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	Status
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
A Phase 1 Multicenter Study Evaluating the Safety and Tolerability of GCC19CART for Subjects With Relapsed or Refractory Metastatic Colorectal Cancer	Colorectal Cancer	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 Phase 1 Dose Escalation and Expanded Cohort Study of P-MUC1C-ALLO1 in Adult Subjects With Advanced or Metastatic Solid Tumors	Solid Tumors	Recruiting
Phase 1, Open-Label, Single Center Study of KYV-101, an Autologous Fully-Human Anti-CD19 Chimeric Antigen Receptor T Cell (CD19 CAR T) Therapy, in Participants With Treatment Refractory Progressive Multiple Sclerosis	Multiple Sclerosis	Recruiting
PNOC018 A Phase 1 Clinical Trial of Autologous T Cells Expressing a TCR Specific for H3.3K27M With Inhibition of Endogenous TCR (KIND T Cells) in HLA-A*0201-positive Participants With Newly Diagnosed H3.3K27M-positive Diffuse Midline Gliomas	Diffuse Midline Glioma	Recruiting
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)	Transfusion-Dependent Beta Thalassemia (TDT)	Recruiting
Safety and Anti-HIV Activity of Autologous CD4+ and CD8+ T Cells Transduced With a Lentiviral Vector Encoding Bi-specific Anti-gp120 CAR Molecules (LVgp120duoCAR-T) in Anti-retroviral Drug-treated HIV-1 Infection	HIV	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 Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)	Sickle Cell Disease	Recruiting
A Phase 1 Clinical Trial of Anti-CD19 Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Non-Hodgkin Lymphoma	Non-Hodgkin Lymphoma	Recruiting
A Multi-Center, Phase 2 Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell (GRASP, BMT CTN 2001)	Sickle Cell Disease	Recruiting
A Phase 1b Study of WU-NK-101 in Combination With Cetuximab for Advanced and/or Metastatic Colorectal Cancer (CRC) and Advanced and/or Metastatic Squamous Cell Carcinoma of the Head and Neck (SCCHN)	Metastatic Colorectal Cancer Squamous Cell Carcinoma of the Head and Neck	Recruiting
Muscle Cell Mediated Therapy for Tongue Dysphagia: An Investigation of Cook MyoSite Autologous Muscle Derived Cells	Tongue Dysphagia	Recruiting
A Phase 1, Open-Label, Dose-Finding Study of BMS-986453, Dual Targeting BCMAxGPRC5D Chimeric Antigen Receptor T Cells, in Participants With Relapsed and/or Refractory Multiple Myeloma	Multiple Myeloma (MM)	Recruiting
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
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 Single-Center, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Alpha Thalassemia Major	Alpha Thalassemia Major	Enrolling by invitation
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
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

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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.

Work with the UCSF Alpha Clinic