Observing the natural course of transthyretin amyloidosis
Official title: A Non-interventional, Prospective, Multi-country Study Collecting Real-world Data on the Characteristics, Treatment Patterns, and Outcomes of Patients With Transthyretin (ATTR) Amyloidosis
Run by AstraZeneca · NCT06465810 · Data updated 2026-11-12
This study is looking to see how transthyretin amyloidosis progresses over time in over 1,600 adults while also gathering information on how well treatments like eplontersen work.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 18 Years to 130 Years |
| Sex | All |
| Enrollment | 1850 (estimated) |
| Sponsor | AstraZeneca Industry-sponsored |
| Locations | 89 sites (La Jolla, California and 88 other locations) |
| Status | Recruiting |
| Started | June 25, 2024 |
| Main results expected | December 31, 2031 |
| Study ends | December 31, 2031 |
| Registry record updated | September 23, 2026 |
| Trial ID | NCT06465810 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of ATTR amyloidosis who can provide informed consent.
✕Probably not a fit
Patients with primary or light chain amyloidosis, asymptomatic ATTR mutation carriers, or those participating in interventional trials for ATTR amyloidosis may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
The MaesTTRo study is an international, longitudinal, non-interventional study that aims to enroll over 1,600 adult patients diagnosed with transthyretin (ATTR) amyloidosis. The study will collect primary data through patient-reported outcome questionnaires and secondary data from routine clinical practices to observe the disease's natural progression and treatment patterns. Patients will be followed for a minimum of 3 years, with data collected during routine visits, ensuring minimal disruption to their care. The study also aims to gather information on the effectiveness of treatments like eplontersen, a gene silencing therapy targeting TTR proteins.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the natural history of ATTR amyloidosis and improve treatment strategies for patients.
How similar studies have gone: While this study employs a novel observational approach, similar studies have shown success in understanding the natural history of other amyloidosis types.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
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La Jolla, California, United States Recruiting -
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Jinan, Shandong, China Recruiting
+39 more sites. See ClinicalTrials.gov for the full list.
Study contacts
- Study coordinator: AstraZeneca Clinical Study Information Center
- Email: information.center@astrazeneca.com
- Phone: 1-877-240-9479
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Acoramidis for ATTR-CM: Cardiac Imaging Study
This study tests whether acoramidis can improve heart structure and function in people with transthyretin amyloid cardiomyopathy.
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Long-term safety of eplontersen in adults with transthyretin amyloidosis, including those with prior liver transplant or severe liver impairment
This study will see if eplontersen remains safe over time for adults with transthyretin amyloidosis, including people who have had a liver transplant or have severe liver problems.
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Annular ligament biopsy during endoscopic carpal tunnel surgery to screen for transthyretin amyloidosis
This study tests whether a biopsy of the annular ligament during carpal tunnel surgery can reliably indicate transthyretin amyloidosis and match the results of the standard synovial biopsy.
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Eplontersen exposure during pregnancy and breastfeeding: outcomes
This project collects reports to see if exposure to eplontersen during pregnancy or breastfeeding affects mothers, fetuses, or infants with transthyretin amyloidosis.
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Understanding Atrioventricular Regurgitation in ATTR Amyloid Cardiomyopathy
This study is trying to understand how common certain heart valve problems are in people with ATTR amyloid cardiomyopathy and create better ways to measure their severity.
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Preventing transthyretin amyloidosis in young carriers of a genetic variant
This study is testing if a medication called acoramidis can help prevent transthyretin amyloidosis in young adults who carry a specific genetic variant that puts them at risk for the disease.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.