Familial dilated cardiomyopathy
MONDO:0016333A a genetic form of heart disease that occurs when heart (cardiac) muscle becomes thin and weakened in at least one chamber of the heart, causing the open area of the chamber to become enlarged (dilated). As a result, the heart is unable to pump blood as efficiently as usual. To compensate, the heart attempts to increase the amount of blood being pumped through the heart, leading to further thinning and weakening of the cardiac muscle. Over time, this condition results in heart failure.
Also known as: hereditary dilated cardiomyopathy, DCM, dilated cardiomyopathy, familial, hypokinetic dilated cardiomyopathy, familial, idiopathic dilated cardiomyopathy
68 clinical trials for this condition and its sub-types, 7 tagged with Familial dilated cardiomyopathy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Familial dilated cardiomyopathy
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Leber hereditary optic neuropathy 18 trials
1 sub-type
- Leber optic atrophy and dystonia 0 trials
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Familial isolated dilated cardiomyopathy 0 trials · 11 incl. sub-types
45 sub-types
- Dilated cardiomyopathy 1HH 5 trials
- Dilated cardiomyopathy 1A 2 trials
- Dilated cardiomyopathy 1C 1 trial
- Dilated cardiomyopathy 1FF 1 trial
- Dilated cardiomyopathy 1P 1 trial
- Dilated cardiomyopathy 3B 1 trial
- Cardiomyopathy, dilated, 2D 0 trials
- Cardiomyopathy, dilated, 2E 0 trials
- Cardiomyopathy, dilated, 2F 0 trials
- Cardiomyopathy, dilated, 2G 0 trials
- Cardiomyopathy, dilated, 2H 0 trials
- Cardiomyopathy, dilated, 2c 0 trials
- Dilated cardiomyopathy 1AA 0 trials
- Dilated cardiomyopathy 1B 0 trials
- Dilated cardiomyopathy 1BB 0 trials
- Dilated cardiomyopathy 1CC 0 trials
- Dilated cardiomyopathy 1D 0 trials
- Dilated cardiomyopathy 1DD 0 trials
- Dilated cardiomyopathy 1E 0 trials
- Dilated cardiomyopathy 1EE 0 trials
- Dilated cardiomyopathy 1G 0 trials
- Dilated cardiomyopathy 1GG 0 trials
- Dilated cardiomyopathy 1H 0 trials
- Dilated cardiomyopathy 1I 0 trials
- Dilated cardiomyopathy 1II 0 trials
- Dilated cardiomyopathy 1JJ 0 trials
- Dilated cardiomyopathy 1K 0 trials
- Dilated cardiomyopathy 1KK 0 trials
- Dilated cardiomyopathy 1L 0 trials
- Dilated cardiomyopathy 1M 0 trials
- Dilated cardiomyopathy 1NN 0 trials
- Dilated cardiomyopathy 1O 0 trials
- Dilated cardiomyopathy 1Q 0 trials
- Dilated cardiomyopathy 1R 0 trials
- Dilated cardiomyopathy 1S 0 trials
- Dilated cardiomyopathy 1U 0 trials
- Dilated cardiomyopathy 1V 0 trials
- Dilated cardiomyopathy 1W 0 trials
- Dilated cardiomyopathy 1X 0 trials
- Dilated cardiomyopathy 1Y 0 trials
- Dilated cardiomyopathy 1Z 0 trials
- Dilated cardiomyopathy 2A 0 trials
- Dilated cardiomyopathy 2B 0 trials
- Left ventricular noncompaction 10 0 trials
- Left ventricular noncompaction 8 0 trials
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Barth syndrome 5 trials
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Kearns-Sayre syndrome 5 trials
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Histiocytoid cardiomyopathy 3 trials
1 sub-type
- Cardiac lipidosis, familial 0 trials
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Emery-Dreifuss muscular dystrophy 1 trial · 3 incl. sub-types
4 sub-types
- Autosomal dominant Emery-Dreifuss muscular dystrophy 0 trials · 3 incl. sub-types Sub-types →
- Emery-Dreifuss muscular dystrophy 3, autosomal recessive 0 trials
- X-linked Emery-Dreifuss muscular dystrophy 0 trials Sub-types →
- Scapuloperoneal myopathy 0 trials Sub-types →
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Cardiomyopathy, dilated, 100 0 trials
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Cardiomyopathy, dilated, 1LL 0 trials
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Cardiomyopathy, dilated, 1MM 0 trials
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Cardiomyopathy, dilated, 1QQ 0 trials
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Cardiomyopathy, dilated, 2I 0 trials
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Cardiomyopathy, dilated, 2K 0 trials
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Cardiomyopathy, dilated, 2M 0 trials
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Cardiomyopathy, dilated, 2j 0 trials
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Cardiomyopathy, dilated, 2l 0 trials
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Cardiomyopathy, dilated, 3C 0 trials
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Dilated cardiomyopathy 1J 0 trials
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Hypertrophic cardiomyopathy 25 0 trials
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Myofibrillar myopathy 1 0 trials
Most studied deeper sub-types
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Could a short course of steroids calm a dangerous heart rhythm?
Disease control By invitation onlyThis trial tests whether an 8-week course of the immunosuppressive drug prednisone can reduce episodes of ventricular tachycardia (a potentially fatal fast heart rhythm) and improve heart function in people with non-ischemic cardiomyopathy and confirmed heart inflammation. Partic…
Phase 4 • Sponsor: Roderick Tung • Aim: Disease control
Last updated Jul 29, 2026 00:00 UTC
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One-Time gene therapy aims to halt rare muscle disease
Disease control OngoingThis study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the…
Phase 3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Desperate hope: gene therapy tested in one patient with rare blindness
Disease control Expanded access (ended)This expanded access program gave a single patient with Leber Hereditary Optic Neuropathy (a genetic cause of vision loss) an experimental gene therapy called GS010. The treatment was injected into both eyes to test safety. Only one person was involved, so the results are very li…
Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Experimental gene therapy targets rare muscle disease in first human test
Disease control OngoingThis early-phase trial tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can produce the missing beta-sarcoglycan protein …
Phase 1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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Gene therapy aims to restore sight in rare blindness condition
Disease control OngoingThis phase 3 trial tests a gene therapy called NR082 for Leber's hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. About 95 people aged 12 to 75 with a specific ND4 mutation will receive a single injection of the therapy or a sham procedure. T…
Phase 3 • Sponsor: Wuhan Neurophth Biotechnology Limited Company • Aim: Disease control
Last updated Jun 27, 2026 09:05 UTC
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Gene therapy hope for kids with rare muscle-wasting disease
Disease control OngoingThis early-stage trial tests a single intravenous dose of a gene therapy (ATA-200) in 4 children aged 6-12 with limb-girdle muscular dystrophy type 2C/R5 (LGMD2C), a rare genetic muscle-weakening condition. The goal is to see if the treatment is safe and tolerable by delivering a…
Phase 1 • Sponsor: Atamyo Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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Super MRI could spot hidden heart damage
Diagnosis OngoingThis study tests whether a new, more powerful 7 Tesla MRI can better detect scarring and swelling in the heart muscle of people with cardiomyopathy. Researchers will scan 13 adults aged 20-70 to see if the higher-resolution images improve diagnosis. The goal is to see if this adv…
Sponsor: University of Pennsylvania • Aim: Diagnosis
Last updated Jun 27, 2026 09:02 UTC
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Researchers hunt for muscle clues in rare dystrophy
Knowledge-focused OngoingThis pilot study looks at biomarkers in the blood of people with fragile sarcolemmal muscular dystrophy, a condition that makes muscle membranes weak. Researchers will collect blood samples at rest and after exercise during four 5-day hospital stays. The goal is to better underst…
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC
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Can a gene registry unlock the secrets of childhood heart failure?
Knowledge-focused OngoingThis study gathers health information from children under 18 who have cardiomyopathy linked to mutations in the MYBPC3 gene. Researchers aim to map the disease's natural course, identify risk factors, and measure how it affects quality of life. By reviewing past and future medica…
Sponsor: Tenaya Therapeutics • Aim: Knowledge-focused
Last updated Sep 04, 2026 00:00 UTC
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New study tracks rare genetic heart condition to guide future treatments
Knowledge-focused OngoingThis study follows about 35 people with dilated cardiomyopathy caused by a BAG3 gene mutation, a condition that weakens the heart muscle. Over three years, researchers will monitor heart function, symptoms, and quality of life using tests like imaging and blood work. The goal is …
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:07 UTC
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Massive genetic study aims to unlock secrets of rare metabolic diseases
Knowledge-focused By invitation onlyThis study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …
Sponsor: Region Stockholm • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:38 UTC
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New digital tool aims to tame uncontrolled asthma in GP practices
Knowledge-focused OngoingThis study tests a digital tool called AsthmaOptimiser that helps doctors assess asthma control and find ways to improve treatment during a single visit. About 746 adults with asthma who have had recent flare-ups will take part. The goal is to see if the tool can identify better …
Sponsor: General Practitioners Research Institute • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:02 UTC
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Massive gene hunt launched for mysterious mitochondrial diseases
Knowledge-focused By invitation onlyThis study aims to discover new genetic mutations that cause mitochondrial disorders by analyzing tissue samples from up to 6,900 participants. It includes people with suspected or known mitochondrial diseases, such as MELAS or Leigh's Disease, who lack a genetic diagnosis. The r…
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC
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Scientists watch LGMD progress in 205 patients over years
Knowledge-focused OngoingThis study follows 205 people with four types of limb-girdle muscular dystrophy (LGMD) to understand how the disease changes over time. Participants will have their muscle strength, movement, and breathing tested regularly for up to 5 years. No treatment is given; the goal is to …
Sponsor: Sarepta Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC
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New study aims to map rare muscle disease progression
Knowledge-focused CancelledThis study was designed to track the natural course of gamma-sarcoglycanopathy (LGMDR5), a rare muscle-weakening disease, over two years. Researchers planned to measure changes in muscle strength, walking ability, and daily function in patients aged 6 to 35. The goal was to bette…
Sponsor: Atamyo Therapeutics • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:54 UTC