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 Study of nMSCs for Dilated Cardiomyopathy", "This Phase 1 study is testing the safety of a new treatment called allogeneic neonatal mesenchymal stromal cells (nMSCs) for people with Dilated Cardiomyopathy (DCM), a condition where the heart muscle becomes stretched and thin. Researchers want to see if giving nMSCs intravenously (into a vein) is safe and helps improve heart function. You may be able to join if you are between 4 and 40 years old and have DCM. The study will look for serious side effects and the highest safe dose of nMSCs over about 12 months. This study is currently recruiting a total of 36 participants.", "design": "This is a single-site, open-label study with two groups: young adults (Phase 1A) and children (Phase 1B). It aims to enroll 36 participants.", "commitments": "You will receive nMSC infusions intravenously three times, 30 days apart. There will be a baseline visit, a phone call after the last infusion, and in-person visits at 3, 6, and 12 months, for a total of 14 months.", "compensation": "Not stated in the trial record.", "follow_up": "Participants will be followed for about 12 months after the intervention to assess safety and maximum tolerated dose.", }

Study design: Not specified.
What's involved: Not specified in the trial record.
Compensation: Not stated in the trial record.
Follow-up: Not specified.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

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)

Recruiting

PHASE1Ages 4–40InterventionalTreatment

Emory University

~36 participants

Updated 2026-06-26 on ClinicalTrials.gov

What's tested:Allogeneic Neonatal mesenchymal stromal cells (nMSCs)

At a glance

Recruiting sites

5 of 5 listed sites are recruiting right now

RecruitingSuspended, closed, or not yet open

What they're measuring

Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater

Measured over End of study, around 12 months post-intervention

+1 more outcome measured

Conditions

Dilated Cardiomyopathy

Where it's being run

5 sites across 1 states

Georgia5

Study leadership

William Mahle, MD · PRINCIPAL_INVESTIGATOR · Emory University

Who to contact

William Mahle, MD

[email protected]

404-256-2593

Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Phase 1A: Age greater than or equal to 16 years and less than 40 years (≥16 years, \<40 years).

Phase 1B: Age greater than or equal to 4 years and less than 16 years (≥4 years, \<16 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

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, mitral or tricuspid) disease, or significant systemic ventricular outflow obstruction or aortic arch obstruction anticipated to require surgical or transcatheter intervention within 6 months.

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

Unsafe/unfeasible to enroll due to PI/designee discretion

What this trial measures

Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greaterEnd of study, around 12 months post-intervention
Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater AE that is probably or related to the IP throughout the duration of the study will be recorded. Results can vary from 0 to 100% and higher proportion correlates with better outcome. TCAE Grade 3 is defined as severe or medically significant not immediately life-threatening; hospitalization or prolongation of hospitalization. Grade 4 and 5 AEs include composite of: death, life-threatening events, initial or prolonged hospitalization, disability of permanent damage and congenital anomaly/birth defects.
Maximum tolerated dose (MTD) in patients with dilated cardiomyopathyEnd of study, around 12 months post-intervention
If two patients in a dosing group have a related SAE or Dose Limiting Toxicity (DLT), then the previous dosing group will be defined as MTD.

Trialytics