Stage of Program: Clinical Trial, Phase 2
A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease
A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach
A Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy
Phase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect
The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair and The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele
UC Davis is conducting a clinical trial for in utero repair of myelomeningocele (MMC), the most severe form of spina bifida. MMC is a birth defect that occurs due to incomplete closure of the developing spinal cord, resulting in neurological damage to the exposed cord. This damage leads to lifelong lower body paralysis, and bladder and […]
Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease
Boston Children’s Hospital is conducting a gene therapy clinical trial for sickle cell disease (SCD). This project is part of an agreement between CIRM and the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, to co-fund cell and gene therapy programs under the NHLBI’s “Cure Sickle Cell” Initiative. The […]
Mesenchymal Stromal Cells for ARDS (COVID positive and COVID negative)
Acute respiratory distress syndrome (ARDS) is a life-threatening lung injury that occurs when fluid leaks into the lungs. As a result, breathing becomes difficult and oxygen cannot get into the body. ARDS is one of the most serious and lethal consequence of COVID-19. This clinical trial will use Mesenchymal Stromal Cells (MSCs) for the treatment of ARDS, […]
A Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects with Retinitis Pigmentosa
Retinitis pigmentosa is blinding eye disease that affects approximately 150,000 individuals in the US and 1.5 million people around the world. It is caused by the destruction of light-sensing cells in the back of the eye known as photoreceptors. This leads to gradual vision loss and eventually blindness. The approach for this trial involves injecting […]
Phase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus
Children with type 1 diabetes (T1D) face lifelong struggles with controlling their blood sugar levels and, despite careful management, an increased risk of severe complications. Currently, there is no approved therapy that maintains or restores the insulin-producing pancreatic beta cells that are destroyed by this disease. Researchers at Caladrius Biosciences will take cells, called regulatory T cells […]
Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy
Capricor is using donor cells derived from the heart to treat patients suffering from Duchenne Muscular Dystrophy (DMD), a genetic disorder that leads to progressive muscle degeneration, including heart muscle. One of the leading causes of death for children with DMD is heart failure and the aim of this treatment is to help improve heart […]