Table 1. Clinical trials of stem cell therapy for single ventricle CHD.
| Trial | Year | Design | Sponsor and Collaborators | Cell Type | Route | Timing | Status |
|---|---|---|---|---|---|---|---|
| Transcoronary Infusion of Cardiac Progenitor Cells in Patients with Single Ventricle Physiology (TICAP) | 2011 | Non-randomized, controlled phase I | Okayama University, National Cerebral and Cardiovasc. Ctr. (Japan) | CPC (autologous) | IC | Stage II or III operation | Reported |
| Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease (PERSEUS) | 2013 | Randomized, controlled phase II | Okayama University, Translational Research Informatics Ctr., Kobe | CPC (autologous) | IC | Stage II or III operation | Reported |
| Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome | 2013 | Single treatment group phase I | Mayo Clinic, University of Oklahoma | UCB-derived MN cells (autologous) | IM | Stage II operation | Ongoing |
| Allogeneic hMSC Injection in Patients with Hypoplastic Left Heart Syndrome (ELPIS) | 2015 | Randomized, controlled phase I/II | University of Miami, University of Maryland | MSC (allogeneic) | IM | Stage II operation | Ongoing |
| Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous BM-Derived MN Cells | 2015 | Single treatment group phase I | Mayo Clinic | BM-derived MN cells (autologous) | IC | After stage III (with ventricular dysfunction) | Ongoing |
| Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON) | 2016 | Randomized, controlled phase III | Japanese Regenerative Medicine Co., Ltd | JRM-001 (cardiac stem cells)† | IC | Stage II or III operation | Ongoing |
| Mesoblast Stem Cell Therapy for Patients with Single Ventricle and Borderline LV | 2017 | Randomized, controlled phase I/II | Boston Children’s Hospital | MPC (allogeneic) | IM | Stage II or LV recruitment operation | Ongoing |
| Safety of Autologous Cord Blood Cells in HLHS Patients During Norwood Heart Surgery | 2018 | Single treatment group phase I | Royal Children’s Hospital (Melbourne, AU) | UCB-derived MN cells (autologous) | IC | Stage I operation | Ongoing |
| Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome | 2018 | Randomized, controlled phase I | University of Miami, University of Maryland, Emory University | CPC (autologous) | IC | Stage II operation | Not yet enrolling |
All data obtained from ClinicalTrials.gov and verified on 23 February 2018. Stages refer to palliative procedures for single ventricle CHD. †, a proprietary agent, described as such in the NIH listing. Okayama University is a participating site under the direction of Dr. Hidemasa Oh. MSC, mesenchymal stem cell; MPC, mesenchymal precursor cell; CPC, cardiac progenitor cell; UCB, umbilical cord blood; MN, mononuclear; BM, bone marrow; MPC, mesenchymal precursor cell; IC, intracoronary; IM, intramyocardial.