Clinical trial NCT04569903: Evaluation of a Claims-based Algorithm for the Identification of Transthyretin-mediated Amyloidosis (ATTR) Amyloidosis in Medical Records
Official title: Evaluation of a Claims-based Algorithm for the Identification of ATTR Amyloidosis in Medical Records
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Transthyretin Amyloidosis
Lead sponsor: Yale University
Sponsor class: OTHER
Collaborators: name: Alnylam Pharmaceuticals Inc
First posted: 2020-09-30
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SCREENING

Brief summary: 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.

Eligibility criteria: Inclusion Criteria:

* Identified by the ATTR diagnostic algorithm and matched by Yale's list of potential subjects defined as:

  1. subjects within the claims dataset that are predicted to be at risk of having ATTR who are also being managed within YNHHS
  2. 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).

     Exclusion Criteria:
* 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

Primary outcomes:
measure: Diagnostic performance of algorithm in identifying patients with ATTR amyloidosis
timeFrame: 2 years
Secondary outcomes:
measure: Proportion of diagnosed patients
timeFrame: 2 years
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Yale New Haven Hospital
state: Connecticut
Study officials:
affiliation: Yale University
name: Edward Miller, MD
role: PRINCIPAL_INVESTIGATOR
References: