Early Identification and Treatment of Rare Cardiomyopathy Cohorts

NCT06794710 | Not Yet Recruiting

• 1 other identifier: RARE2024
• Study Type: interventional
• Target: 300
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to further develop an imaging-guided cohort of rare cardiomyopathies based on the existing database. The investigators will standardize the construction of a cohort that integrates a clinical data repository, serum biobank, myocardial tissue bank, and imaging database. In the current cohort, the investigators will systematically screen for biomarkers indicative of pathological changes in challenging cardiomyopathies. Multidimensional data will be integrated to establish and optimize a heart failure risk assessment model, which will then be validated in a prospective cohort. The effectiveness of the model in assessing different risk groups will be evaluated, with the goal of achieving precise prevention of heart failure from the source.

Conditions

Hypertrophic Cardiomyopathy (HCM); Dilated Cardiomyopathy (DCM); Metabolic Cardiomyopathy; Restrictive Cardiomyopathy

Keywords

rare cardiomyopathy, cardiac multimodal imaging, myocardial impairment

Outcome Measures

Primary Outcomes (1)

• The Heart Failure Incidence of Rare cardiomyopathy

  The heart failure incidence will be diagnosed by identify biomarkers combined multimodal imaging with clinical data, blood samples, myocardial tissue samples.

  From the date of recruitment, heart failure will be assessed within 24 hours, followed by assessments every six months during the follow-up period, up to 24 months.

Secondary Outcomes (4)

• Assessment of Changes in Cardiac Morphological Characteristics

  From the date of recruitment, heart failure will be assessed within 24 hours, followed by assessments every six months during the follow-up period, up to 24 months.

• Quantitative Assessment of Changes in Cardiac Tissue Characteristics

  From the date of recruitment, heart failure will be assessed within 24 hours, followed by assessments every six months during the follow-up period, up to 24 months.

• NT-proBNP

  From the date of recruitment, heart failure will be assessed within 24 hours, followed by assessments every six months during the follow-up period, up to 24 months.

• VO2max

  From the date of recruitment, heart failure will be assessed within 24 hours, followed by assessments every six months during the follow-up period, up to 24 months.

Study Arms (2)

Non-model-based prediction group

PLACEBO COMPARATOR

Heart Faliure Patient Group without diagnosed by multimodal imaging, and they will receive a traditional pharmacological treatment for heart failure.Traditional anti heart failure drug therapy included diuretics, angiotensin-converting enzyme inhibitors (ACEIs)/angiotensin receptor antagonists (ARBs), beta blockers, positive inotropic drugs, aldosterone receptor blockers (MRA), sodium glucose cotransporter 2 inhibitors (SGLT2i), and retinoids.

Drug: diuretics, ACEIs/ARBs, beta blockers, positive inotropic drugs, MRAs, SGLT2i, retinoids

Model-guided optimized treatment

EXPERIMENTAL

Patients with heart failure who were diagnosed in Rare Cardiomypathy by multimodal imaging. And They will receive Close follow-up, intensified pharmacological treatment for heart failure, and early rehabilitation guidance. Traditional anti heart failure drug therapy: diuretics, angiotensin-converting enzyme inhibitors (ACEIs)/angiotensin receptor antagonists (ARBs), beta blockers, positive inotropic drugs, aldosterone receptor blockers (MRA), sodium glucose cotransporter 2 inhibitors (SGLT2i), and retinoids.

Drug: diuretics, ACEIs/ARBs, beta blockers, positive inotropic drugs, MRAs, SGLT2i, retinoids; Behavioral: Close follow-up; Behavioral: early rehabilitation guidance

Interventions

diuretics, ACEIs/ARBs, beta blockers, positive inotropic drugs, MRAs, SGLT2i, retinoids DRUG

Patients in this group will receive pharmacological treatment for heart failure.

Also known as: pharmacological treatment for heart failure

Model-guided optimized treatment; Non-model-based prediction group

Close follow-up BEHAVIORAL

High risk patients receive close follow-up

Model-guided optimized treatment

early rehabilitation guidance BEHAVIORAL

Early rehabilitation guidance such as cardiopulmonary exercise tests and cardiac rehabilitation therapy

Model-guided optimized treatment

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18-75 years old.
• Patients preliminarily diagnosed with heart failure and scheduled to receive drug therapy after being evaluated by cardiology departments.
• No history of structural heart disease, and the Framingham score \<5 (for patients with the Framingham score ≥5, coronary artery disease will be excluded by coronary angiography/coronary CT/exercise platelet).
• Creatinine clearance ≥50ml/min (Cockcroft-Gault formula).
• LVEF ≥50% assessed by Echocardiography.
• QT interval \< 470 ms.
• Providing written informed consent.

You may not qualify if:

• Presence of acute/chronic renal impairment (GFR \<50/ml/min/1.73m2).
• History of cardiovascular disease such as confirmed coronary artery disease, valvular disease, cardiomyopathy, congenital heart disease, and heart failure.
• Presence of contraindications to CMR.

Sponsors & Collaborators

• RenJi Hospital: lead

Study Sites (1)

Renji Hospital

Shanghai, Shanghai Municipality, 200030, China

Location

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic; Cardiomyopathy, Dilated; Cardiomyopathy, Restrictive

Interventions

Diuretics; Adrenergic beta-Antagonists; MRAS protein, human; Retinoids; Drug Therapy

Condition Hierarchy (Ancestors)

Cardiomyopathies; Heart Diseases; Cardiovascular Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Aortic Valve Disease; Heart Valve Diseases; Cardiomegaly; Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Natriuretic Agents; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses; Adrenergic Antagonists; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Pigments, Biological; Biological Factors; Therapeutics

Study Officials

• Meng Jiang, MD, PhD

  RenJi Hospital, School of Medicine, Shanghai Jiantong University

  STUDY CHAIR

Central Study Contacts

Meng Jiang, MD, PhD

CONTACT

13788912766; jiangmeng0919@163.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 30, 2024
• First Posted: January 27, 2025
• Study Start: February 1, 2025
• Primary Completion (Estimated): May 30, 2027
• Study Completion (Estimated): September 30, 2027
• Last Updated: January 27, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)