RecruitingPhase 1NCT06464588

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)

Sponsor

Emory University

Enrollment

36 participants

Start Date

Jul 14, 2025

Study Type

INTERVENTIONAL

Conditions

Dilated Cardiomyopathy

Summary

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.

Eligibility

Min Age: 4 YearsMax Age: 30 Years

Inclusion Criteria10

Phase 1A: Age greater than or equal to 18 years and less than 30 years (≥18 years, \<30 years).
Phase 1B: Age greater than or equal to 4 years and less than 18 years (≥4 years, \<18 years)
Subjects must be able to sign their own consent for Phase 1A of the study.
Diagnosis of dilated cardiomyopathy (DCM) defined as
Any Congenital Cardiac Malformation with systemic ventricular systolic dysfunction; Idiopathic Cardiomyopathy; Familial/Inherited and/or Genetic Cardiomyopathy; History of Myocarditis; Acquired (Chemotherapy, Iatrogenic, Infection, Rheumatic, Nutritional); Ischemic (e.g. Kawasaki Disease, post-operative); Left ventricular noncompaction; Coronary Artery Disease
Left ventricular ejection fraction less than or equal to 45% documented by two-dimensional echocardiogram or cardiac MRI within the prior six months.
Left ventricular dilation as defined by echocardiography left ventricular and end-diastolic dimension Z score \> +2.0
Biventricular physiology with systemic left ventricle
Must receive guideline directed heart failure as defined by the American Heart Association, American College of Cardiology, and Heart Failure Society of America 118
Have been unresponsive or poorly responsive to at least 3 months of maximum guideline directed treatments.

Exclusion Criteria32

Listed for heart transplantation (as UNOS status 1A) or hospitalized while waiting for transplant (while on inotropes or with ventricular assist device)
Cardiovascular surgery of percutaneous intervention to palliate or correct congenital cardiovascular malformations within 3 months of the screening visit. Patients anticipated to undergo corrective heart surgery during the 12 months after entry into Part 1A/1B.
Previous heart transplant recipient
Unoperated primary obstructive or severe regurgitant valve (aortic, pulmonary, or tricuspid) disease, or significant systemic ventricular outflow obstruction or aortic arch obstruction.
Severe mitral valve disease
Restrictive or hypertrophic cardiomyopathy
Cardiogenic shock
Currently on extracorporeal membrane oxygenation support
Ventricular assist device support
Lethal, uncontrollable arrhythmia defined as an arrhythmia resulting in hemodynamic instability requiring need for defibrillation, continuous intravenous anti-arrhythmic medication or mechanical circulatory support
Patients with persistent atrial fibrillation requiring specific pharmacotherapy
Amyloidosis
Ischemic dilated cardiomyopathy
Clinical history of malignant neoplasm within 5 years (with the exception of curatively treated basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma)
Serious neurologic disorder including loss of vision, stroke, or paralysis
High-grade pulmonary embolism requiring interventional catheter procedure or pulmonary hypertension requiring use of pulmonary vasodilators including phosphodiesterase inhibitor or nitric oxide
High-grade renal failure \[eGFR\<45\] mL/min/1.73 m2 - serum potassium \>5.3 mmol/L
Multiple organ failure
Non-cardiac condition that limits life span for \<1 year
Uncontrolled diabetes (HbA1c \>9%) at screening
Active infection (including endocarditis) requiring pharmacotherapy
Sepsis
Active hemorrhagic disease (e.g., gastrointestinal bleeding, injury)
History of cardiac transplantation
Immune system-altering medications, or immunosuppressive therapy at the time of enrolment or within the prior 12 weeks
Dystrophin-associated cardiomyopathy confirmed by standard cardiomyopathy panel testing
Confirmed myocarditis at time of screening
Elevated LFTs greater than 2 times upper limit of normal at time of consent
Elevated WBC greater than upper limit of normal as defined by local lab at time of consent
Presence of HLA antibodies specific for therapeutic study product
History of noncompliance, alcohol abuse, recreational drug use, or incarceration within the last year
Currently pregnant or breastfeeding

Print for Your Doctor

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

BIOLOGICALAllogeneic Neonatal mesenchymal stromal cells (nMSCs)

nMSCs will be administered intravenously in the predefined dose per each group. The rate of infusion will be approximately 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels.