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

NCT06355934 | Recruiting

• 1 other identifier: D8450R00004
• Study Type: observational
• Target: 52,121
• Locations: 10 countries
• Sites: 26

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.

Conditions

ATTR Amyloidosis

Keywords

ATTR-CM, ATTRv-PN

Outcome Measures

Primary Outcomes (19)

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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 Outcomes (6)

• Health Care Resource Utilization (HCRU) - Outpatient visits

  From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.

• Health Care Resource Utilization (HCRU) - 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.

• Health Care Resource Utilization (HCRU) - Emergency department visits

  From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.

• Health Care Resource Utilization (HCRU) - Hospitalizations, length of stay

  From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.

• Health Care Resource Utilization (HCRU) - Hospitalizations

  From up to 12 years prior to ATTR amyloidosis diagnosis, assessed throughout the study, up to a maximum of 12 years.

Study Arms (6)

ATTR cardiomyopathy

Patients with Transthyretin Amyloidosis Cardiomyopathy

Other: no intervention

ATTR polyneuropathy

Patients with Transthyretin Amyloidosis Neuropathy

Other: no intervention

ATTR unknown

Patients with ATTR unknown genotype

Other: no intervention

ATTR with mixed phenotype

Patients with ATTR mixed phenotype

Other: no intervention

ATTR wild type

Patients with Transthyretin Amyloidosis wild type

Other: no intervention

ATTR hereditary

Patients with Transthyretin Hereditary

Other: no intervention

Interventions

no intervention OTHER

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

ATTR cardiomyopathy; ATTR hereditary; ATTR polyneuropathy; ATTR unknown; ATTR wild type; ATTR with mixed phenotype

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• AstraZeneca: lead

Study Sites (26)

Research Site

Eden Prairie, Minnesota, 55344, United States

COMPLETED

Research Site

Calgary, Alberta, T2P 4K7, Canada

RECRUITING

Research Site

Beijing, China

COMPLETED

Research Site

Changsha, China

COMPLETED

Research Site

Chengdu, China

COMPLETED

Research Site

Guangzhou, China

COMPLETED

Research Site

Wuhan, China

COMPLETED

Research Site

Horsens, Denmark

COMPLETED

Research Site

Berlin, Germany

COMPLETED

Research Site

Tokyo, Japan

COMPLETED

Research Site

Lisbon, Portugal

RECRUITING

Research Site

Porto, Portugal

NOT YET RECRUITING

Research Site

A Coruña, Spain

COMPLETED

Research Site

Barcelona, Spain

COMPLETED

Research Site

Bilbao, Spain

COMPLETED

Research Site

El Palmar, Spain

COMPLETED

Research Site

Huelva, Spain

COMPLETED

Research Site

Las Palmas de Gran Canaria, Spain

COMPLETED

Research Site

Madrid, Spain

COMPLETED

Research Site

Majadahonda, Spain

COMPLETED

Research Site

Palma de Mallorca, Spain

COMPLETED

Research Site

Salamanca, Spain

COMPLETED

Research Site

Valencia, Spain

COMPLETED

Research Site

Södertälje, Sweden

COMPLETED

Research Site

Umeå, Sweden

COMPLETED

Research Site

London, United Kingdom

COMPLETED

Related Publications (1)

• Gillmore JD, Hahn K, Smith JG, Conceicao I, Tian Z, Grogan M, Pao C, Wittbrodt E, Jarbrink K, Papas MA, Davis MK. Rationale and Design of ANTHOLOGY: An ATTR Amyloidosis Real-World Evidence Program Aiming to Address Gaps in Amyloidosis Care. Cardiol Ther. 2025 Sep;14(3):477-490. doi: 10.1007/s40119-025-00402-y. Epub 2025 Mar 19.

  PMID: 40108078 DERIVED

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

1-877-240-9479; information.center@astrazeneca.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 12, 2024
• First Posted: April 10, 2024
• Study Start: August 21, 2023
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: April 23, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• Access Criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Locations

CN (5); DK (1); DE (1); SE (2); GB (1); PT (2); CA (1); JP (1); US (1); ES (11)