Clinical trial NCT06465810: Non-interventional Study of Patients With Transthyretin (ATTR) 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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Transthyretin Amyloidosis
ATTR-CM
ATTRv-PN
ATTR
ATTR-Mixed
hATTR
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
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
Collaborators: name: ICON plc
First posted: 2024-06-20
Enrollment: 1600
Sex: ALL
Minimum age (years): 18
Maximum age (years): 130
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with a diagnosis of amyloid transthyretin (ATTR) amyloidosis

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.

Detailed description: MaesTTRo is an international, longitudinal, non-interventional study of adult patients with transthyretin (ATTR) amyloidosis.

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.

For patients enrolled in the United States, a tokenization process (creation of a unique, encrypted identifier called a token, in place of personal identifiable information) will be used to collect additional de-identified data (e.g., healthcare resource use, healthcare costs) from other sources that are part of patients' routine medical care (electronic medical, hospital, or pharmacy records). Only de-identified data will be analyzed. Patients will be given a choice within the informed consent form to opt in or opt out of participating in the tokenization process.

Eligibility 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

Primary outcomes:
measure: Demographic characteristics (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Treatment patterns (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Clinical characteristics (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Findings from biopsy (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: 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
measure: 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
measure: Findings from Echocardiography (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: ECG variables (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Sural nerve and tibial nerve amplitude (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Biomarker results (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Urine test results (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Clinical manifestations (signs and symptoms) of ATTR amyloidosis (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: 36-Item Short Form Health Survey Version 2 (SF-36v2) Physical Component Summary score (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Norfolk Quality of Life-Diabetic Neuropathy total score (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: New York Heart Association (NYHA) classification (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: National Amyloidosis Centre (NAC) ATTR staging (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Familial amyloid polyneuropathy (FAP) (Coutinho) staging (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Polyneuropathy disability (PND) score (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Left Ventricular Ejection Fraction (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: 6-minute walk test (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Charlson comorbidity index (CCI) and CCI components (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Other comorbidities of interest (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Healthcare resource utilization (overall and in patients initiating a treatment with eplontersen)
timeFrame: From time of enrollment for up to 7 years
measure: Mortality (overall and in patients initiating a treatment with eplontersen)
timeFrame: Throughout study follow-up (up to 7 years)
Secondary outcomes:
measure: Comparison of demographic and clinical characteristics of patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of findings from biopsy in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of findings from Cardiovascular magnetic resonance imaging (CMR) in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of findings from Echocardiography in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of ECG variables of patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of findings from Bone tracer cardiac scintigraphyin patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of sural nerve and tibial nerve amplitude in patients prescribed eplontersen at any time during the observation eriod to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of biomarker results in patients prescribed eplontersen at any time during the observation eriod to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of urine test results in patients prescribed eplontersen at any time during the observation eriod to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of Clinical manifestations (signs and symptoms) of ATTR amyloidosis in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of 36-Item Short Form Health Survey Version 2 (SF-36v2) Physical Component Summary score in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of Norfolk Quality of Life-Diabetic Neuropathy total score in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of New York Heart Association (NYHA) classification in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of Familial amyloid polyneuropathy (FAP) (Coutinho) staging in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of Left Ventricular Ejection Fraction in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of Charlson comorbidity index (CCI) and CCI components in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of other comorbidities of interest in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of healthcare resource utilization in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
measure: Comparison of mortality in patients prescribed eplontersen to patients on other ATTR treatments
timeFrame: Up to 7 years
Other outcomes:
measure: Risk factors for worsening ATTR progression
timeFrame: up to 7 years
measure: Healthcare costs in patients with ATTR amyloidosis
timeFrame: Up to 7 years
measure: Serious adverse events in patients treated with ATTR amyloidosis treatments
timeFrame: Up to 7 years
Locations:
city: La Jolla
country: United States
facility: Research Site
state: California
city: Los Angeles
country: United States
facility: Research Site
state: California
city: San Francisco
country: United States
facility: Research Site
state: California
city: San Francisco
country: United States
facility: Research Site
state: California
city: New Haven
country: United States
facility: Research Site
state: Connecticut
city: Washington
country: United States
facility: Research Site
state: District of Columbia
city: Indianapolis
country: United States
facility: Research Site
state: Indiana
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Kansas City
country: United States
facility: Research Site
state: Missouri
city: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: New Brunswick
country: United States
facility: Research Site
state: New Jersey
city: Manhasset
country: United States
facility: Research Site
state: New York
city: New York
country: United States
facility: Research Site
state: New York
city: New York
country: United States
facility: Research Site
state: New York
city: Durham
country: United States
facility: Research Site
state: North Carolina
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Greenville
country: United States
facility: Research Site
state: South Carolina
city: Germantown
country: United States
facility: Research Site
state: Tennessee
city: Nashville
country: United States
facility: Research Site
state: Tennessee
city: Dallas
country: United States
facility: Research Site
state: Texas
city: Richmond
country: United States
facility: Research Site
state: Virginia
city: Seattle
country: United States
facility: Research Site
state: Washington
city: Milwaukee
country: United States
facility: Research Site
state: Wisconsin
city: Vancouver
country: Canada
facility: Research Site
state: British Columbia
city: Vancouver
country: Canada
facility: Research Site
state: British Columbia
city: Halifax
country: Canada
facility: Research Site
state: Nova Scotia
city: London
country: Canada
facility: Research Site
state: Ontario
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Wuerzburg
country: Germany
facility: Research Site
state: Bavaria
city: Frankfurt
country: Germany
facility: Research Site
state: Essen
city: Hannover
country: Germany
facility: Research Site
state: Lower Saxony
city: Aachen
country: Germany
facility: Research Site
state: NRW
city: Mainz
country: Germany
facility: Research Site
state: Rhineland Palatinate
city: Homburg
country: Germany
facility: Research Site
state: Saarland
city: Berlin
country: Germany
facility: Research Site
city: Glasgow
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
Study officials:
References: