International Cardiac Amyloidosis Registry

NCT06894290 | Recruiting

• 1 other identifier: ICAR
• Study Type: observational
• Target: 300
• Locations: 5 countries
• Sites: 6

Brief Summary

Cardiac amyloidosis is a relatively rare disease. However, with the newer imaging techniques that have become available to us in recent years, determining the diagnosis is becoming more common. There are several variants. Each variant involves protein accumulation between heart muscle cells. This leads to the heart stiffening and, as a result, the heart has a hard time filling. This can lead to heart failure with complaints such as fluid retention, cardiac arrhythmias such as atrial fibrillation, conduction abnormalities that sometimes require a pacemaker, and clot formation in the heart that can cause a stroke and narrowing of the aortic valve. Getting the correct diagnosis is important because specific treatment is available in some cases for the different variants of cardiac amyloidosis. This research is needed to better understand the course of this disease profile and which patients respond well to the specific treatment. The aim of this research is to find out more about the course of the cardiac amyloidosis disease. The investigators see to what extent patients deteriorate in their condition due to the disease and how often they need to be hospitalized. Furthermore, the investigators want to learn to what extent certain abnormalities (on e.g. cardiac ultrasound) can predict how quickly clinical deterioration occurs. Finally, for certain forms of cardiac amyloidosis there is a specific drug treatment. Through this research, the investigators want to try to determine who really benefits from the specific medication in order to prescribe it to the right patients.

Conditions

Cardiac Amyloidosis

Keywords

Cardiac amyloidosis; ATTR amyloidosis; AL amyloidosis; Echocardiography

Outcome Measures

Primary Outcomes (1)

• Mortality

  The main study endpoint is all-cause mortality

  1 year

Secondary Outcomes (3)

• Progression

  1 year

• HFH

  1 year

• Events

  1 year

Study Arms (1)

CA

Patients diagnosed with cardiac amyloidosis. Either ATTR-CA or AL-CA.

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 consecutive patients with diagnosis of AL or ATTR diagnosis will be prospectively included from 1st of January 2023 in the registry (if consent is provided). Currently, about 10 patients per year are diagnosed in the LUMC, therefore multicenter cooperation is needed to include sufficient patients. We have a consortium of at least 15 centers that are willing to participate. Therefore we estimate an inclusion of \~150 patients per year. The rate of diagnosis will probably increase with increasing awareness for amyloidosis, with potentially increasing inclusion rates. An estimated 1/3 of patient will have AL and 2/3 will have ATTR amyloidosis.

You may qualify if:

• Diagnosed with AL or ATTR cardiac amyloidosis following the recommendations of the European Society of Cardiology \[1\] which include:
• Signs and symptoms, ECG, echo or CMR suggestive of cardiac amyloidosis and
• negative hematological test and bone scintigraphy Perugini stage 2 or 3 (is diagnostic for ATTR amyloidosis)
• negative hematological test and bone scintigraphy Perugini stage 1 and histological confirmation (either extracardiac or cardiac biopsy) with subtyping of ATTR amyloidosis
• positive hematological test and bone scintigraphy Perugini stage 1-3 and histological confirmation (usually cardiac biopsy) with subtyping of ATTR amyloidosis
• positive hematological rest and bone scintigraphy Perugini stage 0, MRI consistent with amyloidosis and histological confirmation (either extracardiac or cardiac biopsy) with subtyping AL amyloidosis

You may not qualify if:

• Unable to provide informed consent due to insufficient language proficiency or intellectual capabilities

Sponsors & Collaborators

• Leiden University Medical Center: lead
• Pfizer: collaborator

Study Sites (6)

AZ St Jan

Bruges, 8000, Belgium

RECRUITING

UZ Antwerpen

Edegem, 2650, Belgium

RECRUITING

Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Roma

Roma, 00168, Italy

RECRUITING

Kitasato University Hospital

Sagamihara, 252-0375, Japan

RECRUITING

Leiden University Medical Center

Leiden, 2333ZA, Netherlands

RECRUITING

Santa Marta Hospital, CHULC, Lisbon

Lisbon, 1169-024, Portugal

RECRUITING

Related Publications (3)

• Bukhari S, Khan SZ, Bashir Z. Atrial Fibrillation, Thromboembolic Risk, and Anticoagulation in Cardiac Amyloidosis: A Review. J Card Fail. 2023 Jan;29(1):76-86. doi: 10.1016/j.cardfail.2022.08.008. Epub 2022 Sep 17.

  PMID: 36122817 BACKGROUND

• Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27.

  PMID: 30145929 BACKGROUND

• Garcia-Pavia P, Rapezzi C, Adler Y, Arad M, Basso C, Brucato A, Burazor I, Caforio ALP, Damy T, Eriksson U, Fontana M, Gillmore JD, Gonzalez-Lopez E, Grogan M, Heymans S, Imazio M, Kindermann I, Kristen AV, Maurer MS, Merlini G, Pantazis A, Pankuweit S, Rigopoulos AG, Linhart A. Diagnosis and treatment of cardiac amyloidosis. A position statement of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases. Eur J Heart Fail. 2021 Apr;23(4):512-526. doi: 10.1002/ejhf.2140. Epub 2021 Apr 7.

  PMID: 33826207 BACKGROUND

MeSH Terms

Conditions

Amyloid Neuropathies, Familial; Immunoglobulin Light-chain Amyloidosis

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; Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias

Study Officials

• Nina Ajmone Marsan, MD, PhD

  Leiden University Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nina Ajmone Marsan, MD, PhD

CONTACT

+31715262020; N.ajmone@lumc.nl

Madelien Regeer, MD, PhD

CONTACT

+31715262020; m.v.regeer@lumc.nl

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: March 12, 2025
• First Posted: March 25, 2025
• Study Start: June 30, 2023
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026
• Last Updated: March 25, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

BE (2); JP (1); IT (1); PT (1); NL (1)