Retinal Fundus Imaging and OCT Imaging for Ocular Detection of ATTR-CM

NCT07690436 | Not Yet Recruiting

• 1 other identifier: D8455R00001
• Study Type: observational
• Target: 500
• Locations: 4 countries
• Sites: 10

Brief Summary

This multicenter pilot study will enroll adult participants with a confirmed clinical diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) and heart failure (HF) controls with documentation within the past 2 years that either excludes ATTR-CM or indicates a low probability of ATTR-CM. Ocular imaging and other study data will be collected to assess the feasibility of developing and preliminarily evaluating a machine learning model to discriminate ATTR-CM cases from HF controls without ATTR-CM.

Conditions

Transthyretin Amyloid Cardiomyopathy

Keywords

Transthyretin Amyloid Cardiomyopathy; ATTR-CM; Ocular Imaging; Heart Failure; Machine Learning; Optical coherence tomography; Retinal fundus imaging

Outcome Measures

Primary Outcomes (2)

• Comparison of per-participant confidence scores distributions between ATTR-CM cases and HF controls without ATTR-CM

  Per-participant confidence score distributions generated by the machine learning (ML) model will be compared between participants with clinically confirmed transthyretin amyloid cardiomyopathy (ATTR-CM) and heart failure controls without ATTR-CM.

  At a single assessment time point within 90 days following informed consent form signing

• Qualitative assessment of features learned indicative of ATTR pathology

  Features learned by the machine learning (ML) model that are indicative of transthyretin amyloid pathology will be qualitatively assessed.

  At a single assessment time point within 90 days following informed consent form signing

Secondary Outcomes (2)

• Sensitivity, specificity, and Area Under the Receiver Operating Characteristic Curve (AUROC) of the ML model to correctly identify ATTR-CM cases based on ocular imaging data

  At a single assessment time point within 90 days following informed consent form signing

• Descriptive comparison of score distributions across study population strata and demographics

  At a single assessment time point within 90 days following informed consent form signing

Study Arms (2)

ATTR-CM

Participants with a confirmed clinical diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM).

HF Control

Participants with guideline-directed medical therapy-directed heart failure and documentation within the past 2 years that either excludes transthyretin amyloid cardiomyopathy (ATTR-CM) or indicates a low probability of ATTR-CM.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will comprise adult participants with a confirmed clinical diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) and heart failure (HF) controls who have documentation within the past 2 years that either excludes ATTR-CM or indicates a low probability of ATTR-CM.

You may qualify if:

• \. Participant must be 18 years and older at the time of signing the informed consent form.
• \. Participants with either of the following:
• \- Positive ATTR-CM cases: Participants with a clinical diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) with amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining, or DPD-Tc, PYP-Tc, or HMDP-Tc scintigraphy with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio.
• \- Control population: Participants with a clinical diagnosis of guideline-directed medical therapy-directed heart failure (HF) and either:
• a) Subgroup A: Definitively excluded ATTR-CM amyloidosis: negative cardiac or non-cardiac tissue biopsy for amyloid, or negative technetium scintigraphy within the past 2 years.
• b) Subgroup B: Low probability of ATTR-CM amyloidosis, not definitively excluded: absence of amyloid-suggestive features on echocardiography (ECHO), electrocardiography (ECG), or cardiac magnetic resonance imaging (MRI) within the past 2 years, with documented clinical assessment indicating HF etiology unlikely attributable to amyloidosis; no amyloid-specific testing (technetium scintigraphy or biopsy) performed.
• \. Participant or legally authorized representative (LAR) must sign the informed consent form.

You may not qualify if:

• \. Any known eye condition that may preclude clear imaging of the retina.
• \. Any known history of amyloid light-chain (AL) amyloidosis.
• \. Any ocular surgery that, in the opinion of the investigator, makes participation in the study undesirable.
• \. Any medical condition that, in the opinion of the investigator, makes participation in the study undesirable, for example, if the participant is critically unwell or requires ongoing emergency treatment.
• \. Involvement in the planning and/or conduct of the study.
• \. In the opinion of the investigator, the participant is unlikely to comply with study procedures, restrictions, and requirements.
• \. Previous enrollment in the present study.

Sponsors & Collaborators

• AstraZeneca: lead

Study Sites (10)

Research Site

Palo Alto, California, 94305, United States

Location

Research Site

Chicago, Illinois, 60611, United States

Location

Research Site

Boston, Massachusetts, 02114, United States

Location

Research Site

Portland, Oregon, 97239-3098, United States

Location

Research Site

Germantown, Tennessee, 38138, United States

Location

Research Site

Essen, 45147, Germany

Location

Research Site

Würzburg, 97078, Germany

Location

Research Site

Guimarães, 4835-044, Portugal

Location

Research Site

L'Hospitalet de Llobregat, 08907, Spain

Location

Research Site

Palma de Mallorca, 07198, Spain

Location

MeSH Terms

Conditions

Heart Failure

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

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

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 2, 2026
• First Posted: July 8, 2026
• Study Start: July 2, 2026
• Primary Completion (Estimated): June 3, 2027
• Study Completion (Estimated): June 3, 2027
• Last Updated: July 8, 2026

Record last verified: 2026-07

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

US (5); PT (1); ES (2); DE (2)