PL_GNT01_ISR_Grant 53234273

NCT06714019 | Enrolling By Invitation

• 1 other identifier: 53234273
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

We carry out a prospective non-interventional, longitudinal, observational multicentre registry designed to improve our understanding of the epidemiology of TTR amyloidosis in our country. The main objective of the proposed study is to determine the occurrence of TTR amyloidosis and describe clinical profile of patients in the population of our country.

Conditions

Transthyretin Cardiomyopathy (TTR-CM); Transthyretin (TTR) Amyloid Cardiomyopathy; Transthyretin Amyloidosis

Keywords

transthyretin; transthyretin amyloidosis; ATTR; Transthyretin amyloid cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• disease progression

  Signs and symptoms of the disease are evaluated, general examinations are conducted, and laboratory data, measures of neurologic and cardiovascular function, and quality of life are assessed according to the standard of care for patients.

  From enrollment for at least 12 month

Eligibility Criteria

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

Study Population

All individuals with wild-type TTR amyloidosis and patients with a confirmed TTR mutation with or without a diagnosis of TTR amyloidosis will be eligible to be enrolled in the registry. Enrolment is voluntary and dependent on each subject's written informed consent.

You may qualify if:

• Adults over 18 years old with confirmed diagnosis of TTR amyloidosis

You may not qualify if:

• Refusal to participate in the study. Light-chain amyloidosis.

Sponsors & Collaborators

• National Institute of Cardiology, Warsaw, Poland: lead
• Pfizer: collaborator

Study Sites (1)

National Institute of Cardiology

Warsaw, 04-628, Poland

Location

MeSH Terms

Conditions

Amyloid Neuropathies, Familial; Amyloidosis, Hereditary, Transthyretin-Related

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Target Duration: 12 Months
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PhD

Study Record Dates

• First Submitted: November 27, 2024
• First Posted: December 3, 2024
• Study Start: February 11, 2019
• Primary Completion: October 1, 2025
• Study Completion: November 1, 2025
• Last Updated: December 3, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

PL (1)