Familial isolated dilated cardiomyopathy
MONDO:0700335A rare familial cardiomyopathy characterized by the dilation of left ventricle and progressively impairing of systolic ventricular function, in the absence of abnormal loading conditions or coronary artery disease sufficient to cause global systolic impairment. The disease may cause heart failure or arrhythmia. The disease is isolated when no additional atypical cardiac or extracardiac manifestations are present.
Also known as: familial isolated dilated cardiomyopathy, familial or idiopathic dilated cardiomyopathy
19 clinical trials for this condition and its sub-types.
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Broader categories
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New drug trial targets rare genetic heart disease
Disease control Recruiting nowThis early-stage study tests a new medicine called AZD4063 in 31 adults with a specific genetic form of dilated cardiomyopathy (PLN R14del). The main goal is to check the drug's safety and how the body processes it. Participants receive the drug as an injection under the skin and…
Phase 1 • Sponsor: AstraZeneca • Aim: Disease control
Last updated Sep 06, 2026 00:00 UTC
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One-Time gene infusion aims to repair hearts in rare genetic condition
Disease control Recruiting nowThis early-stage trial tests a single intravenous dose of AFTX-201, a gene therapy designed to deliver a working copy of the BAG3 gene to heart cells. It enrolls 22 adults aged 18–55 with dilated cardiomyopathy and a BAG3 mutation. The study primarily checks safety and tolerabili…
Phase 1/2 • Sponsor: Affinia Therapeutics • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC
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New gene therapy trial hopes to strengthen hearts in rare genetic condition
Disease control Recruiting nowThis early-stage study tests a one-time gene therapy called ALXN2350 for adults with a specific genetic heart condition (BAG3-related dilated cardiomyopathy). The heart muscle becomes weak and enlarged, making it hard to pump blood. The treatment aims to fix the faulty gene to im…
Phase 1/2 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
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Gene therapy trial aims to fix heart muscle in rare genetic disease
Disease control Recruiting nowThis early-stage trial tests a single dose of a gene therapy called NVC-001 in 21 adults with a genetic form of dilated cardiomyopathy (LMNA mutation). The therapy uses a harmless virus to deliver a modified gene into heart cells, aiming to protect the heart muscle. The main goal…
Phase 1/2 • Sponsor: Nuevocor Pte. Ltd. • Aim: Disease control
Last updated Aug 09, 2026 00:00 UTC
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One-Time gene shot aims to mend broken hearts
Disease control Recruiting nowThis early-stage trial tests a one-time gene therapy called RP-A701 in 8 adults with dilated cardiomyopathy caused by a faulty BAG3 gene. The therapy uses a harmless virus to deliver a working copy of the gene, aiming to improve heart function. The main goal is to check safety, b…
Phase 1 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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Heart scans may unlock hidden risks in minority communities
Knowledge-focused Recruiting nowThis study is looking at whether advanced heart imaging, especially cardiac MRI, can help identify the cause of cardiomyopathy and predict risks like death or heart failure in a diverse group of patients. The research focuses on adults in the Bronx, including many from Hispanic a…
Sponsor: Montefiore Medical Center • Aim: Knowledge-focused
Last updated Aug 12, 2026 00:00 UTC
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10,000 heart patients join online registry to unlock secrets of cardiomyopathy
Knowledge-focused Recruiting nowThis study is building a large online registry of 10,000 adults with cardiomyopathy or myocarditis, plus those with a strong family history. Researchers will track participants over time to see how many experience serious events like heart failure, dangerous heart rhythms, or dea…
Sponsor: Imperial College London • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:01 UTC
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AI could help predict sudden cardiac death in hereditary heart disease patients
Knowledge-focused Recruiting nowThis study aims to improve how doctors predict the risk of sudden cardiac death or heart failure in people with inherited heart diseases. Researchers will use artificial intelligence to analyze data from 1,000 participants, including medical history, ECGs, imaging, genetic tests,…
Sponsor: Nantes University Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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Scientists hunt for 'Modifier Genes' that could explain why some LMNA patients fare better than others
Knowledge-focused Recruiting nowThis study aims to identify genetic factors that affect how severe muscle and heart problems become in people with LMNA gene mutations. Researchers will collect skin and muscle samples from 40 participants and use advanced DNA and RNA analysis to look for protective or aggravatin…
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:10 UTC
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Can we predict who will die suddenly from heart problems?
Knowledge-focused Recruiting nowThis study follows 1500 people who already have an implantable cardioverter-defibrillator (ICD) to prevent sudden cardiac death. Researchers will track heart function, genetics, and blood markers to find better ways to predict who is at highest risk. The goal is to improve how do…
Sponsor: Johns Hopkins University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:13 UTC
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Heart tissue analysis aims to unlock better diagnosis
Knowledge-focused Recruiting nowThis study looks at heart tissue samples from 216 adults who already need a biopsy as part of their care. Researchers want to find unique molecular patterns in different heart conditions, including after a heart transplant. The goal is to improve how these diseases are diagnosed …
Sponsor: University Hospital, Essen • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:12 UTC