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.
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
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.
Single group
Allogeneic Neonatal mesenchymal stromal cells (nMSCs)
Biological/VaccinenMSCs 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.
Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater
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 cardiomyopathy
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.
Change of left ventricular ejection fraction (LVEF) from baseline
Change of left ventricular ejection fraction (LVEF) between baseline, 3-month, 6-month, and 12-month follow-up determined by echocardiography and cardiac MRI.
Change in N-terminal pro b-type natriuretic peptide (NT-proBNP) levels from baseline
N-terminal pro b-type natriuretic peptide (NT-proBNP) levels will be collected on infusion days, and all post infusion follow up visits.
Change of 6-minute walk test (6MWT) results
A 6-minute walk test (6MWT) will be completed at baseline, 3-month, 6-month, and 1-year visits to measure functional status if the participant is developmentally appropriate. Distance in meters will be measured until the participant can either walk 6 minutes, or they become too exhausted.
Change in quality-of-life validated survey scores in Peds Quality of Life (QOL) survey
Change in quality-of-life validated survey scores in Peds Quality of Life (QOL) survey (≥4 years - \< 18 years). Total possible score ranges from 0 to 100 and higher scores indicate better QOL.
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Emory University
Lead sponsor
The Marcus Foundation
Collaborator
