Review Article

Comparing the Therapeutic Potential of Stem Cells and their Secretory Products in Regenerative Medicine

Table 2

Completed clinical trials that evaluated the safety, efficacy, and feasibility of stem cell therapy.

Type of stem cellTreated diseaseTrial designNumber of cellsRoute of administrationOutcomesReferences

Pluripotent stem cellsIschemic heart diseaseAn open label, phase I study to assess both the feasibility and safety of epicardial delivery of a fibrin gel embedded with human embryonic stem cell- (ESC-) derived CD15+ IsI-1+ progenitorsNAEpicardial transplantation of cells embedded in fibrin patchNot yet availableNCT02057900
Advanced Stargardt’s macular dystrophyA follow-up of a phase I/II, open-label, nonrandomized, 4-cohort, dose escalation, multicenter clinical trial to evaluate the long-term safety and tolerability of human ESC-derived retinal pigment epithelium (hESC-RPE) cellular therapy to hESC-RPE/kgSubretinal injectionNot yet availableNCT02941991
Stargardt’s macular dystrophyA phase I/II, open-label, nonrandomized, sequential, multicenter clinical trial to evaluate the safety and tolerability of hESC-RPE cellular therapy in patients with SMD and to evaluate potential efficacy endpoints to be used in future studies of hESC-RPE cellular therapy to hESC-RPE/kgSubretinal injectionNot yet availableNCT01469832
Outer retinal degenerationsA phase I/II, open label, nonrandomized, prospective study to determine the safety of hESC-RPE subretinal injection hESC- RPE/kgSubretinal injectionNot yet availableNCT02903576
Stargardt’s macular dystrophyA phase I/II, open-label, nonrandomized clinical trial to evaluate the safety and tolerability of subretinal injection of hESC-RPE to hESC-RPE/kgSubretinal injectionNot yet availableNCT01345006
Age-related macular degenerationA phase I/II, open-label, nonrandomized, sequential, multicenter clinical trial to evaluate the safety and tolerability of subretinal injection of hESC-RPE to hESC-RPE/kgSubretinal injectionNot yet availableNCT01344993
Age-related macular degenerationA phase I/II trial, open-label, nonrandomized study to evaluate the long-term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry age-related macular degeneration from one to five years following the surgical procedure to implant the MA09-hRPE cells to hESC-RPE/kgSubretinal injectionNot yet availableNCT02463344
Advanced Stargardt’s macular dystrophyA phase I/II trial, open-label, nonrandomized study to evaluate the long-term MA09-hRPE cellular therapy in patients with advanced Stargardt’s macular dystrophy from one to five years following the surgical procedure to implant the MA09-hRPE cells to hESC-RPE/kgSubretinal injectionNot yet availableNCT02445612
Multipotent stem cellsDiabetic peripheral neuropathyA phase II study to investigate the effects of mesenchymal stem cell (MSC) transfusionNAIntravenous infusionNot yet availableNCT02387749
Idiopathic pulmonary fibrosisA phase 1b study to evaluate the safety and feasibility, particularly with respect to adverse acute hemodynamic effects and profibrosis of MSC treatment or hMSCs/kgIntravenous infusionFeasible and satisfactory short-term safety profile.[141]
NCT01385644
Acute respiratory distress syndromeA phase 1 clinical trial to test the safety of a single dose of allogeneic bone marrow-derived MSCs (BMSCs), , or of hMSCs/kgIntravenous infusionSafe and well tolerated[142]
NCT01775774
Periapical periodontitisA randomized, controlled phase I/II clinical trial to evaluate the safety and efficacy of human umbilical cord-derived mesenchymal stem cells (UC-MSCs) encapsulated in a plasma-derived biomaterial for regenerative endodontic procedures in mature permanent teeth with apical lesions cells/biomaterialEncapsulated in platelet poor plasmaSafe and effective[143]
NCT03102879
Cleft lip and palateA phase I trial to evaluate the feasibility and safety of deciduous dental pulp stem cells cells/biomaterialEmbedded in hydroxyapatite-collagen spongeFeasible and safe[144]
NCT01932164
Knee osteoarthritisA multicentre, phase I/II clinical trial to evaluate the clinical use of allogenic BMSCs cells/kgIntra-articular injectionFeasible and safe[145]
NCT01586312
Knee osteoarthritisA phase 2 study to determine the clinical response to autologous bone marrow aspirate concentrate and platelet-rich plasma injectionNAIntra-articular injectionNot yet publishedNCT02958267
FrailtyA phase I/II randomized, double-blind, placebo-controlled clinical trial to evaluate the safety of MSC therapy cells/kgIntravenous infusionSafe[146]
NCT02065245
Retinal degenerationA phase 1 study to report electroretinographic (ERG) findings in advanced glaucoma treated with a single intravitreal injection of BMSCs cells/kgIntravitreal injectionNot yet availableNCT02330978
Spinal cord injuryA phase I/II study to evaluate the safety and efficacy of intrathecal administration of allogeneic UC-MSCs to patients with spinal cord injury cells/kgIntrathecal injectionNot yet availableNCT02481440
Knee osteoarthritisA phase I/II study to evaluate the feasibility and safety of the implantation of 40 million BMSCs in knees with osteoarthritis of grade II-IV (Kellgren and Lawrence) cells/kgIntra-articular injectionNot yet availableNCT01183728
Chronic ischemic cardiomyopathyA phase II trial to assess the feasibility, safety, and efficacy of trans-endocardial administration of autologous MSCs and cardiac progenitor cells (CPCs), alone, and in combination cells/kg b.w.Trans-endocardial injectionFeasible, safe, and effective[147]
NCT02501811
CardiomyopathyA phase I study to examine the safety and feasibility of allogeneic human MSCs by trans-endocardial injection to cancer survivors cells/kgTrans-endocardial injectionSafe and feasible[148]
NCT02509156
Ischemic cardiomyopathyA phase 1/2 randomized comparison study to test whether allogeneic MSCs are as safe and effective as autologous MSCs, , or cells/kgTrans-endocardial injectionSafe and effective[149]
NCT01087996
Nonischemic dilated cardiomyopathyA phase I/II study to assess the safety of autologous and allogeneic human MSC administration cells/kgTrans-endocardial injectionSafe[150]
NCT01392625
Myocardial infarctionA phase II trial to compare the safety and efficacy of 2 doses of allogeneic BMSCs or cells/kgTrans-endocardial injectionSafe[151]
NCT02013674
Ischemic cardiomyopathyA phase I/II study to demonstrate the safety of trans-endocardial injection of autologous MSCs and bone marrow mononuclear cells or cells/kgTrans-endocardial injectionSafe[152]
NCT00768066
End-stage liver diseaseA phase I/II study to investigate the feasibility, safety, and efficacy of autologous MSC injection MSCsPeripheral or portal vein injectionFeasible, safe, and effective[153]
NCT01440309
Knee osteoarthritisA randomized, phase 2b study to assess the efficacy and safety of a single intra-articular injection of adipose tissue-derived MSCs (AT-MSCs) MSCsIntra-articular injectionSafe and effective with satisfactory functional improvement and pain relief in patients[154]
Heart failureA phase 1/2 randomized controlled trial to evaluate the safety and efficacy of the intravenous infusion of UC-MSCs in patients with chronic stable heart failure and reduced ejection fraction MSCs/kgNASafe and effective with improvement in quality of life[155]
NCT01739777
COVID-19A phase 1 clinical trial to evaluate the safety of human UC-MSC infusion in the treatment of patients with moderate and severe COVID-19 pulmonary disease of UC-MSCsNASafe and well tolerated without serious adverse events[140]

NA: not available.