Actively Recruiting
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)
Led by Emory University · Updated on 2025-09-10
36
Participants Needed
5
Research Sites
102 weeks
Total Duration
On this page
Sponsors
E
Emory University
Lead Sponsor
T
The Marcus Foundation
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a Phase 1 study to determine the safety and efficacy of allogeneic neonatal mesenchymal stromal cells (nMSCs) for the treatment of Dilated Cardiomyopathy. The purpose of the study is to help doctors and scientists learn if allogeneic neonatal mesenchymal stromal cells (nMSCs) infusions are a safe and effective way to improve cardiac function and left ventricular ejection fraction.
CONDITIONS
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to less than 30 years for Phase 1A or age 4 to less than 18 years for Phase 1B
- Ability to sign own consent for Phase 1A
- Diagnosis of dilated cardiomyopathy including congenital cardiac malformations with systolic dysfunction, idiopathic, familial/genetic, myocarditis, acquired, ischemic, left ventricular noncompaction, or coronary artery disease
- Left ventricular ejection fraction ≤ 45% documented by echocardiogram or cardiac MRI within 6 months
- Left ventricular dilation with echocardiography Z score > +2.0
- Biventricular physiology with systemic left ventricle
- Receiving guideline directed heart failure treatments
- Unresponsive or poorly responsive to at least 3 months of maximum guideline directed treatments
You will not qualify if you...
- Listed for heart transplantation as UNOS status 1A or hospitalized waiting for transplant on inotropes or ventricular assist device
- Cardiovascular surgery or intervention within 3 months before screening or expected within 12 months after enrollment
- Previous heart transplant recipient
- Severe valve disease or significant systemic ventricular outflow obstruction
- Restrictive or hypertrophic cardiomyopathy
- Cardiogenic shock
- Currently on extracorporeal membrane oxygenation support
- Ventricular assist device support
- Lethal arrhythmia causing hemodynamic instability needing defibrillation, continuous IV anti-arrhythmic medication, or mechanical support
- Persistent atrial fibrillation requiring specific medication
- Amyloidosis
- Ischemic dilated cardiomyopathy
- Malignant neoplasm within 5 years (except certain treated skin or cervical cancers)
- Serious neurologic disorders including vision loss, stroke, or paralysis
- High-grade pulmonary embolism needing catheter intervention or pulmonary hypertension requiring vasodilators
- High-grade renal failure (eGFR <45) or serum potassium >5.3 mmol/L
- Multiple organ failure
- Non-cardiac condition limiting life expectancy to less than 1 year
- Uncontrolled diabetes (HbA1c >9%) at screening
- Active infection requiring treatment or sepsis
- Active bleeding disorders
- History of cardiac transplantation
- Use of immune-altering or immunosuppressive medications within prior 12 weeks
- Dystrophin-associated cardiomyopathy
- Confirmed myocarditis at screening
- Elevated liver function tests or white blood cell counts beyond normal limits at consent
- Presence of HLA antibodies specific for study product
- History of noncompliance, alcohol abuse, drug use, or incarceration within last year
- Currently pregnant or breastfeeding
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
Hughes Spalding Children's Hospital
Atlanta, Georgia, United States, 30303
Actively Recruiting
2
Egleston Children's Hospital
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Emory Children's Center
Atlanta, Georgia, United States, 30322
Actively Recruiting
4
Emory University Hospital
Atlanta, Georgia, United States, 30322
Actively Recruiting
5
Scottish Rite Children's Hospital
Atlanta, Georgia, United States, 30342
Actively Recruiting
Research Team
W
William Mahle, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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