Clinical study registry for hPSC-based cell therapies

This is a Phase I dose-finding study of FT538 as monotherapy in acute myeloid leukemia (AML) and in combination with monoclonal antibodies in multiple myeloma (MM). The study will consist of a dose-escalation stage and an expansion stage where participants will be enrolled into indication-specific cohorts.

This is a Phase I dose-finding study of FT819 as monotherapy and in combination with IL-2 in subjects with relapsed/refractory B-cell Lymphoma, Chronic Lymphocytic Leukemia and Precursor B-cell Acute Lymphoblastic Leukemia. The study will consist of a dose-escalation stage and an expansion stage where participants will be enrolled into indication-specific cohorts.

This is a Phase 1 dose-finding study of FT-516 in combination with monoclonal antibodies in subjects with advanced solid tumors. The study will consist of a dose-escalation stage and an expansion stage where participants will be enrolled into indication-specific cohorts.

This is a Phase I dose-finding study of FT596 as monotherapy and in combination with Rituximab or Obinutuzumab in subjects with relapsed/refractory B-cell Lymphoma or Chronic Lymphocytic Leukemia. The study will consist of a dose-escalation stage and an expansion stage where participants will be enrolled into indication-specific cohorts.

Subjects who previously took part in the FT500-101 study and received allogeneic NK cell immunotherapy will take part in this long term follow-up study. Subjects will automatically enroll into study FT-003 once they have withdrawn or complete the parent interventional study. The purpose of this study is to provide long-term safety and survival data for subjects who have participated in the parent study. No additional study drug will be given, but subjects can receive other therapies for their cancer while they are being followed for long term safety in this study.

The purpose of this study is to is to evaluate the occurrence of late onset (i.e., greater than 5 years after treatment) safety events of special interest in participants who have received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in an AIRM-sponsored clinical trial. The events of special interest are adverse events (AEs) that are presumed to have a potential causal relationship to the hESC-RPE cells.

Background: Age-related macular degeneration is a common eye disease in people over 50. The "dry" form of the disease can worsen into geographic atrophy, causing blind spots. Researchers want to learn if replacing older eye cells with younger ones can help treat this disease. Objective: To test the safety of putting cells inside the eye as a possible future treatment for dry age-related macular degeneration. Eligibility: People ages 55 and older who have geographic atrophy with loss of vision. People who have had "wet" macular degeneration in either eye are NOT eligible. Design: Participants will be screened with: - Medical history - Physical exam - Blood and urine tests - Eye exam - Eye photos - Fluorescein angiography. An intravenous (IV) line is placed in an arm vein. A dye is injected. A camera takes pictures of the dye as it flows through the eyes' blood vessels. - Electroretinography. An electrode is taped to participants' forehead. They sit in the dark. After 30 minutes, numbing eye drops and contact lenses are placed in their eyes. They watch flashing lights. - Tuberculosis test - Chest X-ray - Electrocardiography. Sticky pads are placed on participants' chest to record the heart's electrical activity. Participants will have at least 14 study visits over 5 and a half years. They will repeat screening tests. Participants will have retinal pigment epithelium (RPE) transplantation surgery in one eye. For this, cells from participants' blood are turned into RPE cells. These cells are placed in their eye through a cut in their retina. They will get dilating eye drops, an IV line, and anesthesia that may make them sleep. A gas bubble will be put in their eye to help it heal. Participants will be contacted yearly for up to 15 years.

This is a single center Phase I clinical trial of FT516 administered intraperitoneally (IP) once a week for 3 consecutive weeks for the treatment of recurrent gynecologic cancers. As this is an early 1st in human study and the 1st intraperitoneal infusion of FT516, the safety of FT516 is confirmed prior to adding enoblituzumab as an intravenous infusion approximately 1 week prior to the 1st dose of FT516 and every 3 weeks beginning on Day 22 (1 week after the last dose of FT516). Each dose of FT516 is followed directly by an IP infusion of interleukin-2 (IL-2) to facilitate natural killer (NK) cell survival. A short course of outpatient lymphodepletion chemotherapy is given prior to the 1st dose of FT516.

This is a Phase 1/1b dose-finding study of FT516 as monotherapy in acute myeloid leukemia (AML) and in combination with CD20 directed monoclonal antibodies in B-cell lymphoma. The study will consist of a dose-escalation stage and an expansion stage where participants will be enrolled into indication-sepcific cohorts.

This Phase I open-label dose escalation study is conducted in two stages with a primary endpoint to identify the maximum tolerated dose (MTD) of FT538 when administered with daratumumab in patients 12 years and older with advanced acute myeloid leukemia (AML) and related myeloid diseases.

This is a Phase I multi-center study to evaluate the safety of FT596 when given with rituximab as relapse prevention in patients who have undergone an autologous hematopoietic stem cell transplant (auto-HSCT) for diffuse large or high-grade B cell lymphoma.

Evaluation of the safety and tolerability of escalating doses of NR1 administered intracerebrally at a single time-point post-injury to subjects with chronic ISS with or without cortical stroke.

The main objective of the study is evaluation of the safety and tolerability of OpRegen - human embryonic stem cell-derived retinal pigment epithelial (RPE)cells. The study will also include initial exploration of the ability of transplanted OpRegen cells to engraft, survive, and moderate disease progression.

The BioVAT-HF trial will test the hypothesis that cardiomyocyte implantation via engineered heart muscle (EHM), the proposed investigational medicinal product (IMP; designated "Biological Ventricular Assist Tissue" or BioVAT), results in sustainable remuscularization and biological enhancement of myocardial performance in the failing heart. EHM are constructed from defined mixtures of induced pluripotent stem cell (iPSC)-derived cardiomyocytes and stromal cells in a bovine collagen type I hydrogel. Comprehensive preclinical testing confirmed the rationale for the clinical translation of the myocardial remuscularization strategy by EHM implantation. The patient target population for EHM therapy is patients suffering from advanced heart failure with reduced ejection fraction (HFrEF; EF: ≤35%) and no realistic option for heart transplantation.

SCUlpTOR is a 24-month research study from the Institute of Bone and Joint Research (IBJR) at the University of Sydney, evaluating the efficacy and cost-effectiveness of stem cell injections compared with placebo in people with knee OA. Currently, the medical opinion about stem cell therapy for treating osteoarthritis remains highly controversial, given the considerable cost of treatment and very limited scientific evidence of efficacy and safety. Therefore, we aim to conduct this trial to ascertain whether or not intra-articular stem cell injections improve symptoms and slow disease progression in people with mild to moderate knee OA. The stem cells that we used was originally sourced from a healthy donor/master cell bank following standard manufacturing process and release tests to optimise safety and batch to batch reproducibility.

This study intends to evaluate the safety and exploratory efficacy of transplantation therapy using neural precursor cells (PSA-NCAM(+) NPC) derived from the human embryonic stem cell line for the treatment of paralysis and other related symptoms from sub-acute spinal cord injury. This clinical study is a single center, open label, single group, phase 1/2a clinical study conducted to evaluate the safety and exploratory efficacy of transplant of PSA-NCAM(+) NPC in patients with sub-acute spinal cord injury. Upon written consent by the subject of voluntary participation in the clinical study, participation in the clinical study is determined by conducting necessary examinations and tests 4 to 14 days before administration of the investigational product in accordance with the protocol. Re-evaluation for conformity is conducted 1 to 3 days prior to administration of the investigational product for maintenance of suitability, and subjects with damage to C4-C7 cords diagnosed as AIS-A are administered with PSA-NCAM(+) NPC. When the Dose Limiting Toxicity (DLT) is not presented in the first three subjects administered with PSA-NCAM(+) NPC, two additional patients are added to the clinical study. When the DLT is presented in two or more of the first three patients, the clinical study is discontinued; when the DLT is presented in one of the three patients, three new patients are added. In case of presentation of the DLT in at least one of the three additional patients, the study is discontinued; the clinical study is continued only when the DLT is not presented in all three patients. Screening visit (Visit 1), surgery and recovery visit (Visit 2 to Visit 6), follow-up visit (Visit 7 to Visit 8 + phone screening I, II, III), additional visit (Visit 9 to Visit 10), and close-out visit (Visit 11) are conducted. A clinical study period of at least 68 weeks is secured after Visit 6 (at least 5 visits and 3 phone screenings). The allowed period of visits scheduled thereafter is ±3 days for Visits 4 to 6, ±7 days for Visits 7 to 8, and ±14 days for Visits 9 to 11. All subjects are to be conducted of follow-up study of a period of 1 year and 5 months at Weeks 1, 2, 4, 8, 12, 24, 48, and 72 after surgery.

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This clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and to monitor for potential side effects.

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The purpose of this trial is to evaluate the long-term safety in subjects previously implanted with VC-01™ combination product.

The Phase I/IIa clinical trial is designed to assess the feasibility of delivery and safety of Human Embryonic Stem Cell-Derived RPE Cells on a parylene membrane (CPCB-RPE1) in patients with advanced, dry age-related macular degeneration. Primary Objective: To test the safety and tolerability of CPCB-RPE1 during and after subretinal implantation in patients with geographic atrophy with evidence of involvement of the central fovea. Secondary Objective: To assess visual acuity, visual field, and retinal function after CPCB-RPE1 implantation. Implanted and fellow eyes will be compared post-implantation to assess the ability of the implant to prevent disease progression. Exploratory Objectives: To assess the feasibility of measuring the change in area of geographic atrophy over time using spectral domain optical coherence tomography or fundus autofluorescence.

The purpose of this trial is to test if VC-02™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and Hypoglycemia Unawareness and maintained safely for up to two years. It will also test if VC-02 is an effective treatment for these subjects.

This study primarily aimed to evaluate the safety of human embryonic stem cell (hESC)-derived mesenchyma stem cells in interstitial cystitis.

To observe the clinical safety of intrauterine injection of human embryonic stem cell derived mesenchymal cells in the treatment of moderate and severe intrauterine adhesion, and to preliminarily explore its clinical effectiveness in promoting endometrial regeneration and repair.

This study is for adults 50 years or older who are losing their clear, sharp central vision. Central vision is needed to be able to read and drive a car. They have been diagnosed with dry age-related macular degeneration (called dry AMD). The macula is the center part of the back of the eye that allows you to see fine detail. In an advanced stage of this disease, areas of the macula die (atrophy), resulting in vision loss. This is called geographic atrophy. This study is looking at a new treatment called ASP7317. It is for slowing or reversing atrophy in dry AMD. ASP7317 is a specially created type of cells derived from human stem cells. ASP7317 cells are injected into the macula of the eye while the person is under anesthesia (local or general). An immunosuppressive medicine (tacrolimus) is also taken around the time of injection of the cells to prevent the body from rejecting them. This study looks at how safe ASP7317 is at 3 different dose levels. Researchers want to learn if the different dose levels of ASP7317 work without causing unwanted medical problems. Each of the 3 doses will be given to 2 groups of people. The first group will be those who have severe vision loss. The second group will be those who have moderate vision loss. The doses are low, medium and high numbers of cells. Tacrolimus will be taken by mouth for 33 days, starting around the time of the injection of ASP7317. In addition, medicines to prevent infection will be taken by mouth for up to 4 weeks starting around the time ASP7317 cells are injected. Each week for the first 4 weeks after the ASP7317 cells have been injected, people taking part in the study will visit the clinic so the researchers can make assessments. Then they will visit again, at weeks 6, 8, 12, 16, 26, and 52 (last week of the study). A substudy will be available at some clinics. These clinics will use a special camera that will allow researchers to look at images of the macular atrophy over time.

This clinical study is looking at a vaccine called AST-VAC2 in adult patients with non-small cell lung cancer (NSCLC) in the advanced and adjuvant settings. The main aim of the study: If the dose can be given safely to patients, learn more about the potential side effects of the vaccine and how they can be managed and also what happens to AST-VAC2 inside the body (looking for effects in the blood, skin or tumour).

HAES is derived from human ES cells and directed to differentiate to cells with ammonia metabolism capacity. HAES transplantation is expected as an effective bridging therapy to liver transplantation for patients with urea cycle disorder. This study is conducted to assess the safety and efficacy of HAES transplantation for the neonatal onset patients who have difficulty in instant liver transplantation surgery due to low body weight (6 kg or less).

FT500 is an off-the-shelf, iPSC-derived NK cell product that can bridge innate and adaptive immunity, and has the potential to overcome multiple mechanisms of immune checkpoint inhibitor (ICI) resistance. The preclinical data provide compelling evidence supporting the clinical investigation of FT500 as monotherapy and in combination with ICI in subjects with advanced solid tumors.

This is a Phase I study with the primary objective of identifying the maximum tolerated dose (MTD) of FT516 using 3 dose-escalation strategies (number of doses and cell dose) for the treatment of coronavirus disease 2019 (COVID-19). This study provides initial estimates of safety and efficacy based on stable respiratory function, as well as, determining the feasibility for full-scale studies designed both for efficacy and safety.

This project intends to transplant a clinical level human embryonic stem cells derived retinal pigment epithelium into subretinal space to treat eetinitis pigmentosa(RP) diseases. Through the statistical analysis EDTRS, BCVA, OCT, ERG, Fluorescein angiography, Ophthalmic AB ultrasound changes between before and after the treatment to assess the efficacy and safety 0f RPE transplants to treat RP disease.

There is a high incidence of women suffering from Primary Ovarian Insufficiency (POI). So far, there was no treatment sufficient enough to cure POI. Cell therapy is a rapidly developing field and have shown immense promise in the treatment of ovarian dysfunction. In this study, the investigator will evaluate the safety of MSC-like cell therapy in women suffering from POI.

Phase I/II open-label, safety, tolerability and preliminary efficacy study of implantation into one eye of hESC-derived RPE (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)) in patients with retinitis pigmentosa due to monogenic mutation. Study non randomized single group assignment consisting in 2 sequential cohorts of patients: - First cohort of 2 patients with very advanced loss of visual acuity (legally blind) - Second cohort of 10 patients with less advanced loss of visual acuit.

This is a pilot, multi-centre, open-label randomised controlled study to assess the early efficacy of intravenous (IV) administration of CYP-001 in adults admitted to an intensive care unit (ICU) with COVID-19.

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This is a single center, single arm and open-label study to investigate the safety and efficacy of iPS-NCS with Parkinson's Disease.

This is a single centre, single arm, open-label study, to investigate the safety and efficacy of Autologous induced islet body With Type 1 diabetes.

This is a single centre, single arm, open-label study, to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.

The purpose of this trial is to test if VC-01™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and maintained safely for two years. It will also test if VC-01 is an effective treatment for subjects with Type 1 Diabetes.

A phase1/2, open label, dose escalation, safety and early efficacy study of CAStem for the treatment of severe COVID-19 associated with or without ARDS.

This is a single centre, single arm, open-label, to investigate the safety and efficacy of EPC transplantation in the brain.

This project intends to transplant human embryonic stem cells derived retinal pigment epitheliums into subretinal space of patients to treat dry age-related macular degeneration(dry-AMD). And we will assess the safety and efficacy of RPE transplants to treat dry AMD.

This study will evaluate the safety and efficacy of intracerebral transplantation of human embryonic stem cells-derived neural precursor cells in patients with Parkinson's Disease.

Heart failure has a high morbidity and mortality because the heart is one of the least regenerative organs in a human body. Drug treatments for heart failure manage symptoms but do not restore lost myocytes. Cellular replacement therapy is a potential approach to repair damaged myocardial tissue, restore cardiac function, which has become a new strategy for the treatment of heart failure. The purpose of this study is to assess the safety, feasibility and efficacy of intramyocardial delivery of regenerated cardiomyocytes at the time of coronary artery bypass grafting in patients with chronic ischemic cardiomyopathy.

This is a safety follow-up study. Patients enrolled in B4711001 will be followed for a further 4 years with regular visits to assess safety.

Human embryonic stem cell derived mesenchymal stem cells like cell for the meniscus injury, and observe the safety of the cells for meniscus injury

The purpose of this study is to assess the safety, tolerability and efficacy of two infusions of CYP-001 in adults with steroid-resistant GvHD.

This study will evaluate the safety of an investigational cell transplantation therapy, ISC-hpNSC, in patients with Parkinson's disease. All patients will receive the therapy, which consists of human neural stem cells. Three dose levels will be examined in the study.

To evaluate the safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry AMD To evaluate the safety of the surgical procedures when used to implant MA09-hRPE cells To assess the number of hRPE cells to be transplanted in future studies To evaluate on an exploratory basis potential efficacy endpoints to be used in future studies of MA09-hRPE cellular therapy.

This is a study of transplantation of Astrocytes derived from human embryonic stem cells, in patients with Amyotrophic Lateral Sclerosis (ALS). There will be no change in the routine ALS treatment of the patients enrolled into the study. Treatment will be administered in addition to the appropriate standard of care treatment. The study hypothesis is that transplantation of Astrocyte(AstroRx) cells can compensate for the malfunctioning of patients' own astrocytes by restoring physiological capabilities like the reuptake of excessive glutamate, reducing oxidative stress, reducing other toxic compounds, as well as by secreting different neuroprotective factors.

Based on the safety evaluation of primates, the best cell transplantation scheme was integrated. One patient with CHF caused by coronary heart disease, one patient with CHF caused by dilatation and one patient with CHF caused by Keshan disease were selected and treated with autologous iPS differentiated cardiomyocyte intravenous transplantation. The safety evaluation of human body was completed and combined with subjective and objective indexes respectively. Structural and functional indicators were used to evaluate the therapeutic effect of cell transplantation. The results of animal experiments confirmed the safety and effectiveness of intravenous myocardial cell transplantation, and clarified its possible mechanism.

Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related macular degeneration in whom there is rapidly progressing vision loss.

The purpose of this study was to determine the safety and therapeutic effect of sub-retinal transplantation of human embryo stem cell derived retinal pigment epitheliums (hESC-RPE) in patients with macular degeneration diseases, and explore new treatment modalities for macular degeneration diseases (Age-related macular degeneration and Stargardt's macular dystrophy).

The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.

The purpose of his study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry Age-Related Macular Degeneration (AMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells.

The purpose of this study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced Stargardt's Macular Dystrophy (SMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells.

This study is a Phase I/II , open label,non randomized, prospective study to determine the safety of human embryonic stem cell derived Retinal pigmented epithelium (hESC RPE) sub retinal injections versus hESC RPE seeded on a polymeric substrate implanted in the sub retinal space.

To evaluate the safety and tolerability of human somatic cell nuclear transfer embryonic stem cell derived retinal pigmented epithelial(SCNT-hES-RPE) cellular therapy in patients with advanced dry AMD.

This is a single centre, single arm, open-label study, to investigate the safety and efficacy of induction of neural stem cells transplantation in the brain.

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The purpose of this study is to evaluate the safety of cross sequential escalating doses of AST-OPC1 administered among 5 cohorts at a single time-point between 21 and 42 days post injury, inclusively, to subjects with subacute cervical spinal cord injuries (SCI).

The purpose of the study is to assess the feasibility and safety of a transplantation of cardiac-committed progenitor cells derived from human embryonic stem cells in patients with severe heart failure.

The purpose of this trial is to test if VC-02™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and maintained safely for up to four (4) months.

In order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the differentiation of hematopoietic stem cells in vivo and the hemoglobin beta-chain (HBB) protein expression in the body of recovery, etc., as well as to make a research on the efficacy and safety of hematopoietic stem cells from beta-thalassemia induced pluripotent stem cells.

This project intends to transplant a clinical level human embryonic stem cells derived retinal pigment epithelium into subretinal space to treat eetinitis pigmentosa(RP) diseases.

To evaluate the safety of 3 regimens of short-term, low-dose systemic IMT as rejection prophylaxis prior to and/or following transplant of MA09-hRPE cells.

Pathologic myopia is a major cause of legal blindness worldwide. In myopic macular degeneration (MMD), there is degeneration of the retinal pigment epithelial (RPE) layer, and associated photoreceptors, resulting in vision loss. There is currently no standard treatment for MMD. Transplantation of intact sheets of RPE and suspensions of isolated individual RPE cells as well as autologous translocation of RPE cells has been attempted as treatment for AMD. Human photoreceptors are comprised of two cell types-rods and cones. Both have a close relationship with the outermost retinal cells, the retinal pigmented epithelium (RPE). The RPE is located between the choroid and the photoreceptors. The RPE maintains photoreceptor function by recycling photopigments,delivering, metabolizing and storing vitamin A, phagocytosing rod photoreceptor outer segments, transporting iron and small molecules between retina and choroid, maintaining Bruch's membrane and absorbing stray light to allow better image resolution. In essence, the RPE layer is critical to the function and health of photoreceptors and the retina as a whole. Human PRE (hRPE) transplantation may be a viable option for treatment of degenerative diseases of the retina. MA09-hRPE cells are fully differentiated human RPE cells derived from embryonic stem cells. Transplanted hRPE cells prepared by Advanced Cell Technology have been studied in rodent models of macular degenerative disease. The data suggests that the subretinal injection of ACT's hRPE cell products rescues, or at least delays, loss of visual function in two animal models of retinal degenerative diseases. The main purpose of this study is to evaluate the safety and tolerability of MA09-hRPE cellular therapy in patients with Myopic Macular Degeneration (MMD). Another objective is to evaluate potential efficacy endpoints to be used in future studies of RPE cellular therapy.

The purpose of this study is: To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD . To evaluate potential efficacy endpoints to be used in future studies RPE cellular therapy.

This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with dry Age Related Macular Degeneration (AMD) and to perform exploratory evaluation of potential efficacy endpoints to be used in future studies retinal pigment epithelium (RPE) cellular therapy.

This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD).

The purpose of this study is: To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD Group. When-MA09-hRPE cell transplantation to evaluate the safety of surgical procedures. In future studies intended to assess the number of transplanted hRPE cells. In the past, MA09-hRPE cell therapy used in the study was to evaluate the validity of the potential. Homologous retinal pigment epithelial cells derived from embryonic stem cells, future studies of drugs that are used in representing the potential validity to evaluate the optimal dose.

The purpose of the study is to evaluate the safety of GRNOPC1 administered at a single time-point between 7 and 14 days post injury, inclusive, to patients with neurologically complete spinal cord injuries (SCI).

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This project intends to transplant human embryonic stem cells derived retinal pigment epitheliums into subretinal space of patients to treat dry age-related macular degeneration(dry-AMD).And we will assess the safety and efficacy of RPE transplants to treat dry AMD.

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