RecruitingNot Applicable

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

United States, Canada, China1,850 participantsStarted 2024-06-25

Plain-language summary

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.

Who can participate

Age range

18 Years – 130 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Demographic characteristics (overall and in patients initiating a treatment with eplontersen)

Timeframe: From time of enrollment for up to 7 years

Treatment patterns (overall and in patients initiating a treatment with eplontersen)

Timeframe: From time of enrollment for up to 7 years

Clinical characteristics (overall and in patients initiating a treatment with eplontersen)

Timeframe: From time of enrollment for up to 7 years

Findings from biopsy (overall and in patients initiating a treatment with eplontersen)

Timeframe: 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)

Timeframe: From time of enrollment for up to 7 years

Findings from Bone tracer cardiac scintigraphy (overall and in patients initiating a treatment with eplontersen)

Timeframe: From time of enrollment for up to 7 years

Trial details

NCT IDNCT06465810

SponsorAstraZeneca

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2031-12-29

Contact for this trial

AstraZeneca Clinical Study Information Center

1-877-240-9479 information.center@astrazeneca.com

View on ClinicalTrials.gov More Transthyretin Amyloidosis trials

Plain-language summary

Who can participate

Age range

18 Years – 130 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Demographic characteristics (overall and in patients initiating a treatment with eplontersen)

Timeframe: From time of enrollment for up to 7 years

Treatment patterns (overall and in patients initiating a treatment with eplontersen)

Timeframe: From time of enrollment for up to 7 years

Clinical characteristics (overall and in patients initiating a treatment with eplontersen)

Timeframe: From time of enrollment for up to 7 years

Findings from biopsy (overall and in patients initiating a treatment with eplontersen)

Timeframe: 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)

Timeframe: From time of enrollment for up to 7 years

Findings from Bone tracer cardiac scintigraphy (overall and in patients initiating a treatment with eplontersen)

Timeframe: From time of enrollment for up to 7 years