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

NCT ID: NCT06465810

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

1850 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-06-25

Study Completion Date

2031-12-29

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Transthyretin Amyloidosis ATTR-CM ATTRv-PN ATTR ATTR-Mixed hATTR

Keywords

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Amyloidosis Transthyretin Hereditary transthyretin-mediated (hATTR) amyloidosis hATTR amyloidosis Hereditary ATTR amyloidosis Wild-type amyloidosis wtATTR amyloidosis ATTRv amyloidosis ATTRwt amyloidosis Polyneuropathy Familial amyloid polyneuropathies ATTR Transthyretin amyloidosis TTR-mediated amyloidosis Polyneuropathies Amyloid neuropathies Amyloid neuropathies, familial Amyloidosis, familial Eplontersen Non-interventional Observational Real-world

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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ATTR cardiomyopathy (ATTR-CM)

Patients with ATTR-CM at enrollment

Treatment of transthyretin (ATTR) amyloidosis in observational study setting

Intervention Type DRUG

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

Hereditary polyneuropathy (ATTRv-PN)

Patients with ATTRv-PN at enrollment

Treatment of transthyretin (ATTR) amyloidosis in observational study setting

Intervention Type DRUG

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

ATTR-Mixed

Patients with a mixed ATTR amyloidosis phenotype

Treatment of transthyretin (ATTR) amyloidosis in observational study setting

Intervention Type DRUG

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

Interventions

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Treatment of transthyretin (ATTR) amyloidosis in observational study setting

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

130 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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ICON plc

INDUSTRY

Sponsor Role collaborator

AstraZeneca

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site

La Jolla, California, United States

Site Status RECRUITING

Research Site

Los Angeles, California, United States

Site Status RECRUITING

Research Site

San Francisco, California, United States

Site Status RECRUITING

Research Site

San Francisco, California, United States

Site Status RECRUITING

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New Haven, Connecticut, United States

Site Status RECRUITING

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

Site Status RECRUITING

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Chicago, Illinois, United States

Site Status RECRUITING

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Indianapolis, Indiana, United States

Site Status RECRUITING

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Boston, Massachusetts, United States

Site Status RECRUITING

Research Site

Boston, Massachusetts, United States

Site Status NOT_YET_RECRUITING

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Boston, Massachusetts, United States

Site Status RECRUITING

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Rochester, Minnesota, United States

Site Status RECRUITING

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Kansas City, Missouri, United States

Site Status RECRUITING

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St Louis, Missouri, United States

Site Status RECRUITING

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New Brunswick, New Jersey, United States

Site Status RECRUITING

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Manhasset, New York, United States

Site Status RECRUITING

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New York, New York, United States

Site Status RECRUITING

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New York, New York, United States

Site Status RECRUITING

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Durham, North Carolina, United States

Site Status RECRUITING

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Danville, Pennsylvania, United States

Site Status RECRUITING

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Philadelphia, Pennsylvania, United States

Site Status RECRUITING

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Philadelphia, Pennsylvania, United States

Site Status RECRUITING

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Greenville, South Carolina, United States

Site Status RECRUITING

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Germantown, Tennessee, United States

Site Status RECRUITING

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Nashville, Tennessee, United States

Site Status RECRUITING

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Dallas, Texas, United States

Site Status RECRUITING

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Richmond, Virginia, United States

Site Status RECRUITING

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Seattle, Washington, United States

Site Status RECRUITING

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Milwaukee, Wisconsin, United States

Site Status RECRUITING

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Vancouver, British Columbia, Canada

Site Status RECRUITING

Research Site

Vancouver, British Columbia, Canada

Site Status RECRUITING

Research Site

Halifax, Nova Scotia, Canada

Site Status RECRUITING

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London, Ontario, Canada

Site Status RECRUITING

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Toronto, Ontario, Canada

Site Status RECRUITING

Research Site

Rimouski, Quebec, Canada

Site Status NOT_YET_RECRUITING

Research Site

Würzburg, Bavaria, Germany

Site Status NOT_YET_RECRUITING

Research Site

Frankfurt am Main, Hesse, Germany

Site Status RECRUITING

Research Site

Hanover, Lower Saxony, Germany

Site Status RECRUITING

Research Site

Aachen, North Rhine-Westphalia, Germany

Site Status RECRUITING

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Cologne, North Rhine-Westphalia, Germany

Site Status RECRUITING

Research Site

Mainz, Rhineland-Palatinate, Germany

Site Status RECRUITING

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Homburg, Saarland, Germany

Site Status RECRUITING

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Berlin, , Germany

Site Status RECRUITING

Research Site

Bilbao, Basque Country, Spain

Site Status RECRUITING

Research Site

Salamanca, Castille and León, Spain

Site Status NOT_YET_RECRUITING

Research Site

Birmingham, West Midlands, United Kingdom

Site Status RECRUITING

Research Site

Glasgow, , United Kingdom

Site Status RECRUITING

Research Site

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Canada Germany Spain United Kingdom

Central Contacts

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AstraZeneca Clinical Study Information Center

Role: CONTACT

Phone: 1-877-240-9479

Email: [email protected]

References

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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.

Reference Type DERIVED
PMID: 40108078 (View on PubMed)

Other Identifiers

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D8450R00003

Identifier Type: -

Identifier Source: org_study_id