Recruiting
Phase 1

nMSCs

Sponsor:

Emory University

Code:

NCT06464588

Conditions

Dilated Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 4 - 40

Healthy Volunteers: Not accepted

Interventions

Allogeneic Neonatal mesenchymal stromal cells (nMSCs)

Study Details

Brief 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.

Conditions

Dilated Cardiomyopathy

Study ID

NCT06464588

Start date

Jul 14, 2025

Status verified date

Jun, 2026

Completion date

Jul, 2027

Anticipated

Primary completion date

Jul, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 4 - 40

Healthy Volunteers: Not accepted

Inclusion Criteria

  • 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 Criteria

  • 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

Study Design

Enrollment

36 participants

Anticipated

Allocation

Non randomized

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Adult Cohort

Adults (≥16 years to <40 years) will be enrolled into all dose levels (as tolerated) of Phase 1A. Open label nMSCs will be administered intravenously in the following defined dose groups. The rate of infusion will be approximately 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels:

  • Dose level 1: 5.0x107 nMSCs at 0, 15 and 30 days
  • Dose level 2: 1.0.x108 nMSCs at 0, 15 and 30 days
  • Dose level 3: 2.5x108 nMSCs at 0, 15 and 30 days

Dose escalation will follow 3+3 study design parameters. The treatment between the first and second patients of each dose level will be staggered at least one month after the first patient's first infusion within each dose level. The next dosing group will be initiated at least one month after the last subject in a particular dose level has received the last dose treatment. Once MTD has been determined, 3 additional patients will be enrolled to ensure a total of 6 patients are enrolled in MTD level for confirmation.

experimental: Pediatric Cohort

Pediatric participants (4 to ≤16 years) will be enrolled as determined in Phase 1B(3+3 study design; open label). nMSCs will be administered intravenously (IV) in the following defined dose groups. The rate of infusion will be approx. 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels:

  • Dose Level 1: 0.7x106 nMSCs/kg
  • Dose Level 2: 1.43x106 nMSCs/kg
  • Dose Level 3: 2.85x106 nMSCs/kg

Dose escalation will follow 3+3 study design parameters. Treatment between 1st and 2nd patients of each dose level will be staggered at least 1 month after the 1st patient's first infusion within each dose level. The next dosing group will be initiated one month after the last subject in a particular dose level has received the last dose treatment. Once MTD has been determined, 3 additional patients will be enrolled to ensure a total of 6 patients are enrolled in MTD level for confirmation. Following IV delivery of nMSCs, patients will be followed at 3m, 6m and 1yr.

Interventions

Allogeneic 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.

Primary outcome measure

  • Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater [ Time Frame: End of study, around 12 months post-intervention ]
  • Maximum tolerated dose (MTD) in patients with dilated cardiomyopathy [ Time Frame: End of study, around 12 months post-intervention ]

Central Contacts and Locations

Central contacts

William Mahle, MD

404-256-2593wmahle@emory.edu

Locations

Grady Memorial Hospital

Recruiting

Atlanta, Georgia, United States, 30303

Principal Investigator:

William Mahle, MD

Hughes Spalding Children's Hospital

Recruiting

Atlanta, Georgia, United States, 30303

Principal Investigator:

William Mahle, MD

Emory University Hospital

Recruiting

Atlanta, Georgia, United States, 30322

Principal Investigator:

William Mahle, MD

Arthur M. Blank Hospital

Recruiting

Atlanta, Georgia, United States, 30329

Principal Investigator:

William Mahle, MD

Scottish Rite Children's Hospital

Recruiting

Atlanta, Georgia, United States, 30342

Principal Investigator:

William Mahle, MD

More Information

Sponsor

Emory University

Last update posted

Jun 26, 2026

Last verified

Jun, 2026

Keywords

  • Dilated Cardiomyopathy
  • Neonatal Mesenchymal Cells (nMSCs)

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Emory University on 2026-06-26.