanxi, 중국, 030001
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Sichuan
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Chengdu, Sichuan, 중국, 610041
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Chengdu, Sichuan, 중국, 610072
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Xi'An
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Shaanxi, Xi'An, 중국, 710061
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Zhejiang
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Hangzhou, Zhejiang, 중국, 310009
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British Columbia
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Vancouver, British Columbia, 캐나다, V5Z 1M9
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Vancouver, British Columbia, 캐나다, V6Z 1Y6
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Nova Scotia
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Halifax, Nova Scotia, 캐나다, B3RIV9
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Ontario
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London, Ontario, 캐나다, N6A 5A5
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Toronto, Ontario, 캐나다, M2J 4W8
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Quebec
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Montreal, Quebec, 캐나다, H2X 0A9
- 아직 모집하지 않음
- Research Site
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Rimouski, Quebec, 캐나다, G5L 5T1
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- Research Site
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
Patients with a diagnosis of amyloid transthyretin (ATTR) amyloidosis
설명
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
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: COHORT
- 시간 관점: PROSPECTIVE
그룹/코호트 수
코호트 및 개입
그룹/코호트그룹/코호트 |
개입 / 치료개입 / 치료 |
|---|---|
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ATTR cardiomyopathy (ATTR-CM)
Patients with ATTR-CM at enrollment |
Data will be collected on patients with ATTR amyloidosis in a real-world setting |
|
Hereditary polyneuropathy (ATTRv-PN)
Patients with ATTRv-PN at enrollment |
Data will be collected on patients with ATTR amyloidosis in a real-world setting |
|
ATTR-Mixed
Patients with a mixed ATTR amyloidosis phenotype |
Data will be collected on patients with ATTR amyloidosis in a real-world setting |
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Demographic characteristics (overall and in patients initiating a treatment with eplontersen)
기간: From time of enrollment for up to 7 years
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From time of enrollment for up to 7 years
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Treatment patterns (overall and in patients initiating a treatment with eplontersen)
기간: From time of enrollment for up to 7 years
|
In order to address this objective, the following treatments will be assessed:
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From time of enrollment for up to 7 years
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Clinical characteristics (overall and in patients initiating a treatment with eplontersen)
기간: From time of enrollment for up to 7 years
|
In order to address this objective, the following clinical characteristics will be assessed:
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From time of enrollment for up to 7 years
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Findings from biopsy (overall and in patients initiating a treatment with eplontersen)
기간: From time of enrollment for up to 7 years
|
In order to address this objective, the following biopsy information will be collected:
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From time of enrollment for up to 7 years
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Findings from Cardiovascular magnetic resonance imaging (CMR) (overall and in patients initiating a treatment with eplontersen)
기간: 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
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Findings from Bone tracer cardiac scintigraphy (overall and in patients initiating a treatment with eplontersen)
기간: 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
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Findings from Echocardiography (overall and in patients initiating a treatment with eplontersen)
기간: 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
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ECG variables (overall and in patients initiating a treatment with eplontersen)
기간: From time of enrollment for up to 7 years
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In order to address this objective, the following ECG information will be collected:
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From time of enrollment for up to 7 years
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Sural nerve and tibial nerve amplitude (overall and in patients initiating a treatment with eplontersen)
기간: 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
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Biomarker results (overall and in patients initiating a treatment with eplontersen)
기간: From time of enrollment for up to 7 years
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In order to address this objective, the following biomarker results will be collected:
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From time of enrollment for up to 7 years
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Urine test results (overall and in patients initiating a treatment with eplontersen)
기간: From time of enrollment for up to 7 years
|
In order to address this objective, the following test results will be collected:
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From time of enrollment for up to 7 years
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Clinical manifestations (signs and symptoms) of ATTR amyloidosis (overall and in patients initiating a treatment with eplontersen)
기간: 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
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36-Item Short Form Health Survey Version 2 (SF-36v2) Physical Component Summary score (overall and in patients initiating a treatment with eplontersen)
기간: 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
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Norfolk Quality of Life-Diabetic Neuropathy total score (overall and in patients initiating a treatment with eplontersen)
기간: 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
|
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Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score (overall and in patients initiating a treatment with eplontersen)
기간: 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
|
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New York Heart Association (NYHA) classification (overall and in patients initiating a treatment with eplontersen)
기간: 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
|
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National Amyloidosis Centre (NAC) ATTR staging or Mayo staging (overall and in patients initiating a treatment with eplontersen)
기간: 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
|
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Familial amyloid polyneuropathy (FAP) (Coutinho) staging (overall and in patients initiating a treatment with eplontersen)
기간: 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
|
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Polyneuropathy disability (PND) score (overall and in patients initiating a treatment with eplontersen)
기간: 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
|
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Left Ventricular Ejection Fraction (overall and in patients initiating a treatment with eplontersen)
기간: 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
|
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6-minute walk test (overall and in patients initiating a treatment with eplontersen)
기간: 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)
기간: From time of enrollment for up to 7 years
|
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)
기간: From time of enrollment for up to 7 years
|
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)
기간: From time of enrollment for up to 7 years
|
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)
기간: 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
기간: From time of Enrollment for up to 7 Years
|
|
From time of Enrollment for up to 7 Years
|
2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Comparison of demographic and clinical characteristics of patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
기간: Up to 7 years
|
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 biopsy in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
기간: Up to 7 years
|
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
기간: 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
기간: 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
기간: Up to 7 years
|
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
기간: 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
기간: 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
기간: Up to 7 years
|
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
기간: Up to 7 years
|
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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: Up to 7 years
|
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
기간: Up to 7 years
|
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
기간: Up to 7 years
|
Mortality will be compared in patients prescribed eplontersen to patients on other ATTR treatments |
Up to 7 years
|
기타 결과 측정
기타 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Risk factors for worsening ATTR progression
기간: 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
기간: 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
기간: Up to 7 years
|
Serious adverse events in patients treated with ATTR amyloidosis treatments will be measured |
Up to 7 years
|
공동 작업자 및 조사자
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- D8450R00003
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
Plan to share only the redacted CSR synopsis
IPD 공유 기간
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.
IPD 공유 액세스 기준
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.
IPD 공유 지원 정보 유형
- CSR
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .