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

N
Noel Dasgupta

Primary Investigator

Enrolling By Invitation
18 years or above
All
Phase N/A
5 participants needed
1 Location

Brief description of study

What is the purpose of this study?
MaesTTRo is an international, longitudinal, non-interventional study of adult patients with transthyretin (ATTR) amyloidosis. 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.
 
THIS STUDY IS ENROLLING BY INVITATION ONLY

Detailed description of study

What will happen during the study?
  • The study plans to enroll a minimum of 1,600 patients with ATTR amyloidosis, including a minimum of 1,500 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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: ATTR, ATTR-CM, ATTR-Mixed, ATTRv-PN, Transthyretin Amyloidosis, hATTR
  • Age: 18 years or above
  • Gender: All

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

Updated on 21 Apr 2025. Study ID: CTSI-AZ-MAESTTRO, PATH-AZ-MAESTTRO, 24652
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