From: Research and Development Strategy for Future Embryonic Stem Cell-Based Therapy in Japan
| Study title | Cell type (product name) | Sponsor/collaborator | Trial location | Disease | Stage of trial | Cell delivery | Status of trial | Registration number | Estimated or actual study completion date | Note/reference |
|---|---|---|---|---|---|---|---|---|---|---|
| A. Eye disease | ||||||||||
| Safety and Tolerability of Sub-retinal Transplantation of hESC-Derived RPE (MA09-hRPE) Cells in Patients with Advanced Dry Age-Related Macular Degeneration (Dry AMD) | hESC-derived RPE (MA09-hRPE) |
Astellas Institute for Regenerative Medicine | United States | Dry AMD | Phase I/II | Cell suspension | Completed | NCT01344993 | August 1, 2015 | < Progress reports > Lancet. 2015 Feb 7;385(9967):509-16. Lancet. 2012 Feb 25;379(9817):713-20. < Follow-up study > Invest Ophthalmol Vis Sci. 2016 Apr 1;57(5):ORSFc1-9. |
| Sub-retinal transplantation of hESC-derived RPE (MA09-hRPE) cells in patients with SMD | hESC-derived RPE (MA09-hRPE) | Astellas Institute for Regenerative Medicine | United States | Stargardt’s macular dystrophy (SMD) | Phase I/II | Cell suspension | Completed | NCT01345006 | August 1, 2015 | |
| Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients | hESC-derived RPE (MA09-hRPE) | Astellas Institute for Regenerative Medicine | United States | Stargardt’s macular dystrophy (SMD) | Phase I/II | Cell suspension | Active, not recruiting | NCT02445612 | December 1, 2019 | Follow-up study for NCT1345006 |
| Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Patients With AMD | hESC-derived RPE (MA09-hRPE) | Astellas Institute for Regenerative Medicine | United States | Dry AMD | Phase I/II | Cell suspension | Active, not recruiting | NCT02463344 | December 1, 2019 | Follow-up study for NCT1344993 |
| Safety and tolerability of sub-retinal transplantation of hESC-RPE cells in patients with SMD | hESC-derived RPE (MA09-hRPE) | Astellas Institute for Regenerative Medicine | United Kingdom | Stargardt’s macular dystrophy (SMD) | Phase I/II | Cell suspension | Completed | NCT01469832 | September 1, 2015 | < Progress reports > Ophthalmology. 2018 Nov;125(11):1765-1775. |
| A Follow up Study to Determine the 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 (MA09-hRPE) | Astellas Institute for Regenerative Medicine | United Kingdom | Stargardt’s macular dystrophy (SMD) | Phase I/II | Cell suspension | Active, not recruiting | NCT02941991 | December 1, 2019 | Follow-up study for NCT1469832 |
| A Phase 1b Dose Escalation Evaluation of Safety and Tolerability and a Phase 2 Proof of Concept Investigation of Efficacy and Safety of ASP7317 for Atrophy Secondary to Age-related Macular Degeneration | hESC-derived RPE (ASP7317) | Astellas Institute for Regenerative Medicine | United States | AMD | Phase I/II | Cell suspension | Recruiting | NCT03178149 | October 1, 2026 | Clinical study of ASP7317, a new cell line to replace MA09-Hrpe |
| 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 (AMD) | hESC-derived RPE (MA09-hRPE) | CHABiotech (licensed from Astellas Institute) | Korea | Dry AMD | Phase I/II | Cell suspension | Unknown | NCT01674829 | April 1, 2016 | < Progress report > Stem Cell Reports. 2015 May 12;4(5):860-72. < Progress report of NCT01674829 > JAMA Ophthalmol. 2017 Mar 1;135(3):287-289. |
| Safety and Tolerability of MA09-hRPE Cells in Patients with Stargardt’s Macular Dystrophy (SMD) | hESC-derived RPE (MA09-hRPE) | CHABiotech (licensed from Astellas Institute) | Korea | Stargardt's macular dystrophy (SMD) | Phase I | Cell suspension | Unknown | NCT01625559 | June 1, 2015 | |
| A study of implantation of RPE in subjects with acute wet age-related macular degeneration | hESC-derived RPE (PF-05206388) | Pfizer/University College, London | United Kingdom | Wet AMD | Phase I | Membrane-immobilized monolayer sheet | Active, not recruiting | NCT01691261 | December 1, 2019 | < Progress report including nonclinical and clinical studies > Nat Biotechnol. 2018 Mar 19. doi: 10.1038/nbt.4114. |
| Retinal Pigment Epithelium Safety Study For Patients In B4711001 | hESC-derived RPE (PF-05206388) | Pfizer | United Kingdom | Wet AMD | Phase I | Membrane-immobilized monolayer sheet | Active, not recruiting | NCT03102138 | October 4, 2020 | Follow-up study for NCT01691261 |
| Safety and Efficacy Study of OpRegen for Treatment of Advanced Dry-Form Age-Related Macular Degeneration | hESC-derived RPE (OpRegen) | Lineage Cell Therapeutics (former BioTime)/Cell Cure Neurosciences | United States and Israel | Dry AMD | Phase I/II | Cell suspension | Recruiting | NCT02286089 | December 1, 2024 | https://lineagecell.com/products-pipeline/opregen/ < Nonclinical study > Transl Vis Sci Technol. 2017 Jun; 6(3): 17 |
| Study of subretinal implantation of human ESC-derived RPE cells in advanced dry AMD | hESC-derived RPE (CPCB-RPE1) | Regenerative Patch Technologies | United States | Dry AMD/geographic Atrophy | Phase I/II | Membrane- immobilized monolayer sheet | Active, not recruiting | NCT02590692 | June 1, 2023 | < Nonclinical study > Graefes Arch Clin Exp Ophthalmol. 2016 Aug;254(8):1553-65. < Progress report of clinical study (phase I/II) > Science Translational Medicine 04 Apr 2018: Vol. 10, Issue 435, eaao4097 http://www.sankeibiz.jp/business/news/180405/prl1804051101040-n1.htm |
| Clinical Study of Subretinal Transplantation of Human Embryo Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Macular Degeneration Diseases | hESC-derived RPE | Southwest Hospital | China | AMD and Stargardt | Phase I/II | Cell suspension | Active, not recruiting | NCT02749734 | December 1, 2019 | No available information |
| Subretinal Transplantation of Retinal Pigment Epitheliums in Treatment of Age-related Macular Degeneration Diseases | hESC-derived RPE | Chinese Academy of Sciences/Beijing Tongren Hospital | China | Dry AMD | Phase I/II | Cell suspension | Recruiting | NCT02755428 | December 1, 2019 | No available information |
| Treatment of Dry Age Related Macular Degeneration Disease With Retinal Pigment Epithelium Derived From Human Embryonic Stem Cells | hESC-derived RPE | Chinese Academy of Sciences/The First Affiliated Hospital of Zhengzhou University | China | Dry AMD | Phase I/II | Cell suspension | Recruiting | NCT03046407 | December 1, 2020 | No available information |
| Stem Cell Therapy for Outer Retinal Degenerations | hESC-derived RPE | Federal University of São Paulo | Brazil | Dry AMD/wet AMD/Stargardt | Phase I/II | Cell suspension or monolayer in a polymeric substrate | Unknown | NCT02903576 | June 1, 2019 | No available information |
| A Safety surveillance study in subjects with macular degenerative disease treated with human ESC-derived retinal pigment epithelial cell therapy | hESC-derived RPE (MA09-hRPE) | Astellas Institute for Regenerative Medicine | United States | Macular degeneration | Phase I/II | Cell suspension | Enrolling by invitation | NCT03167203 | December 1, 2029 | A long-term (up to 15 years) safety surveillance study |
| Safety and Efficacy of Subretinal Transplantation of Clinical Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Retinitis Pigmentosa | hESC-derived RPE | Qi Zhou/Beijing Tongren Hospital | China | Retinitis pigmentosa | Phase I | - | Recruiting | NCT03944239 | December 1, 2020 | No available information |
| Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation | hESC-derived RPE | Centre d'Etude des Cellules Souches | France | Retinitis pigmentosa (due to monogenic mutation) | Phase I/II | - | Recruiting | NCT03963154 | December 15, 2021 | No available information |
| B. Other disease | ||||||||||
| Transplantation of Human Embryonic Stem Cell-derived Progenitors in Severe Heart Failure (ESCORT) | hESC-derived CD15+ Isl-1+ cardiac progenitors | Assistance publique, Hôpitaux de Paris | France | Severe heart failure | Phase I | Cells embedded in fibrin patch | Completed | NCT02057900 | March 22, 2018 | < Nonclinical study > Eur Heart J. 2015 Mar 21;36(12):743-50. < Progress reports of clinical study > Eur Heart J. 2015 Aug 7;36(30):2011-7. J Am Coll Cardiol. 2018 Jan 30;71(4):429-438. |
| A Safety, Tolerability, and Efficacy Study of VC-01™ Combination Product in Subjects With Type I Diabetes Mellitus | hESC-derived pancreatic precursor cells (VC-01™ combination product) | ViaCyte/California Institute for Regenerative Medicine (CIRM) | United States/Canada | Type 1 diabetes | Phase I/II | PEC-01 cells encapsulated in a medical device | Active, not recruiting | NCT02239354 | January 1, 2021 | VC-01™, a combination product (PEC-01™ cells + Encaptra® DDS) (see PMID: 29369575) |
| One-Year Follow-up Safety Study in Subjects Previously Implanted With VC-01™ | hESC-derived pancreatic precursor cells (VC-01™ combination product) | ViaCyte | United States | Type 1 diabetes | Observational study | PEC-01 cells encapsulated in a medical device | Enrolling by invitation | NCT02939118 | November 1, 2021 | VC-01™ (PEC-Encap™) delivers the PEC-01 pancreatic progenitor cells in a immunoprotective device https://viacyte.com/products/pec%e2%80%90encap-vc-01 |
| A Safety and Tolerability Study of VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus | hESC-derived pancreatic precursor cells (VC-02™ combination product, aka PEC-Direct) | ViaCyte | Canada | Type 1 diabetes | Phase I | PEC-01 cells loaded into a delivery device | Completed | NCT03162926 | February 15, 2018 | VC-02™ (PEC-Direct™) delivers the PEC-01 pancreatic progenitor cells in a non-immunoprotective device https://viacyte.com/products/pec-direct |
| A Safety, Tolerability, and Efficacy Study of VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus and Hypoglycemia Unawareness | hESC-derived pancreatic precursor cells (VC-02™ combination product, aka PEC-Direct) | ViaCyte | United States | Type 1 diabetes | Phase I/II | PEC-01 cells loaded into a delivery device | Recruiting | NCT03163511 | March 1, 2022 | |
| Safety Study of GRNOPC1 in Spinal Cord Injury | hESC-derived oligodendrocyte progenitors (GRNOPC1/AST-OPC1) | Asterias Biotherapeutics | United States | Spinal cord injury | Phase I | Cell suspension | Completed (took over from Geron) | NCT01217008 | July 1, 2013 | < Nonclinical study > Regen Med. 2015 Nov;10(8):939-58. |
| Dose Escalation Study of AST-OPC1 in Spinal Cord Injury | hESC-derived oligodendrocyte progenitors (AST-OPC1) | Asterias Biotherapeutics | United States | Spinal cord injury | Phase I/II | Cell suspension | Completed | NCT02302157 | December 1, 2018 | https://www.cirm.ca.gov/our-progress/awards/phase-iiia-dose-escalation-safety-study-ast-opc1-patients-cervical-sensorimotor (You can check progress report on the trial) |
| A Study to Evaluate the Safety of Neural Stem Cells in Patients With Parkinson's Disease | Human parthenogenetic stem cell-derived neural stem cells(ISC-hpNSC) | Cyto Therapeutics/International Stem Cell Corporation | Australia | Parkinson's disease | Phase I | Cell suspension | Active, not recruiting | NCT02452723 | June 1, 2020 | < Nonclinical study > Sci Rep. 2016 Sep 30;6:34478. |
| Safety and Efficacy Study of Human ESC-Derived Neural Precursor Cells in the Treatment of Parkinson’s Disease | Human embryonic stem cells-derived neural precursor cells | Chinese Academy of Sciences/The First Affiliated Hospital of Zhengzhou University | China | Parkinson's Disease | Phase I/II | Cell suspension | Recruiting | NCT03119636 | December 1, 2020 | < Nonclinical study > Stem Cell Reports. 2018 Jul 10;11(1):171-182. |
| A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS) | Astrocytes derived from human embryonic stem cells (AstroRx) | Kadimastem | Israel | ALS (amyotrophic lateral sclerosis) | Phase I/II | Cell suspension | Recruiting | NCT03482050 | August 1, 2020 | < Nonclinical study > Stem Cell Res Ther. 2018; 9: 152. |
| Safety Observation on hESC Derived MSC Like Cell for the Meniscus Injury | hESC-derived MSC-like cell | Tongji Hospital/Chinese Academy of Sciences | China | Meniscus injury | Phase I | - | Active, not recruiting | NCT03839238 | September 30, 2020 | No available information |
