Actively Recruiting
A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells (nMSCs) in Young Adult and Pediatric Patients With Dilated Cardiomyopathy
Led by Emory University · Updated on 2025-09-10
36
Participants Needed
5
Research Sites
N/A
Total Duration
On this page
Sponsors
E
Emory University
Lead Sponsor
T
The Marcus Foundation
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of allogeneic neonatal mesenchymal stromal cells (nMSCs) for treating Dilated Cardiomyopathy in young adult and pediatric patients aged 4 to 30 years. This Phase 1 open-label study aims to determine if intravenous infusions of these cells can improve heart function, specifically left ventricular ejection fraction, while assessing the maximum tolerated dose and monitoring for serious side effects. The study includes two groups: adults aged 18 to 30 years (Phase 1A) and children aged 4 to under 18 years (Phase 1B). Participants receive intravenous nMSC infusions at escalating doses on days 0, 15, and 30. Pediatric doses are based on body weight. Treatments last about 30 to 60 minutes per infusion. Phase 1B starts after adult phase completion and review. After infusions, pediatric patients stay overnight near the hospital for safety monitoring; adults do not require overnight stays. Participants have a baseline visit before treatment, followed by check-ins after each infusion, phone contact 30 days after the last infusion, and in-person visits at 3, 6, and 12 months. Assessments include blood tests, echocardiograms, electrocardiograms, physical exams, a 6-minute walk test, cardiac MRI, and quality-of-life questionnaires. Researchers track heart function changes, patient safety, and quality of life during the total 14-month participation. Left ventricular ejection fraction and adverse events are key outcome measures.
CONDITIONS
Brief 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 between 4 and 30 years (4 to under 18 years for pediatric phase, 18 to under 30 years for adult phase)
- Able to sign consent for adults or assent with parental permission for children
- Diagnosed with dilated cardiomyopathy or related conditions with left ventricular ejection fraction ≤45% confirmed by echocardiogram or cardiac MRI within 6 months
- Left ventricular dilation with end-diastolic dimension Z score > +2.0
- Biventricular physiology with systemic left ventricle
- Receiving guideline-directed heart failure treatment
- 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 with inotropes or ventricular assist device
- Cardiovascular surgery or intervention within 3 months or planned corrective surgery within 12 months
- Previous heart transplant recipient
- Severe valve disease or systemic ventricular outflow obstruction
- Restrictive or hypertrophic cardiomyopathy
- Cardiogenic shock
- On extracorporeal membrane oxygenation or ventricular assist device support
- Uncontrollable arrhythmia requiring defibrillation or continuous intravenous medication
- Persistent atrial fibrillation requiring specific medication
- Amyloidosis or ischemic dilated cardiomyopathy
- Malignant neoplasm within 5 years except certain skin or cervical cancers
- Serious neurologic disorders
- High-grade pulmonary embolism 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 span to under 1 year
- Uncontrolled diabetes (HbA1c >9%)
- Active infection or sepsis requiring treatment
- Active hemorrhagic disease
- History of cardiac transplantation
- Immune system-altering or immunosuppressive medications within 12 weeks
- Dystrophin-associated cardiomyopathy
- Confirmed myocarditis at screening
- Elevated liver function tests or white blood cell count above normal
- Presence of HLA antibodies against 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
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 1 month
Participants receive intravenous infusions of allogeneic neonatal mesenchymal stromal cells (nMSCs) administered approximately at days 0, 15, and 30. The infusion lasts about 30 to 60 minutes per session. Pediatric participants have a check-in visit the morning after each infusion with an overnight stay near the hospital, while adults do not require overnight stays.
3 infusion visits and up to 3 post-infusion visits for pediatric participants
Duration - Approximately 11 months
Participants attend follow-up visits to assess safety and cardiac function at 3 months, 6 months, and 1 year after the last infusion. These visits include physical exams, echocardiograms, electrocardiograms, lab tests, questionnaires, and other assessments.
3 in-person visits at 3, 6, and 12 months
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
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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