This study is intended to evaluate the safety and feasibility of intramyocardial injection of allogeneic mesenchymal cells during the Bi-Directional Cavopulmonary Anastomosis (BDCPA) surgery for hypoplastic left heart syndrome (HLHS) pediatric patients.
This is an open label study, intended as a safety and efficacy assessment prior to a full comparator study. In this Phase I study, cells administered will be delivered in 6-10 intramyocardial injections that will be tested in 20 patients and 10 patients will be controls with a total of 30 HLHS patients. A total of 30 patients with HLHS will be enrolled in a staged enrollment process. In this open-labeled study, a maximum of 20 patients will eventually receive intramyocardial injection of the allogeneic mesenchymal stem cells and 10 control patients with no cell injection. The enrollment of the patients will occur in two stages groups: Group A and Group B. In Group A, 10 consecutive HLHS patients will be initially enrolled in the allogenic MSCs treatment arm to determine feasibility and safety. After 6 months of the last enrolled patient in Group A, all Group A patients will be assessed in order to determine whether it is feasible and safe, including the harvesting, processing, and administering of the allogeneic MSCs. Thereafter, Group B will start enrolling a total of 20 HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio, respectively, in order to have 10 allogeneic MSCs-treated patient and 10 control patients. At the completion of this Phase I clinical study, the total enrolled cohort will be 20 patients treated with allogeneic MSCs and 10 patients in the control arm.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Allogeneic Human Mesenchymal Stem Cells
Placebo
University of Maryland Medical Center
Baltimore, Maryland, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Monitor major adverse cardiac events
Monitoring of events includes death, sustained/symptomatic ventricular tachycardia requiring intervention with inotropic support, aggravation of heart failure, new myocardial infarction, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.
Time frame: 1 month after injection
Composite Change from baseline in right ventricular function, right ventricular end-diastolic volume, right ventricular end-systolic volume, right ventricular end-systolic diameter and tricuspid regurgitation
as measured by serial echocardiograms and MRI scans.
Time frame: baseline, 24 weeks and 1 year
Number of participants with incidence of mortality or need for transplantation after the BDCPA operation
Assessed through the number of participants with adverse events, hospitalizations, or transplantations.
Time frame: One year after injections
Composite Changes in somatic growth velocity over time (weight, height, head circumference) from the BDCPA operation
Changes in weight, height, and head circumference will be measured
Time frame: 12 months post operative
Assessment of Co-morbidity
Assessment of cardiovascular mortality, all-cause morbidity, Cardiovascular morbidity, re-hospitalizations, need for transplantation.
Time frame: up to 12 months follow-up
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