A PILOT Study on LSGB vs EMB in the Diagnosis of Cardiac Transthyretin Amyloidosis

NCT05375279 | Unknown

• 1 other identifier: 70095215
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Transthyretin (TTR) is a plasma protein mainly synthesized in the liver, recognized as a transporter of thyroxine and retinol-binding protein. Unstable changes in two types of TTR (wild type or variant) become misfolded, aggregate, and ultimately forms amyloid fibrils. Amyloid Transthyretin Cardiac amyloidosis (ATTR-CA) is an infiltrative cardiomyopathy caused by extracellular deposition of insoluble transthyretin (TTR) amyloid fibrils in the heart muscle. Cardiac amyloidosis (CA) has been recognized as a common cause of heart failure with preserved ejection fraction (HFpEF) among elderly persons, with increasing incidence. There are different ways of diagnosing ATTR-CA. These include cardiac magnetic resonance imaging, nuclear scintigraphy, and tissue biopsy, the gold standard. Tissues biopsy extracted from the adipose, lip salivary gland (LSG), and heart muscle (endomyocardial biopsy or EMB). Tissue diagnosis is the prerequisite of provincial support of the disease-modifying agent. However, with the convenience, ease, less risk of bleeding, and high sensitivity, LSG may offer an alternative to the more invasive EMB to diagnose suspected CA. To test the hypothesis that LSGB can replace EMB in tissue diagnosis of ATTR-CM. This Pilot study is designed to evaluate two invasive diagnostic methodologies: LSGB and the EMB. A total of 20 patients who underwent EMB within the last six months with confirmed Amyloid Transthyretin -wild type (ATTRwT) will be invited to participate. In addition, patients who signed the informed consent form will be scheduled for LSGB within two weeks.

Conditions

Amyloidosis

Keywords

Amyloidosis; Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• LSG biopsy with positive amyloid deposits will be observed in a higher proportion in patients with positive EMB.

  Proportions of patients who will test positive and negative will be determined.

  6 months

Study Arms (1)

LSGB vs EMB

Patients who already underwent EMB within the last six months with confirmed Amyloid Transthyretin -wild type (ATTRwT) will undergo LSGB.

Procedure: Lip Salivary Gland Biopsy

Interventions

Lip Salivary Gland Biopsy PROCEDURE

Obtaining few salivary glands from the partipants' lip that will be studied using the Congo Red staining method.

LSGB vs EMB

Eligibility Criteria

• Age: 18 Years - 96 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients who are confirmed diagnosis of Amyloid Transthyretin Cardiomyopathy - wild type (ATTRwt) through Echocardiography, Cardiac Magnetic Resonance Imaging, pyrophosphate scan, and genetic testing and had an endomyocardial biopsy (or will undergo endomyocardial biopsy).

You may qualify if:

• Patients who are confirmed diagnosis of Amyloid Transthyretin Cardiomyopathy - wild type (ATTRwt) through Echocardiography, Cardiac Magnetic Resonance Imaging, pyrophosphate scan, and genetic testing and had an endomyocardial biopsy (or will undergo endomyocardial biopsy).

You may not qualify if:

• Patients who had negative amyloid deposits in the endomyocardial biopsy.

Sponsors & Collaborators

• Unity Health Toronto: lead
• Pfizer: collaborator

Study Sites (1)

London Health Sciences Centre

London, Ontario, N6A 5A5, Canada

Location

MeSH Terms

Conditions

Amyloidosis; Cardiomyopathies

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Heart Diseases; Cardiovascular Diseases

Study Officials

• Gordon W Moe, MD, FRCPC, FACC

  Unity Health Toronto

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gordon W. Moe, MD, FRCPC, FACC

CONTACT

416-864-5192; Gordon.Moe@unityhealth.to

Carlos Fernando, MD, MSc, MBA

CONTACT

416-864-6060; Carlos.Fernando@unityhealth.to

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Investigator

Study Record Dates

• First Submitted: May 10, 2022
• First Posted: May 16, 2022
• Study Start: June 15, 2022
• Primary Completion: December 31, 2022
• Study Completion: March 31, 2023
• Last Updated: May 23, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)