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.
- Subjects with hypoplastic left heart syndrome (all types) requiring BDCPA surgery.
- Have HLHS and restrictive or intact atrial septum.
- Be undergoing the Norwood procedure that do not have HLHS.
- Have significant coronary artery sinusoids.
- Require mechanical circulatory support prior to surgery.
- Have an underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
- Parent or guardian unwilling or unable to comply with necessary follow-up(s)
- Be serum positive for HIV, hepatitis BsAg or viremic hepatitis C.
- Be unsuitable for inclusion in the study, in the opinion of the investigator.
- Need for concomitant surgery for aortic coarctation or tricuspid valve repair.