Retinal Fundus Imaging and OCT Imaging for Ocular Detection of ATTR-CM
ConditionTransthyretin Amyloid Cardiomyopathy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorAstraZeneca
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
1. Participant must be 18 years and older at the time of signing the informed consent form.
- 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.
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.
Disqualifiers
1. Any known eye condition that may preclude clear imaging of the retina.
2. Any known history of amyloid light-chain (AL) amyloidosis.
3. Any ocular surgery that, in the opinion of the investigator, makes participation in the study undesirable.
4. 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.
Trial 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.
Trial design
Design model
Case-control
Time perspective
Cross-sectional
Treatments tested in this trial
Not listed
Trial groups
500 Participants
are grouped into 2 trial groupsGroup A: ATTR-CM
Group B: HF Control
Trial outcomes
Primary outcomes
1
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.
Time frame
At a single assessment time point within 90 days following informed consent form signing2
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.
Time frame
At a single assessment time point within 90 days following informed consent form signingSecondary outcomes
1
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
Sensitivity, specificity, and area under the receiver operating characteristic curve will be used to describe the performance of the machine learning model for distinguishing participants with clinically confirmed transthyretin amyloid cardiomyopathy (ATTR-CM) from heart failure controls without ATTR-CM based on ocular imaging data.
Time frame
At a single assessment time point within 90 days following informed consent form signing2
Descriptive comparison of score distributions across study population strata and demographics
Score distributions generated by the machine learning model will be descriptively compared across study population strata and demographic groups.
Time frame
At a single assessment time point within 90 days following informed consent form signingSponsors and contacts
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Source ClinicalTrials.gov