Transthyretin AmyloidosisEarly Phase 1NCT04569903
Evaluation of a Claims-based Algorithm for the Identification of Transthyretin-mediated Amyloidosis (ATTR) Amyloidosis in Medical Records
A Early Phase 1 study testing Computer algorithm for ATTR in people with Transthyretin Amyloidosis, run by Yale University at 1 site in United States.
Trial summary
The primary objective of this study is to evaluate the diagnostic performance of an algorithm in identifying patients with ATTR amyloidosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Amyloidosis
- What is being tested?
- Computer algorithm for ATTR
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2022to 1 Feb 2025
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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
Who may be able to join
- Identified by the ATTR diagnostic algorithm and matched by Yale's list of potential subjects defined as:
- subjects within the claims dataset that are predicted to be at risk of having ATTR who are also being managed within YNHHS
- patients who need to be contacted and offered additional clinical evaluation to determine whether they have a diagnosis of ATTR (non-hereditary or Hereditary ATTR amyloidosis).
Who cannot join
- Patients who have opted out of research in the Epic system will be excluded entirely from the study
- Patients who are pregnant or who may become pregnant
Trial locations and contacts
1 site in 1 country.
United States1 site
Yale New Haven Hospital
New Haven, Connecticut, 06520
About this study
Official title: Evaluation of a Claims-based Algorithm for the Identification of ATTR Amyloidosis in Medical Records
The primary objective of this study is to evaluate the diagnostic performance of an algorithm in identifying patients with ATTR amyloidosis.
Read the detailed description
A screening strategy to identify ATTR in the large background population of patients with one or more common ATTR manifestations, would be of significant clinical value.
In addition, novel ATTR therapies have been recently made available or are currently in development in late-stage clinical trials. As early diagnosis and treatment is expected to achieve better outcomes, this makes the development and validation of an easily implemented, rapid and electronically-enabled diagnostic algorithm especially important.
A medical and pharmacy claims-based algorithm was developed to potentially identify patients at risk of having ATTR. The goal of this study is to evaluate the ability of the algorithm to identify patients with ATTR by performing diagnostic clinical work up in patients that the algorithm identifies in a large dataset of patients at Yale.
The primary objective of this study is to evaluate the diagnostic performance of the algorithm in identifying patients with ATTR amyloidosis.
The secondary objective of this study is to estimate the clinical benefit of the algorithm, as measured by the added diagnostic value, i.e. the proportion or rate of patients who were previously undiagnosed. The total obtained prevalence will be assessed and informally compared to the referral-based prevalence of ATTR amyloidosis patients at Yale.
- Conditions
- Transthyretin Amyloidosis
- Being tested
- Computer algorithm for ATTR
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Computer algorithm for ATTRDevice
Patients will be evaluated for the identification of ATTR Amyloidosis through a claims-based algorithm
| Study group | Role | What they receive |
|---|---|---|
| Computer algorithm for ATTR | Experimental | Patients will be evaluated for the identification of ATTR Amyloidosis through a claims-based algorithm Receives: Device: Computer algorithm for ATTR |
What is being measured
The primary outcome is the main question the study is designed to answer.
Diagnostic performance of algorithm in identifying patients with ATTR amyloidosis
Potential thresholds for defining diagnostic positivity based on the calculated algorithmic scores will be explored and the corresponding positive predictive value (PPV) will serve as indicator for the diagnostic performance. Negative predictive values (NPV) may be explored if the actual distribution of score data will allow for it.
Measured: 2 years
Secondary outcomes (1)
Proportion of diagnosed patients
The proportion or rate of patients who were previously undiagnosed of ATTR Amyloidosis
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2020
- Study start1 Aug 2022
- Primary completion1 Feb 2025
- Study completion1 Feb 2025
Why it stopped: No patients consented to enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin AmyloidosisStudies of the same condition, open and under way.
- More trials run by Yale UniversityThe 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
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Yale University
- Sponsor type
- University, hospital or other
- Collaborators
- Alnylam Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Edward Miller, MD, Principal investigator, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04569903
- Sponsor's protocol ID
- 2000026611
- Other IDs
- 000
- Status last verified
- Sep 2025
- First submitted
- 24 Sep 2020
- Last updated
- 18 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of a Claims-based Algorithm for the Identification of ATTR Amyloidosis in Medical Records
- ClinicalTrials.gov ID
- NCT04569903
- Acronym / sponsor ID
- 2000026611
- Conditions
- Transthyretin Amyloidosis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No patients consented to enrollment
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Yale UniversityUniversity, hospital or other
- Collaborators
- Alnylam Pharmaceuticals
- Study start
- 1 Aug 2022 (actual)
- Primary completion
- 1 Feb 2025 (actual)
- Study completion
- 1 Feb 2025 (actual)
- First posted
- 30 Sep 2020 (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.
The primary objective of this study is to evaluate the diagnostic performance of an algorithm in identifying patients with ATTR amyloidosis.
Detailed description
A screening strategy to identify ATTR in the large background population of patients with one or more common ATTR manifestations, would be of significant clinical value.
In addition, novel ATTR therapies have been recently made available or are currently in development in late-stage clinical trials. As early diagnosis and treatment is expected to achieve better outcomes, this makes the development and validation of an easily implemented, rapid and electronically-enabled diagnostic algorithm especially important.
A medical and pharmacy claims-based algorithm was developed to potentially identify patients at risk of having ATTR. The goal of this study is to evaluate the ability of the algorithm to identify patients with ATTR by performing diagnostic clinical work up in patients that the algorithm identifies in a large dataset of patients at Yale.
The primary objective of this study is to evaluate the diagnostic performance of the algorithm in identifying patients with ATTR amyloidosis.
The secondary objective of this study is to estimate the clinical benefit of the algorithm, as measured by the added diagnostic value, i.e. the proportion or rate of patients who were previously undiagnosed. The total obtained prevalence will be assessed and informally compared to the referral-based prevalence of ATTR amyloidosis patients at Yale.
Study design
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Computer algorithm for ATTR | Experimental | Device: Computer algorithm for ATTRPatients will be evaluated for the identification of ATTR Amyloidosis through a claims-based algorithm |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Identified by the ATTR diagnostic algorithm and matched by Yale's list of potential subjects defined as:
Key exclusion criteria (2 listed)
- Patients who have opted out of research in the Epic system will be excluded entirely from the study
- Patients who are pregnant or who may become pregnant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Computer algorithm for ATTRPatients will be evaluated for the identification of ATTR Amyloidosis through a claims-based algorithm | Device | Computer algorithm for ATTR |
Primary outcome
Diagnostic performance of algorithm in identifying patients with ATTR amyloidosis
Time frame: 2 years
Potential thresholds for defining diagnostic positivity based on the calculated algorithmic scores will be explored and the corresponding positive predictive value (PPV) will serve as indicator for the diagnostic performance. Negative predictive values (NPV) may be explored if the actual distribution of score data will allow for it.
1 secondary outcome
Proportion of diagnosed patients
Time frame: 2 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2025.
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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. This study is not recruiting at the moment.
Edward Miller, MD principal investigator
Yale University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Computer algorithm for ATTR in people with Transthyretin Amyloidosis, run by Yale University at 1 site in United States.
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 3 inclusion and 2 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: withdrawn, as of the sponsor's last update on 18 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Diagnostic performance of algorithm in identifying patients with ATTR amyloidosis (time frame: 2 years). 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 18 Sep 2025.