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)
NCT Number: NCT06464588
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1
Conditions: Dilated Cardiomyopathy
Interventions: BIOLOGICAL: Allogeneic Neonatal mesenchymal stromal cells (nMSCs)
Sponsor: Emory University
Collaborators: The Marcus Foundation
Sex: ALL
Age: CHILD, ADULT
Enrollment: 36
Study Design: Allocation: NON_RANDOMIZED|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-10
Completion Date: 2027-07
Locations: Hughes Spalding Children's Hospital, Atlanta, Georgia, 30303, United States|Egleston Children's Hospital, Atlanta, Georgia, 30322, United States|Emory Children's Center, Atlanta, Georgia, 30322, United States|Emory University Hospital, Atlanta, Georgia, 30322, United States|Scottish Rite Children's Hospital, Atlanta, Georgia, 30342, United States

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.

Primary Outcome Measures: 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., End of study, around 12 months post-intervention|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., End of study, around 12 months post-intervention
Secondary Outcome Measures: 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., Baseline, 3-month, 6-month, and 12-month post-intervention|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., Baseline, 3-month, 6-month, and 12-month post-intervention|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., Baseline, 3-month, 6-month, and 12-months post-intervention|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., Baseline, 3-month, 6-month and 12-month post-intervention|Change in Kansas City Cardiomyopathy Questionnaire, Change in Kansas City Cardiomyopathy Questionnaire will be recorded (≥18 years - ≤30 years). KCCQ scores are scaled from 0 to 100 and summarized in 25-point ranges, where scores represent health status as follows: 0 to 24: very poor to poor; 25 to 49: poor to fair; 50 to 74: fair to good; and 75 to 100: good to excellent., Baseline, 3-month, 6-month and 12-month post-intervention