Clinical Trial
Study Details
Study Overview
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.
Contacts and Locations
Research Site
La Jolla, California 92037
United States
Research Site
Los Angeles, California 90095
United States
Research Site
San Francisco, California 94025
United States
Research Site
San Francisco, California 94143
United States
Research Site
New Haven, Connecticut 006510
United States
Research Site
Washington D.C., District of Columbia 20010
United States
Research Site
Chicago, Illinois 60637
United States
Research Site
Indianapolis, Indiana 06200
United States
Research Site
Baltimore, Maryland 21218
United States
Research Site
Boston, Massachusetts 02111
United States
Research Site
Boston, Massachusetts 02114
United States
Research Site
Boston, Massachusetts 02115
United States
Research Site
Rochester, Minnesota 55905
United States
Research Site
Kansas City, Missouri 64111
United States
Research Site
St Louis, Missouri 63110
United States
Research Site
New Brunswick, New Jersey 08901
United States
Research Site
Manhasset, New York 11030
United States
Research Site
New York, New York 10016
United States
Research Site
New York, New York 10027
United States
Research Site
New York, New York 10029
United States
Research Site
Durham, North Carolina 27710
United States
Research Site
Portland, Oregon 97239
United States
Research Site
Danville, Pennsylvania 17822
United States
Research Site
Philadelphia, Pennsylvania 19104
United States
Research Site
Philadelphia, Pennsylvania 19107
United States
Research Site
Greenville, South Carolina 29607
United States
Research Site
Germantown, Tennessee 38138
United States
Research Site
Nashville, Tennessee 037214
United States
Research Site
Dallas, Texas 75246
United States
Research Site
Houston, Texas 77030
United States
Research Site
Richmond, Virginia 23298
United States
Research Site
Seattle, Washington 98915
United States
Research Site
Milwaukee, Wisconsin 53226
United States
Research Site
Vancouver British Columbia V5Z 1M9
Canada
Research Site
Vancouver British Columbia V6Z 1Y6
Canada
Research Site
Halifax Nova Scotia B3RIV9
Canada
Research Site
London Ontario N6A 5A5
Canada
Research Site
Toronto Ontario M2J 4W8
Canada
Research Site
Montreal Quebec H2X 0A9
Canada
Research Site
Rimouski Quebec G5L 5T1
Canada
Research Site
Hefei
Anhui, 230001
China
Research Site
Beijing
Beijing Municipality, 100029
China
Research Site
Beijing
Beijing Municipality, 100034
China
Research Site
Beijing
Beijing Municipality, 100191
China
Research Site
Beijing
Beijing Municipality, 100730
China
Research Site
Chongqing
Chongqing Municipality, 400000
China
Research Site
Fuzhou
Fujian, 350005
China
Research Site
Xiamen
Fujian, 361016
China
Research Site
Guangzhou
Guangdong, 510080
China
Research Site
Guangzhou
Guangdong, 510515
China
Research Site
Guiyang
Guizhou, 550004
China
Research Site
Shijiazhuang
Hebei, 050000
China
Research Site
Wuhan
Hubei, 430000
China
Research Site
Wuhan
Hubei, 430030
China
Research Site
Changsha
Hunan, 410011
China
Research Site
Changsha
Hunan, 410013
China
Research Site
Nanjing
Jiangsu, 210029
China
Research Site
Suzhou
Jiangsu, 215006
China
Research Site
Nanchang
Jiangxi, 330000
China
Research Site
Dalian
Liaoning, 116011
China
Research Site
Jinan
Shandong, 250012
China
Research Site
Shanghai
Shanghai Municipality, 200025
China
Research Site
Shanghai
Shanghai Municipality, 200031
China
Research Site
Shanghai
Shanghai Municipality, 200040
China
Research Site
Taiyuan
Shanxi, 030001
China
Research Site
Chengdu
Sichuan, 610041
China
Research Site
Chengdu
Sichuan, 610072
China
Research Site
Shaanxi
Xi'An, 710061
China
Research Site
Hangzhou
Zhejiang, 310009
China
Research Site
DE-97072 Würzburg
Germany
Research Site
60590 Frankfurt am Main
Germany
Research Site
30625 Hanover
Germany
Research Site
52074 Aachen
Germany
Research Site
50937 Cologne
Germany
Research Site
55131 Mainz
Germany
Research Site
66421 Homburg
Germany
Research Site
10117 Berlin
Germany
Research Site
21005 Huelva Andalusia
Spain
Research Site
48013 Bilbao Basque Country
Spain
Research Site
35010 Las Palmas de Gran Canaria Canary Islands
Spain
Research Site
37007 Salamanca Castille and León
Spain
Research Site
08035 Barcelona Catalu A
Spain
Research Site
8036 Barcelona Catalu A
Spain
Research Site
08907 Cataluna Catalu A
Spain
Research Site
28222 Majadahonda Madrid
Spain
Research Site
07198 Islas Baleares Palma de Mallorca
Spain
Research Site
Birmingham
B15 2GW
United Kingdom
Research Site
Glasgow
G51 4TF
United Kingdom
Research Site
London
NW3 2QG
United Kingdom