Recruiting

Observational Study

Sponsor:

AstraZeneca

Code:

NCT06465810

Conditions

Transthyretin Amyloidosis

ATTR-CM

ATTRv-PN

ATTR

ATTR-Mixed

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

Treatment of transthyretin (ATTR) amyloidosis in observational study setting

Study Details

Brief 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.

Conditions

Transthyretin Amyloidosis

ATTR-CM

ATTRv-PN

ATTR

ATTR-Mixed

Study ID

NCT06465810

Start date

Jun 25, 2024

Status verified date

Aug, 2026

Completion date

Dec 29, 2031

Anticipated

Primary completion date

Dec 29, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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 Design

Enrollment

1850 participants

Anticipated

Interventions and Outcome Measures

Arms

ATTR cardiomyopathy (ATTR-CM)

Patients with ATTR-CM at enrollment

Hereditary polyneuropathy (ATTRv-PN)

Patients with ATTRv-PN at enrollment

ATTR-Mixed

Patients with a mixed ATTR amyloidosis phenotype

Interventions

Treatment of transthyretin (ATTR) amyloidosis in observational study setting

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

Primary outcome measure

  • Demographic characteristics (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Treatment patterns (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Clinical characteristics (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Findings from biopsy (overall and in patients initiating a treatment with eplontersen) [ Time Frame: 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) [ Time Frame: From time of enrollment for up to 7 years ]
  • Findings from Bone tracer cardiac scintigraphy (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Findings from Echocardiography (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • ECG variables (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Sural nerve and tibial nerve amplitude (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Biomarker results (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Urine test results (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Clinical manifestations (signs and symptoms) of ATTR amyloidosis (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • 36-Item Short Form Health Survey Version 2 (SF-36v2) Physical Component Summary score (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Norfolk Quality of Life-Diabetic Neuropathy total score (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • New York Heart Association (NYHA) classification (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • National Amyloidosis Centre (NAC) ATTR staging or Mayo staging (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Familial amyloid polyneuropathy (FAP) (Coutinho) staging (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Polyneuropathy disability (PND) score (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Left Ventricular Ejection Fraction (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • 6-minute walk test (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Charlson comorbidity index (CCI) and CCI components (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Other comorbidities of interest (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Healthcare resource utilization (overall and in patients initiating a treatment with eplontersen) [ Time Frame: From time of enrollment for up to 7 years ]
  • Mortality (overall and in patients initiating a treatment with eplontersen) [ Time Frame: Throughout study follow-up (up to 7 years) ]
  • Liver Disease and Live Transplant [ Time Frame: From time of Enrollment for up to 7 Years ]

Central Contacts and Locations

Central contacts

AstraZeneca Clinical Study Information Center

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

Locations

Research Site

Recruiting

La Jolla, California, United States, 92037

Research Site

Recruiting

Los Angeles, California, United States, 90095

Research Site

Recruiting

San Francisco, California, United States, 94025

Research Site

Recruiting

San Francisco, California, United States, 94143

Research Site

Recruiting

New Haven, Connecticut, United States, 006510

Research Site

Recruiting

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

Research Site

Recruiting

Chicago, Illinois, United States, 60637

Research Site

Recruiting

Indianapolis, Indiana, United States, 06200

Research Site

Recruiting

Baltimore, Maryland, United States, 21218

Research Site

Recruiting

Boston, Massachusetts, United States, 02111

Research Site

Recruiting

Boston, Massachusetts, United States, 02115

Research Site

Recruiting

Rochester, Minnesota, United States, 55905

Research Site

Recruiting

Kansas City, Missouri, United States, 64111

Research Site

Recruiting

St Louis, Missouri, United States, 63110

Research Site

Recruiting

New Brunswick, New Jersey, United States, 08901

Research Site

Recruiting

Manhasset, New York, United States, 11030

Research Site

Recruiting

New York, New York, United States, 10027

Research Site

Recruiting

New York, New York, United States, 10029

Research Site

Recruiting

Durham, North Carolina, United States, 27710

Research Site

Recruiting

Portland, Oregon, United States, 97239

Research Site

Recruiting

Danville, Pennsylvania, United States, 17822

Research Site

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Research Site

Recruiting

Philadelphia, Pennsylvania, United States, 19107

Research Site

Recruiting

Greenville, South Carolina, United States, 29607

Research Site

Recruiting

Germantown, Tennessee, United States, 38138

Research Site

Recruiting

Nashville, Tennessee, United States, 037214

Research Site

Recruiting

Dallas, Texas, United States, 75246

Research Site

Recruiting

Richmond, Virginia, United States, 23298

Research Site

Recruiting

Seattle, Washington, United States, 98915

Research Site

Recruiting

Milwaukee, Wisconsin, United States, 53226

Research Site

Recruiting

Vancouver, British Columbia, Canada, V5Z 1M9

Research Site

Recruiting

Vancouver, British Columbia, Canada, V6Z 1Y6

Research Site

Recruiting

Halifax, Nova Scotia, Canada, B3RIV9

Research Site

Recruiting

London, Ontario, Canada, N6A 5A5

Research Site

Recruiting

Toronto, Ontario, Canada, M2J 4W8

More Information

Sponsor

AstraZeneca

Last update posted

Aug 19, 2026

Last verified

Aug, 2026

Keywords

  • 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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by AstraZeneca on 2026-08-19.