OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis

ATTR Amyloidosis Clinical Trial

— OverTTuRe  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06355934
• Other study ID #: D8450R00004
• Status: Recruiting
• Start date: August 21, 2023
• Est. completion date: September 30, 2026

Study information

• Verified date: June 2024
• Source: AstraZeneca
• Contact: AstraZeneca Clinical Study Information Center
• Phone: 1-877-240-9479
• Email: information.center[@]astrazeneca.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 55000
• Est. completion date: September 30, 2026
• Est. primary completion date: September 30, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Amyloidosis
• ATTR Amyloidosis

Intervention

Other:
• no intervention
not applicable, this is an observational retrospective data analysis study; no interventions in the study

Locations

Country	Name	City	State
Denmark	Research Site	Horsens
Japan	Research Site	Tokyo
Spain	Research Site	A Coruna
Spain	Research Site	Barcelona
Spain	Research Site	Bilbao
Spain	Research Site	El Palmar
Spain	Research Site	Huelva
Spain	Research Site	Las Palmas de Gran Canaria
Spain	Research Site	Madrid
Spain	Research Site	Majadahonda
Spain	Research Site	Palma de Mallorca
Spain	Research Site	Salamanca
Spain	Research Site	Valencia
Sweden	Research Site	Sodertalje
Sweden	Research Site	Umea
United Kingdom	Research Site	London
United States	Research Site	Eden Prairie	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
AstraZeneca

Countries where clinical trial is conducted

United States,  Denmark,  Japan,  Spain,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Health Care Resource Utilization (HCRU) - Outpatient visits	Annualized events rate for: Overall outpatient visits	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Health Care Resource Utilization (HCRU) - Outpatient visits by specialty	Annualized events rate for: Overall outpatient visits by specialty	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Health Care Resource Utilization (HCRU) - Emergency department visits	Annualized events rate for: Emergency department visits	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay	Annualized events rate for: Hospitalizations (bed days)	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Health Care Resource Utilization (HCRU) - Hospitalizations	Annualized events rate for: Hospitalizations Overlaps (e.g., hospitalization after emergency department visit) are allowed and counted as separate visits	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Health Care Resource Utilization (HCRU) - Health care cost	This variable will describe all-cause direct medical and pharmacy costs only, utilizing the amount charged, where available in the data. The direct medical costs will include costs incurred from inpatient stays, outpatient visits, emergency room visits, procedures, and laboratory tests. The inclusion of pharmacy costs is subject to data availability and will include all pharmacy costs per patient separated on pharmacy dispensed and in-hospital dispensed when possible. When feasible, costs will be divided on all-cause, CV, and other specialties	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Cardiac transplant	Cardiac transplant (Time to event)	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
Primary	All cause mortality	Mortality (time-to-event)	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
Primary	Liver transplant	Liver transplant (time-to-event)	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
Primary	Heart Failure Hospitalization	Heart failure hospitalization (time-to-event)	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
Primary	New ATTR amyloidosis clinical manifestation	New amyloidosis manifestation (time-to-event); Time to the first development of a new clinical manifestation that the patient did not have pre-index)	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
Primary	Hospitalization (any cause)	Time to hospitalization (all-cause)	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
Primary	Neuropathy Impairment Score (NIS)	The NIS Score has a range of 0 to 244 and a higher NIS score indicates poorer function	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Neuropathy Impairment Score Lower Limbs (NIS-LL)	The NIS-LL score has a range of 0-88 and a greater NIS-LL score indicates poorer function	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Neuropathy Impairment Score +7 (NIS+7)	The NIS+7 Score has a range of -26.04 to 270.04 and a higher NIS+7 score indicates poorer function	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Neuropathy Impairment Score modified +7 (mNIS+7)	The mNIS+7 Score has a range of -22.32 to 102.32 and a higher mNIS+7 score indicates poorer function	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Neuropathy symptoms and change (NCS) score	NSC score is a questionnaire composed of 38 questions that assess the presence and severity of these neuropathy symptoms. The NSC score ranges from -114 to 114 for males and -108 to 108 for females. Greater scores indicate worse symptom severity; a negative value indicates an improvement in symptom severity from baseline	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	PND (Polyneuropathy Disability)	The PND is a 6-stage scoring system: Stage 0: no impairment; Stage 1: sensory disturbances but preserved walking capabilities; Stage 2: impaired walking capacity, but ability to walk without a stick or crutches; Stage 3A/B: walking with help of 1 or 2 sticks or crutches; Stage 4: confined to wheel chair or bedridden. A greater stage indicates greater impairment	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Primary	Other relevant clinical measurement of ATTR amyloidosis functional status	Any other relevant clinical measurement of ATTR amyloidosis functional status	From diagnosis of ATTR amyloidosis (index date), assessed throughout the study until end of follow-up, up to a maximum of 12 years
Secondary	Health Care Resource Utilization (HCRU) - Outpatient visits	Annualized events rate for: Overall outpatient visits	From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Secondary	Health Care Resource Utilization (HCRU) - Outpatient visits by specialty	Annualized events rate for: Overall outpatient visits by specialty	From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Secondary	Health Care Resource Utilization (HCRU) - Emergency department visits	Annualized events rate for: Emergency department visits	From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Secondary	Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay	Annualized events rate for: Hospitalizations (bed days)	From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Secondary	Health Care Resource Utilization (HCRU) - Hospitalizations	Annualized events rate for: Hospitalizations Overlaps (e.g., hospitalization after emergency department visit) are allowed and counted as separate visits	From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.
Secondary	Health Care Resource Utilization (HCRU) - Health care cost	This variable will describe all-cause direct medical and pharmacy costs only, utilizing the amount charged, where available in the data. The direct medical costs will include costs incurred from inpatient stays, outpatient visits, emergency room visits, procedures, and laboratory tests. The inclusion of pharmacy costs is subject to data availability and will include all pharmacy costs per patient separated on pharmacy dispensed and in-hospital dispensed when possible. When feasible, costs will be divided on all-cause, CV, and other specialties	From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.