Clinical Trials

As part of the CIRM Alpha Stem Cell Clinics Network, the UCSF Alpha Clinic is pursuing its goal to accelerate the development and delivery of cell and gene therapies to patients through high-quality clinical trials. 

For a complete list of trials across the CIRM Alpha Clinics Network, please visit the Alpha Clinic Network's clinical trials page.

 

Title Disease Statussort descending
SIngle-Stage, Open-Label, Safety and Efficacy Study of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase by Magnetic Resonance MR-guided Infusion Into Midbrain in Pediatric Patients With AADC Deficiency
AADC Deficiency
Recruiting
Pancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Diabetes in the Intra-Muscular Site
Human pancreatic islets in intramuscular site
Recruiting
A First-in-human (FIH) Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)
Drug-resistant unilateral mesial temporal lobe epilepsy (MTLE)
Recruiting
A Feasibility Study Using CLINIMACS® for Alpha/Beta T-Cell Depletion in Stem Cell Transplant
Graft Vs Host Disease
Recruiting
A Phase 1 Multicenter Study Evaluating the Safety and Tolerability of GCC19CART for Subjects With Relapsed or Refractory Metastatic Colorectal Cancer
Colorectal Cancer
Recruiting
Autologous Gene Therapy for Artemis-Deficient SCID
ART-SCID
Recruiting
A Phase I/II Study to Evaluate AZD5851 in GPC3+ Advanced/Recurrent Hepatocellular Carcinoma
Hepatocellular Carcinoma, Liver Cancer
Recruiting
A Phase 1 Dose Escalation and Expanded Cohort Study of P-MUC1C-ALLO1 in Adult Subjects With Advanced or Metastatic Solid Tumors
Solid Tumors
Recruiting
An Open-Label, Dose Escalation, Multi-Center Phase I/II Clinical Trial of ET140203 T Cells in Pediatric Subjects with Relapsed/Refractory Hepatoblastoma (HB), Hepatocellular Neoplasm-Not Otherwise Specified (HCN-NOS), or Hepatocellular Carcinoma (HCC)
Hepatoblastoma, Hepatocellular Carcinoma
Recruiting
Phase 1 Study of Autologous anti-EGFRvIII synNotch Receptor Induced anti-EphA2/IL-13Rα2 CAR (E-SYNC) T Cells in Adult Participants with EGFRvIII+ Glioblastoma
Glioblastoma, Brain Tumor
Recruiting
A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells
Beta-Thalassemia
Recruiting
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of JSP191 for Hematopoietic Cell Transplantation Conditioning to Achieve Engraftment and Immune Reconstitution in Subjects With SCID
Severe Combined Immune Deficiency Syndrome (SCID)
Recruiting
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell D
Sickle Cell Disease
Recruiting
A Multi-Center, Phase 2 Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell (GRASP, BMT CTN 2001)
Sickle Cell Disease
Recruiting
Muscle Cell Mediated Therapy for Tongue Dysphagia: An Investigation of Cook MyoSite Autologous Muscle Derived Cells
Tongue Dysphagia
Recruiting
Transplantation of CRISPR-Cas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
Sickle Cell Disease
Active, not recruiting
Open-label, Multicenter, Phase 1b/2 Clinical Trial to Evaluate the Safety and Efficacy of Autologous Anti-claudin 18.2 Chimeric Antigen Receptor T-cell Therapy in Subjects With Advanced Gastric, Pancreatic, or Other Specified Digestive System Cancers
Gastric/Pancreatic Cancer
Active, not recruiting
An Open-label Phase 1 Study to Evaluate the Safety and Efficacy of AB-1015 in Patients With Resistant/Refractory Epithelial Ovarian Cancer
Ovarian Cancer
Active, not recruiting
Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Subjects With Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis
GM1 Gangliosidosis
Active, not recruiting
Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-02: A Phase 1/2 Feasibility and Safety Study of CD19-CAR T Cell Immunotherapy for CD19+ Leukemia
Leukemia
Active, not recruiting
A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease
Sickle Cell Disease
Active, not recruiting