Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome

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.

Inclusion Criteria:

- Subjects with hypoplastic left heart syndrome (all types) requiring BDCPA surgery.

Exclusion Criteria:

- 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 (B Surface Antigen) 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.