Dilated cardiomyopathy

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22Active trials47Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Dilated cardiomyopathy (DCM), also known as congestive cardiomyopathy, is a heart muscle disease characterized by enlargement (dilation) and impaired contraction of the left ventricle or both ventricles. The heart chambers stretch and thin, reducing the heart's ability to pump blood efficiently throughout the body. This progressive condition primarily affects the cardiovascular system and can lead to heart failure, arrhythmias (abnormal heart rhythms), thromboembolism (blood clots), and sudden cardiac death. DCM can be caused by genetic mutations (familial DCM, accounting for 20–50% of cases), or it may be acquired through viral infections, alcohol use, toxins, autoimmune conditions, or other systemic diseases. In many cases, no identifiable cause is found (idiopathic DCM). Key symptoms include shortness of breath (dyspnea), particularly during exertion or when lying flat, fatigue, exercise intolerance, peripheral edema (swelling of the legs and ankles), palpitations, and in advanced cases, signs of congestive heart failure such as fluid retention and reduced exercise capacity. Some patients may be asymptomatic in early stages, with the condition discovered incidentally through imaging studies. Arrhythmias, including atrial fibrillation and ventricular tachycardia, are common complications that can significantly impact prognosis. Treatment of dilated cardiomyopathy focuses on managing heart failure symptoms and preventing complications. Standard medical therapy includes ACE inhibitors or angiotensin receptor blockers, beta-blockers, mineralocorticoid receptor antagonists, and diuretics. Newer agents such as sacubitril/valsartan (ARNI) and SGLT2 inhibitors have also become part of guideline-directed medical therapy. Anticoagulation may be prescribed to reduce thromboembolic risk. Implantable cardioverter-defibrillators (ICDs) are recommended for patients at high risk of sudden cardiac death, and cardiac resynchronization therapy (CRT) may benefit those with conduction abnormalities. In end-stage disease, left ventricular assist devices (LVADs) or heart transplantation may be considered. Familial cases warrant genetic counseling and screening of first-degree relatives.

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Jun 2026A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

Rocket Pharmaceuticals Inc. — PHASE1

TrialNOT YET RECRUITING
May 2026Comparative Efficacy of ARNI Versus ACE Inhibitor Therapy in Egyptian Children With Dilated Cardiomyopathy

Assiut University

TrialNOT YET RECRUITING
May 2026BAG3-DCM Natural History Study

Rocket Pharmaceuticals Inc.

TrialNOT YET RECRUITING
Apr 2026RHYthm and Myocardial Function Relationship Evaluation in Heart Diseases

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

TrialNOT YET RECRUITING
Mar 2026An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)

Affinia Therapeutics — PHASE1, PHASE2

TrialNOT YET RECRUITING
Feb 2026Mainstreaming Genetic Testing for Non-Ischemic Cardiomyopathy in Western Canada

Thomas Roston — NA

TrialNOT YET RECRUITING
Jan 2026Prognostic Values of Coronary Microvascular Dysfunction in Patients With Dilated Cardiomyopathy

Second Affiliated Hospital, School of Medicine, Zhejiang University

TrialRECRUITING
Dec 2025A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy

AstraZeneca — PHASE1

TrialRECRUITING
Oct 2025Multimodal Analysis of Endomyocardial Biopsies

University Hospital, Essen — NA

TrialRECRUITING
Oct 2025Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age

Sebastiano Lava — PHASE2

TrialNOT YET RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Dilated cardiomyopathy.

20 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

20 recruitingView all trials with filters →
Phase 31 trial
Bromocriptine in Dilated Cardiomyopathy Among Women of Reproductive Age
Phase 3
Actively Recruiting
PI: Kedir N Tukeni, MD (Jimma University) · Sites: Jimma, Oromiya · Age: 1850 yrs
Phase 41 trial
The Effects of Sirolimus in Patients With Dilated Cardiomyopathy Infected With Kaposi Sarcoma-associated Virus
Phase 4
Actively Recruiting
PI: Dao Wen Wang, Doctor (Tongji Hospital) · Sites: Wuhan, Hubei · Age: 1870 yrs
Phase 22 trials
Outcome-targeted Therapy: Principle and Outcome Evaluation: Clinical Study and Phenotype-genotype Correlation
Phase 2
Actively Recruiting
PI: Wei-Chieh Tseng, MD (Principal Investigator) · Sites: Taipei County, Zhongzheng District · Age: 099 yrs
Use of Bone Marrow Derived Stem Cell and G-CSF With Circulatory Assistance in the Treatment of DCM
Phase 2
Actively Recruiting
PI: Anthony Mathur (Queen Mary University of London) · Sites: London · Age: 1885 yrs
Phase 14 trials
Safety and Efficacy of FAP iCDC in End-stage Dilated Cardiomyopathy
Phase 1
Active
PI: Xinyang Hu, PhD (2nd Affiliated Hospital, School of Medicine, Zheji) · Sites: Hangzhou, Zhejiang · Age: 1875 yrs
A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy
Phase 1
Actively Recruiting
· Sites: Amsterdam; Groningen +2 more · Age: 1880 yrs
Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Phase 1
Actively Recruiting
· Sites: Kansas City, Kansas; Cincinnati, Ohio +1 more · Age: 1899 yrs
A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells (nMSCs) in Young Adult (1A) and Pediatric (1B) Patients With Dilated Cardiomyopathy (DCM)
Phase 1
Actively Recruiting
PI: William Mahle, MD (Emory University) · Sites: Atlanta, Georgia; Atlanta, Georgia +3 more · Age: 430 yrs
N/A5 trials
Multimodal Analysis of Endomyocardial Biopsies
N/A
Actively Recruiting
PI: Lars Michel, PD Dr. med. (Department of Cardiology and Vascular Medicine, We) · Sites: Essen, North Rhine-Westphalia · Age: 1899 yrs
The Application of T1 Mapping in Real-World
N/A
Actively Recruiting
PI: Huaying Zhang, Doctor · Sites: Beijing, Beijing Municipality
Dilated Cardiomyopathy Detection Using AI and Screening With Mobile Technology (DCM-DETECT)
N/A
Actively Recruiting
PI: Roy Small, MD (Penn Medicine / Lancaster General Hospital) · Sites: Lancaster, Pennsylvania · Age: 1899 yrs
Multimodal and Multidisciplinary Approach to Optimize Diagnostic, Prognostic, and Therapeutic Management of Patients with Non-ischemic Cardiomyopathies and Arrhythmogenic-inflammatory Phenotypes: a Multicenter, Observational, Retrospective and Prospective Registry Study.
N/A
Actively Recruiting
PI: Paolo Della Bella, MD (San Raffaele Scientific Institute, Milan, Italy) · Sites: Milan, Milano · Age: 1899 yrs
Clinical Evaluation of the AccuCinch® Ventricular Restoration System in Patients Who Present With Symptomatic Heart Failure With Reduced Ejection Fraction (HFrEF): The CORCINCH-HF Study
N/A
Actively Recruiting
PI: Martin B Leon, MD (Columbia University) · Sites: Birmingham, Alabama; Birmingham, Alabama +130 more · Age: 1899 yrs
Other7 trials
Dilated Cardiomyopathy-Cardiac Magnetic Resonance (DCM-CMR) Ancillary Study
Actively Recruiting
PI: Ray Hershberger, MD (Ohio State University) · Sites: Columbus, Ohio · Age: 1899 yrs
Cardiac Magnetic Resonance for Risk Stratification in Dilated Cardiomyopathy
Actively Recruiting
PI: Minjie Lu, PhD (Chinese Academy of Medical Sciences, Fuwai Hospita) · Sites: Beijing, Beijing Municipality
Image-Based Prediction of Ventricular Tachycardia Events in Non-ischemic Cardiomyopathy
Actively Recruiting
· Sites: Milan, Italy; Lispon, Portugal +2 more · Age: 1899 yrs
Pediatric Cardiomyopathy Mutation Analysis
Actively Recruiting
PI: Stephanie Ware, MD, PhD (IU School of Medicine) · Sites: Indianapolis, Indiana
Defining the Genetics, Biomarkers and Outcomes for Dilated Cardiomyopathy
Active
PI: James Ware (Imperial College London) · Sites: Leeds; Leicester +4 more · Age: 099 yrs
Evaluating the Prognostic Impact of Anti-β1AR Antibodies and Anti-L-CaC Antibodies in Patients With Dilated Cardiomyopathy
Actively Recruiting
· Sites: Wuhan, Hubei · Age: 1875 yrs
Prognostic Values of Coronary Microvascular Dysfunction in Patients With Dilated Cardiomyopathy
Actively Recruiting
· Sites: Hangzhou, Zhejiang · Age: 1899 yrs

Specialists

Showing 25 of 47View all specialists →
LM
Lars Michel, PD Dr. med.
Essen, North Rhine-Westphalia
Specialist

Rare Disease Specialist

PI on 1 active trial
SM
Stephan B Felix, MD
BATON ROUGE, LA
Specialist
PI on 1 active trial
AF
Anthony Mathur, MD FRCP FESC
Specialist
PI on 1 active trial
AM
Andrea Natale, MD
AUSTIN, TX
Specialist
PI on 6 active trials1 Dilated cardiomyopathy publication
AP
Agata Bielecka-Dabrowa, PhD
Specialist
PI on 1 active trial
JM
Joshua Hare, MD
MIAMI, FL
Specialist
PI on 1 active trial
GG
Geir Gokstad
Specialist
PI on 1 active trial
SP
Sandeep Gulati, PhD
BRONX, NY
Specialist
PI on 2 active trials
SM
Sachin Parikh, MD
Specialist
PI on 1 active trial
RP
Roberto Scarsini, MD PhD
Specialist
PI on 1 active trial
MM
Maurizio Volterani, MD
Specialist
PI on 1 active trial
RP
Renato AK Kalil, MD, PhD
Specialist
PI on 1 active trial
TM
Timothy Henry, MD
Specialist
PI on 1 active trial
FM
Frank Mazzola, MD
VALLEY VILLAGE, CA
Specialist
PI on 1 active trial
DM
Daniel Bensimhon, MD
GREENSBORO, NC
Specialist
PI on 1 active trial
SM
Sameer Jamal, MD
HACKENSACK, NJ
Specialist
PI on 1 active trial
JM
Johanna P Contreras, MD
Specialist
PI on 1 active trial
BM
Brian Kolski, MD
ORANGE, CA
Specialist
PI on 1 active trial
JP
Javier Bermejo Thomas, MD, PhD
Specialist
PI on 2 active trials
MP
Maciej Banach, Assoc. Prof.
Specialist
PI on 1 active trial
PP
Ping-Ting Lin, Ph.D.
Specialist
PI on 3 active trials
JM
Jim O 'Neill, MD
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Dilated cardiomyopathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Dilated cardiomyopathy

1 articles
ResearchBIORXIVApr 25, 2026
Preprint: Fasting reverses PLN R14del-mediated cardiomyopathy through lysosomal reactivation
Scientists discovered that fasting may help reverse heart damage caused by a specific genetic mutation in the PLN gene called R14del. This mutation causes a com
See all news about Dilated cardiomyopathy

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Dilated cardiomyopathy

What is Dilated cardiomyopathy?

Dilated cardiomyopathy (DCM), also known as congestive cardiomyopathy, is a heart muscle disease characterized by enlargement (dilation) and impaired contraction of the left ventricle or both ventricles. The heart chambers stretch and thin, reducing the heart's ability to pump blood efficiently throughout the body. This progressive condition primarily affects the cardiovascular system and can lead to heart failure, arrhythmias (abnormal heart rhythms), thromboembolism (blood clots), and sudden cardiac death. DCM can be caused by genetic mutations (familial DCM, accounting for 20–50% of cases),

Are there clinical trials for Dilated cardiomyopathy?

Yes — 20 recruiting clinical trials are currently listed for Dilated cardiomyopathy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Dilated cardiomyopathy?

25 specialists and care centers treating Dilated cardiomyopathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.