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

Официальное название

A Multicenter Pilot Study to Investigate Ocular Involvement and the Potential of Ocular Imaging for Non-invasive Screening for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Обзор

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.

Подробное описание

A participant who does not complete the study within 90 days from enrollment will be discontinued from the study. A participant may be discontinued from the study at any time at the discretion of the investigator for behavioral or compliance reasons. A participant may withdraw from the study at any time at the participant's own request for any reason (or without providing any reason) without any implication on participant's rights.

Первичные конечные точки

• Comparison of per-participant confidence scores distributions between ATTR-CM cases and HF controls without ATTR-CM [Срок оценки: At a single assessment time point within 90 days following informed consent form signing]
• Qualitative assessment of features learned indicative of ATTR pathology [Срок оценки: At a single assessment time point within 90 days following informed consent form signing]

Вторичные конечные точки (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 [Срок оценки: 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 [Срок оценки: At a single assessment time point within 90 days following informed consent form signing]

Критерии участия

Критерии включения

• 1\. Participant must be 18 years and older at the time of signing the informed consent form.
• 2\. 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.
• 3\. Participant or legally authorized representative (LAR) must sign the informed consent form.

Критерии исключения

• 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.
• 5\. Involvement in the planning and/or conduct of the study.
• 6\. In the opinion of the investigator, the participant is unlikely to comply with study procedures, restrictions, and requirements.
• 7\. Previous enrollment in the present study.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Случай-контроль

Центры проведения

США · 5 центров

• Research Site — Palo Alto
• Research Site — Chicago
• Research Site — Boston
• Research Site — Portland
• Research Site — Germantown

Германия · 2 центра

• Research Site — Essen
• Research Site — Würzburg

Испания · 2 центра

• Research Site — L'Hospitalet de Llobregat
• Research Site — Palma de Mallorca

Португалия · 1 центр

• Research Site — Guimarães

Идентификаторы

NCT: NCT07690436 · D8455R00001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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