Table 1.
Characteristics of interventional clinical trials involving pluripotent stem cells.
| ID number | Title | Cell type | Origin | Target disease | Country or area | No. patients enrolled | Intervention | Status (2019) |
|---|---|---|---|---|---|---|---|---|
| NCT03222453 | Thalassemia treatment based on the stem cell technology | hiPSC | Autologous | Beta-thalassemia | China | 2 | Injection of hematopoietic stem cells | Results submitted to ClinicalTrials.gov |
| NCT01674829 | A Phase I/IIa, open-label, single-center, prospective study to determine the safety and tolerability of sub-retinal transplantation of human embryonic stem cell-derived retinal pigmented epithelial(MA09-hRPE) cells in patients with advanced dry age-related macular degeneration | hESC-derived RPE; MA09-hRPE | Allogeneic | Dry age-related macular degeneration | South Korea | 12 | Sub-retinal Transplantation | Active, not recruiting |
| NCT02903576 | Stem cell therapy for outer retinal degenerations | hESC-derived RPE | Allogeneic | Age-related macular degeneration, Stargardt’s disease or exudative age-related macular degeneration | Brazil | 18 | Injection of hESC-RPE in suspension versus injection of hESC-RPE seeded in a substrate | Not yet reported, recruitment status unknown |
| NCT01625559 | Safety and tolerability of MA09-hRPE cells in patients with Stargardt’s macular dystrophy (SMD) | hESC-derived RPE cells (MA09-hRPE) | Allogeneic | Stargardt’s macular dystrophy | South Korea | 3 | Sub-retinal transplantation | Not yet reported, recruitment status unknown |
| NCT03763136 | Treating heart failure with hPSC-CMs | hiPSCs- derived cardiomyocytes | Allogeneic | Heart failure | China | 5 | Injection into the myocardium | Recruiting |
| NCT03046407 | Treatment of dry age-related macular degeneration disease with retinal pigment epithelium-derived from human embryonic stem cells | hESC-derived RPE | Allogeneic | Dry age-related macular degeneration | China | 10 | Subretinal transplantation | Recruiting |
| NCT03944239 | Safety and efficacy of subretinal transplantation of clinical human embryonic stem cell-derived retinal pigment epitheliums in treatment of retinitis pigmentosa | hESC-derived RPE | Allogeneic | Retinitis pigmentosa | China | 10 | Subretinal transplantation | Recruiting |
| NCT03482050 | Study to evaluate transplantation of astrocytes derived from human embryonic stem cells, in patients with amyotrophic lateral sclerosis | hESC-derived astrocytes | Allogeneic | Amyotrophic lateral sclerosis | Israel | 21 | intrathecal (spinal) injection | Recruiting |
| NCT02755428 | Subretinal transplantation of retinal pigment epitheliums in treatment of age-related macular degeneration diseases | hESC-derived RPE | Allogeneic | Dry age-related macular degeneration | China | 10 | Subretinal transplantation | Recruiting |
| NCT03119636 | Safety and efficacy study of human ESC-derived neural precursor cells in the treatment of Parkinson’s disease | hESC- derived neural precursor cells | Allogeneic | Parkinson’s disease | China | 50 | Stereotaxic intra-striatal injection | Recruiting |
| NCT02286089 | Safety and efficacy study of opregen for treatment of advanced dry-form age-related macular degeneration | hESC-derived RPE | Allogeneic | Age-related macular degeneration | USA + Israel | 24 | Subretinal transplantation | Recruiting |
| NCT03877471 | Mesenchymal stem cells - like cell transplantation in women with primary ovarian insufficiency | hESC-derived MSC-like cells | Allogeneic | Primary ovarian insufficiency | China | 28 | Injection into ovaries bilaterally | Recruiting |
| NCT03841110 | FT500 as monotherapy and in combination with immune checkpoint inhibitors in subjects with advanced solid tumors | iPSC-derived NK cell cancer immunotherapy | Allogeneic | Advanced solid tumors | USA | 76 | Recruiting | |
| JPRN-UMIN000032989 | Clinical trial of human (allogeneic) induced pluripotent stem cell-derived cardiomyocyte sheet for severe cardiomyopathy | iPS cell-derived cardiomyocytes | Allogeneic | Ischemic cardiomyopathy | Japan | 3 | iPS cell-derived cardiomyocyte sheet transplantation | Recruitment complete |
| NCT03963154 | Interventional study of implantation of hESC-derived RPE in patients With RP due to monogenic mutation | hESC-derived RPE | Allogeneic | Retinitis pigmentosa due to monogenic mutation | France | 12 | single central subretinal implantation of a hESC-derived RPE monolayer in one eye | Recruiting |
| NCT03305029 | The safety and tolerability of sub-retinal transplantation of SCNT-hES-RPE cells in patients with advanced Dry AMD | SCNT hESC-derived RPE | Allogeneic | Dry age-related macular degeneration | South Korea | 3 | Subretinal transplantation | Recruitment status unknown |
| NCT02057900 | Transplantation of human embryonic stem cell-derived progenitors in severe heart failure (ESCORT) | hESC- derived CD15+Isl-1+ progenitors | Allogeneic | Ischemic heart disease | France | 10 | fibrin gel embedding hESC-derived CD15+Isl-1+ progenitors | Completed, results published18 |
| NCT01469832 | Safety and tolerability of sub-retinal transplantation of human embryonic stem cell-derived retinal pigmented epithelial (hESC-RPE) cells in patients with Stargardt’s Macular Dystrophy (SMD) | hESC-derived RPE cells (MA09-hRPE) | Allogeneic | Stargardt’s macular dystrophy | UK | 12 | Subretinal injection | Completed, results published61 |
| NCT01345006 | Sub-retinal transplantation of hESC-derived RPE(MA09-hRPE) cells in patients with Stargardt’s Macular Dystrophy | hESC-derived RPE cells (MA09-hRPE) | Allogeneic | Stargardt’s macular dystrophy | USA | 13 | Subretinal injection | Completed, results published5,62 |
| NCT01344993 | Safety and tolerability of sub-retinal transplantation of hESC-derived RPE (MA09-hRPE) cells in patients with advanced dry age-related macular degeneration | hESC-derived RPE cells (MA09-hRPE) | Allogeneic | Dry age-related macular degeneration | USA | 13 | Subretinal injection | Completed, results published5,62 |
| JPRN-UMIN000011929 | A Study of transplantation of autologous induced pluripotent stem cell (iPSC) derived retinal pigment epithelium (RPE) cell sheet in subjects with exudative age-related macular degeneration | hiPSC-derived RPE | Autologous | Exudative age-related macular degeneration | Japan | 6 | Transplantation of iPSC-derived RPE cell sheet | Complete, results published19 |
| NCT02590692 | Study of subretinal implantation of human embryonic stem cell-derived RPE cells in advanced Dry AMD | hESC-derived RPE | Allogeneic | Dry age-related macular degeneration | USA | 16 | Subretinal implantation | Active, not recruiting |
| NCT03839238 | Safety observation on hESC-derived MSC like cell for the meniscus injury | hESC- derived MSC like cell | Allogeneic | Meniscus injury | China | 18 | hESC- derived MSC like cells | Active, not recruiting |
| NCT02923375 | A study of CYP-001 for the treatment of steroid-resistant acute graft versus host disease | iPSC- derived MSCs | Allogeneic | Graft vs host disease | Australia + UK | 16 | IV infusion on two occasions | Active, not recruiting |
| NCT01691261 | A study of implantation of retinal pigment epithelium in subjects with acute wet age-related macular degeneration | hESC-derived RPE | Allogeneic | Age-related macular degeneration | UK | 2 | Implantation of RPE monolayer immobilized on a polyester membrane. | Active, not recruiting |
| NCT02749734 | Clinical study of subretinal transplantation of human embryo stem cell-derived retinal pigment epitheliums in treatment of macular degeneration diseases | hESC-derived RPE | Allogeneic | Macular degeneration Stargardt’s macular dystrophy | China | 15 | Subretinal transplantation | Recruitment status unknown |
| JPRN-UMIN000033564 | Kyoto trial to evaluate the safety and efficacy of iPSC-derived dopaminergic progenitors in the treatment of Parkinson’s disease | hiPSC-derived dopaminergic progenitors | Parkinson’s disease | Japan | 7 | Transplantation into the corpus striatum | Recruitment suspended | |
| JPRN-UMIN000026003 | A Study of transplantation of allogenic induced pluripotent stem cell (iPSC) derived retinal pigment epithelium (RPE) cell suspension in subjects with neovascular age-related macular degeneration | iPSC-derived RPE | Allogeneic | Neovascular age-related macular degeneration | Japan | 5 | Subretinal transplantation | Recruitment complete; follow-up continuing |
| ChiCTR-OCB-15007054 | Clinical study of subretinal transplantation of clinical human embryonic stem cells derived retinal pigment epitheliums in treatment of dry age-related macular degeneration diseases | hESC-derived RPE | Allogeneic | Dry age-related macular degeneration | China | 10 | Subretinal transplantation | Recruiting |
| ChiCTR-OCB-15005968 | The clinical trial of human embryonic stem cell-derived epithelial cells transplantation in the treatment of severe ocular surface diseases | hESC-derived epithelial cells | Allogeneic | Severe ocular surface disease | China | 20 | Corneal-epithelium-like-cell transplantation | Recruiting |
hESC human embryonic stem cells, hiPSC human induced pluripotent stem cell, RPE retinal pigment epithelium, hRPE human retinal pigment epithelium, MSC mesenchymal stem cell, UK United Kingdom.