MaesTTRo

• Research type

  Research Study

• Full title

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

• IRAS ID

  341622

• Contact name

  Lucia Venneri

• Contact email

  lucia.venneri@nhs.net

• Sponsor organisation

  AstraZeneca

• Clinicaltrials.gov Identifier

  61999, NIHR

• Duration of Study in the UK

  6 years, 4 months, 1 days

• Research summary

  Amyloid transthyretin (ATTR) amyloidosis is a clinically heterogenous and fatal disease caused by the build-up of transthyretin (TTR) amyloid fibrils in various organs and tissues. Phenotypically, ATTR amyloidosis presents as either ATTR cardiomyopathy (ATTR-CM), ATTR polyneuropathy (ATTR-PN), or a mix of both. Depending on the clinical features, most patients experience a mixed phenotype with both neuropathic and cardiac symptoms. There are two forms of ATTR amyloidosis: ATTRv, the
  hereditary form caused by a mutation in the transthyretin gene, and ATTRwt, referred to as a wild-type form where no mutations are present. ATTR amyloidosis is often undetected or misdiagnosed in patients, due to the non-specific, heterogeneous, multisystem presentation.

  This study aims to create a global cohort of patients with 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 intended to increase knowledge about different genotypes and phenotypes of ATTR, diagnosis, management, and treatment of patients with ATTR amyloidosis in a real-world setting.

  Several features will distinguish this study from existing registries and real-world studies:
  • Eligibility for enrollment of all patients with ATTR amyloidosis, irrespective of
  treatments received
  • Good representation of different ATTR amyloidosis phenotypes
  • Hybrid design, which will incorporate both primary data (patient-centric measures)
  and secondary data collection
  • Targeted collection of study variables to minimize study site workload
  • Collection of data available as per routine care
  • Early and frequent collaboration with key external experts on study design, study
  conduct, data interpretation, and publication planning and authorship

• REC name

  North West - Greater Manchester West Research Ethics Committee

• REC reference

  24/NW/0203

• Date of REC Opinion

  2 Aug 2024

• REC opinion

  Further Information Favourable Opinion