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)
Emory University
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.
Description
This study is a single site open label study with 2 phases. The 2 phases will be broken into an adult group Phase 1A with group pediatric group Phase 1B. This study will enroll patients between the ages of 4 years and 30 years old. The investigators will be looking at the safety, feasibility, and maximum tolerated dose of allogeneic neonatal mesenchymal stromal cells (nMSCs) as defined by freedom from CTCAE or Grade 3 AE that is probably or definitely related to the IP throughout the duration of the study. A minimum of 9 and a maximum of 18 patients will be enrolled into both Phase 1A adult g…
Eligibility
- Age range
- 4–30 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria * Phase 1A: Age greater than or equal to 18 years and less than 30 years (≥18 years, \<30 years). * Phase 1B: Age greater than or equal to 4 years and less than 18 years (≥4 years, \<18 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, Nutriti…
Interventions
- BiologicalAllogeneic 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.
Locations (5)
- Hughes Spalding Children's HospitalAtlanta, Georgia
- Egleston Children's HospitalAtlanta, Georgia
- Emory Children's CenterAtlanta, Georgia
- Emory University HospitalAtlanta, Georgia
- Scottish Rite Children's HospitalAtlanta, Georgia