Transthyretin Amyloid CardiomyopathyNCT07690436
Retinal Fundus Imaging and OCT Imaging for Ocular Detection of ATTR-CM
An observational study of Transthyretin Amyloid Cardiomyopathy, run by AstraZeneca at 20 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Amyloid Cardiomyopathy
- How many people
- 500 participants
- Where
- 20 sitesin 5 countries
- Study dates
- 8 Jul 2026to 18 Jun 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
20 sites in 5 countries
United States6 sites, 6 recruiting
- Not yet recruiting
Research Site
Boston, Massachusetts, 02114
- Not yet recruiting
Research Site
Chicago, Illinois, 60611
- Not yet recruiting
Research Site
Germantown, Tennessee, 38138
- Not yet recruiting
Research Site
Jacksonville, Florida, 32224
- Not yet recruiting
Research Site
Palo Alto, California, 94305
- Not yet recruiting
Research Site
Portland, Oregon, 97239-3098
Germany4 sites, 4 recruiting
- Not yet recruiting
Research Site
Berlin, 13353
- Not yet recruiting
Research Site
Essen, 45147
- Not yet recruiting
Research Site
Frankfurt am Main, 60596
- Not yet recruiting
Research Site
Würzburg, 97078
Portugal4 sites, 4 recruiting
- Recruiting
Research Site
Guimarães, 4835-044
- Not yet recruiting
Research Site
Lisbon, 1169-024
- Not yet recruiting
Research Site
Lisbon, 1649-028
- Not yet recruiting
Research Site
Lisbon, 2799-523
Spain3 sites, 3 recruiting
- Not yet recruiting
Research Site
L'Hospitalet de Llobregat, 08907
- Not yet recruiting
Research Site
Madrid, 28040
- Not yet recruiting
Research Site
Palma de Mallorca, 07198
Sweden3 sites, 3 recruiting
- Not yet recruiting
Research Site
Gothenburg, 413 45
- Not yet recruiting
Research Site
Solna, 171 64
- Not yet recruiting
Research Site
Vasterbottens Lan, 907 37
About this study
Official title: A Multicenter Pilot Study to Investigate Ocular Involvement and the Potential of Ocular Imaging for Non-invasive Screening for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
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.
Read the detailed description
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.
- Conditions
- Transthyretin Amyloid Cardiomyopathy
- Keywords
- Transthyretin Amyloid Cardiomyopathy, ATTR-CM, Ocular Imaging, Heart Failure, Machine Learning, Optical coherence tomography, Retinal fundus imaging
- Medical subject headings
- Heart Failure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
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.
Measured: 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
Score distributions generated by the machine learning model will be descriptively compared across study population strata and demographic groups.
Measured: At a single assessment time point within 90 days following informed consent form signing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2026
- Study start8 Jul 2026
- Primary completion18 Jun 2027 (estimated)
- Study completion18 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin Amyloid CardiomyopathyStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07690436
- Sponsor's protocol ID
- D8455R00001
- Status last verified
- Aug 2026
- First submitted
- 2 Jul 2026
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Pilot Study to Investigate Ocular Involvement and the Potential of Ocular Imaging for Non-invasive Screening for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
- ClinicalTrials.gov ID
- NCT07690436
- Acronym / sponsor ID
- D8455R00001
- Conditions
- Transthyretin Amyloid Cardiomyopathy
- Keywords
- Transthyretin Amyloid Cardiomyopathy, ATTR-CM, Ocular Imaging, Heart Failure, Machine Learning, Optical coherence tomography, Retinal fundus imaging
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2026 (actual)
- Primary completion
- 18 Jun 2027 (estimated)
- Study completion
- 18 Jun 2027 (estimated)
- First posted
- 8 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Comparison of per-participant confidence scores distributions between ATTR-CM cases and HF controls without ATTR-CM
Time frame: At a single assessment time point within 90 days following informed consent form signing
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.
Qualitative assessment of features learned indicative of ATTR pathology
Time frame: At a single assessment time point within 90 days following informed consent form signing
Features learned by the machine learning (ML) model that are indicative of transthyretin amyloid pathology will be qualitatively assessed.
2 secondary outcomes
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
Time frame: 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
Time frame: At a single assessment time point within 90 days following informed consent form signing
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
- Individual participant dataSharing planned: Yes. Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
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Effect of Trimetazidine on Coronary Flow in Transthyretin Cardiac Amyloidosis
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PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels
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Active, not recruitingPhase 3
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
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A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
AstraZeneca Clinical Study Information Center contact
20 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Transthyretin Amyloid Cardiomyopathy, run by AstraZeneca at 20 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, Germany, Portugal, Spain, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact AstraZeneca Clinical Study Information Center (1-877-240-9479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Comparison of per-participant confidence scores distributions between ATTR-CM cases and HF controls without ATTR-CM (time frame: At a single assessment time point within 90 days following informed consent form signing). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2026.