Phase 1 Study of Safety and Dose of Intravenous Neonatal Mesenchymal Cells nMSCs in Children and Young Adults With Dilated Cardiomyopathy

What this Trial Is About

Researchers are conducting a Phase 1 study to evaluate the safety and effectiveness of allogeneic neonatal mesenchymal stromal cells nMSCs for treating Dilated Cardiomyopathy DCM. The study aims to determine if intravenous infusions of nMSCs can improve heart function and left ventricular ejection fraction in young adult and pediatric patients aged 4 to 40 years. The study is open-label and involves both adult and pediatric groups to assess safety, feasibility, and the maximum tolerated dose of the cell therapy. Participants will receive intravenous infusions of nMSCs every 30 days for a total of three infusions. Adults 16 to under 40 years will be enrolled in Phase 1A, with escalating doses given on days 0, 15, and 30. Pediatric patients 4 to under 16 years will be enrolled in Phase 1B after adult phase completion and regulatory review, with doses adjusted by body weight and similar infusion schedules. Dose escalation follows a 33 study design. After infusions, follow-up visits will occur at 3 months, 6 months, and 1 year. Participants will undergo assessments including blood tests, echocardiograms, electrocardiograms, physical exams, a 6-minute walk test, cardiac MRI, and quality-of-life questionnaires at various timepoints before and after treatment. Researchers will monitor for adverse events, heart function changes, and quality-of-life improvements over a total participation duration of about 14 months. Financial compensation and parking reimbursement are provided, and patients are not required to stay overnight after infusions.

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)