Clinical trial NCT06355934: OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis
Official title: OverTTuRe: An Observational Multi-Country Study Collecting Real-World Secondary Data on the Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: ATTR Amyloidosis
Keywords: ATTR-CM, ATTRv-PN
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2024-04-10
Enrollment: 55000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This study will include data from patients aged \> 18 years with a diagnosis of ATTR amyloidosis or other evidence confirming ATTR amyloidosis. Patients will be followed until exit from the database (loss to follow-up), death, or end of database period (data collection). Baseline data will apply a lookback period of 12 months. Patient identification will be from 2014-01-01 onwards

Brief summary: The overall aim of this observational study is to generate real-world evidence on the pre- and post-diagnosis disease journeys, including baseline characteristics, treatment patterns and selected clinical, economic, and humanistic outcomes (for example Health Related Quality of Life (HRQoL), Neuropathy impairment score, activities of daily living (ADL) assessments) in patients with ATTR amyloidosis, and to better understand how the disease is presented.

Detailed description: This retrospective, observational, longitudinal, multi-country cohort study aims to describe characteristics, treatment patterns, and outcomes in ATTR amyloidosis patients.

Primary objectives: Describe patient characteristics (for example demographics, family history of ATTR, key comorbidities and humanistic outcomes (e.g. Health Related Quality of Life (HRQoL), Neuropathy impairment score, activities of daily living (ADL) assessments), treatment patterns, and disease outcomes. Characterize and quantify the healthcare resource utilization (HCRU) in ATTR amyloidosis patients who will be followed post-index until the end of follow-up.

Secondary objectives: Describe demographics, clinical characteristics and HCRU in ATTR amyloidosis patients prior to diagnosis.

Eligibility criteria: Inclusion Criteria:

* Patients aged \>18 years at study index date AND
* A reported diagnosis code for amyloidosis OR
* A claim for ATTR-specific treatment OR
* A positive biopsy for amyloidosis and positive immunostaining result of biopsy for ATTR

Exclusion Criteria:

* Evidence of primary (AL) and secondary (AA) amyloidosis AND/OR
* At least one claim/procedure code for stem cell transplant or at least two claims/procedure codes for chemotherapy and autoimmune disease drugs which may represent AL (primary) or AA (secondary) amyloidosis treatments

Primary outcomes:
measure: Health Care Resource Utilization (HCRU) - Outpatient visits
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Health Care Resource Utilization (HCRU) - Outpatient visits by specialty
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Health Care Resource Utilization (HCRU) - Emergency department visits
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Health Care Resource Utilization (HCRU) - Hospitalizations
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Health Care Resource Utilization (HCRU) - Health care cost
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Cardiac transplant
timeFrame: From diagnosis of ATTR amyloidosis (index date) until date of first cardiac transplant, assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: All cause mortality
timeFrame: From diagnosis of ATTR amyloidosis (index date) until date of death due to any cause, assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Liver transplant
timeFrame: From diagnosis of ATTR amyloidosis (index date) until date of first liver transplant, assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Heart Failure Hospitalization
timeFrame: From diagnosis of ATTR amyloidosis (index date) until date of first hospitalization for heart failure, assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: New ATTR amyloidosis clinical manifestation
timeFrame: From diagnosis of ATTR amyloidosis (index date) until date of first diagnosis of new ATTR amyloidosis clinical manifestation, assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Hospitalization (any cause)
timeFrame: From diagnosis of ATTR amyloidosis (index date) until date of first hospitalization for any reason, assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Neuropathy Impairment Score (NIS)
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Neuropathy Impairment Score Lower Limbs (NIS-LL)
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Neuropathy Impairment Score +7 (NIS+7)
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Neuropathy Impairment Score modified +7 (mNIS+7)
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Neuropathy symptoms and change (NCS) score
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: PND (Polyneuropathy Disability)
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
measure: Other relevant clinical measurement of ATTR amyloidosis functional status
timeFrame: From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Secondary outcomes:
measure: Health Care Resource Utilization (HCRU) - Outpatient visits
timeFrame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
measure: Health Care Resource Utilization (HCRU) - Outpatient visits by specialty
timeFrame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
measure: Health Care Resource Utilization (HCRU) - Emergency department visits
timeFrame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
measure: Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay
timeFrame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
measure: Health Care Resource Utilization (HCRU) - Hospitalizations
timeFrame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
measure: Health Care Resource Utilization (HCRU) - Health care cost
timeFrame: From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Other outcomes:
Locations:
city: Eden Prairie
country: United States
facility: Research Site
state: Minnesota
city: Calgary
country: Canada
facility: Research Site
state: Alberta
city: Beijing
country: China
facility: Research Site
city: Changsha
country: China
facility: Research Site
city: Chengdu
country: China
facility: Research Site
city: Guangzhou
country: China
facility: Research Site
city: Wuhan
country: China
facility: Research Site
city: Horsens
country: Denmark
facility: Research Site
city: Berlin
country: Germany
facility: Research Site
city: Tokyo
country: Japan
facility: Research Site
city: Lisbona
country: Portugal
facility: Research Site
city: Porto
country: Portugal
facility: Research Site
city: A Coruna
country: Spain
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
city: Bilbao
country: Spain
facility: Research Site
city: El Palmar
country: Spain
facility: Research Site
city: Huelva
country: Spain
facility: Research Site
city: Las Palmas de Gran Canaria
country: Spain
facility: Research Site
city: Madrid
country: Spain
facility: Research Site
city: Majadahonda
country: Spain
facility: Research Site
city: Palma de Mallorca
country: Spain
facility: Research Site
city: Salamanca
country: Spain
facility: Research Site
city: Valencia
country: Spain
facility: Research Site
city: Sodertalje
country: Sweden
facility: Research Site
city: Umea
country: Sweden
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
Study officials:
References: