ClinicalTrials.gov record NCT06465810

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

A Non-interventional, Prospective, Multi-country Study Collecting Real-world Data on the Characteristics, Treatment Patterns, and Outcomes of Patients With Transthyretin (ATTR) Amyloidosis

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age18 years old to 130 years old
SexAll
LocationCanada, China, Germany, Spain, United Kingdom, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

An international, non-interventional study that follows adults with transthyretin (ATTR) amyloidosis over time to record disease course, treatments, and outcomes. About 1,850 patients are planned; each will be followed at least 3 years and up to 7 years. Data come from routine clinic records and electronic patient-reported outcome questionnaires completed at enrollment and roughly every six months during usual care visits. US patients may be asked to allow additional de-identified healthcare data linkage via a tokenization process.

What it is studying. This study is observing the natural history of ATTR amyloidosis and describing real-world treatment patterns and outcomes, including collecting data on patients who receive eplontersen compared with other treatments.

What the researchers hope to learn. To describe disease characteristics and progression in people with ATTR amyloidosis; to document real-world treatment patterns and outcomes; and to compare clinical, imaging, biomarker, quality-of-life, healthcare use, and mortality outcomes for patients treated with eplontersen versus other ATTR treatments.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Treatment of transthyretin (ATTR) amyloidosis in observational study setting

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an observational study that collects information from your routine medical records and from electronic questionnaires. You will be asked to complete electronic PRO questionnaires at enrollment and about every 6 months (±3 months) during routine clinic visits; clinical, imaging, treatment, and lab data will be abstracted from your health records. No extra study visits are required and you will not be contacted for data collection outside routine care. For patients in the United States, there is an optional tokenization step to collect additional de‑identified healthcare data from other routine sources; you can opt in or out in the consent form.

How the study is structured. A prospective observational cohort study. Participants continue their usual medical care; no treatments are assigned by the study. Investigators will collect patient-reported questionnaires at enrollment and about every six months during routine visits, and will abstract clinical, imaging, laboratory, biopsy, and treatment information from medical records. Follow-up for each patient will be at least 3 years and up to 7 years. US participants may opt in to a tokenization process to allow additional de-identified healthcare data linkage.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in Canada, China, Germany, Spain, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on ATTRv amyloidosis

This trial is also listed on this site under wild-type ATTR amyloidosis.

Clinical trials106 trials listed for ATTRv amyloidosisEvery trial the registry lists for ATTRv amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in ATTRv amyloidosis researchLatest monthly update: September 2026. Includes: Gene-silencing drugs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 25, 2024
Primary completion
Dec 31, 2031 (estimated)
Study completion
Dec 31, 2031 (estimated)
Target enrollment
1850 participants (estimated)
Last updated on registry
Sep 23, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
AstraZeneca
Collaborators
  • ICON plc

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (89)

  • Research SiteRecruiting

    La Jolla, California, 92037 · United States

  • Research SiteRecruiting

    Los Angeles, California, 90095 · United States

  • Research SiteRecruiting

    San Francisco, California, 94025 · United States

  • Research SiteRecruiting

    San Francisco, California, 94143 · United States

  • Research SiteRecruiting

    New Haven, Connecticut, 06510 · United States

  • Research SiteRecruiting

    Washington D.C., District of Columbia, 20010 · United States

  • Research SiteRecruiting

    Chicago, Illinois, 60637 · United States

  • Research SiteRecruiting

    Indianapolis, Indiana, 06200 · United States

  • Research SiteRecruiting

    Baltimore, Maryland, 21218 · United States

  • Research SiteRecruiting

    Boston, Massachusetts, 02111 · United States

Terms used on this page · explanations by MedicalUpdates.ai

cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
interventional
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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