Dilated cardiomyopathy 1HH
MONDO:0013479Any familial isolated dilated cardiomyopathy in which the cause of the disease is a mutation in the BAG3 gene.
Also known as: BAG3 familial isolated dilated cardiomyopathy, CMD1HH, cardiomyopathy, dilated, type 1Hh, dilated cardiomyopathy type 1HH, familial isolated dilated cardiomyopathy caused by mutation in BAG3, cardiomyopathy, dilated, 1HH
13 clinical trials for this condition and its sub-types, 5 tagged with Dilated cardiomyopathy 1HH itself.
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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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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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Tiny study could unlock secrets of genetic heart failure
Knowledge-focused Recruiting nowThis study follows 10 Chinese adults who have a specific gene mutation (BAG3) linked to dilated cardiomyopathy, a condition where the heart becomes enlarged and weak. Researchers will track changes in heart function, symptoms, and biomarkers over time to better understand how the…
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC