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
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
A Phase 1/2a, Open Label, Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of TRX103 in Subjects with Moderate to Severe Treatment-Refractory Crohn’s Disease
Adult Refractory Crohn's Disease
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
Autologous Gene Therapy for Artemis-Deficient SCID
ART-SCID
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
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
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 Study of Inhibitory Interneurons (NRTX-1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Focal Seizures
Epilepsy - Bilateral Temporal Focal Seizures
Recruiting
A Two-Stage, Randomized, Controlled Trial Comparing the Safety and Efficacy of Iltamiocel with Placebo in the Treatment of Female Participants with Chronic Fecal Incontinence and a History of Obstetric Anal Sphincter Injury
Female Fecal Incontinence
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
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
A Feasibility Study Using CLINIMACS® for Alpha/Beta T-Cell Depletion in Stem Cell Transplant
Graft Vs Host Disease
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
A Phase I/II Study to Evaluate AZD5851 in GPC3+ Advanced/Recurrent Hepatocellular Carcinoma
Hepatocellular Carcinoma, Liver Cancer
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
Pancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Diabetes in the Intra-Muscular Site
Human pancreatic islets in intramuscular site
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
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
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
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 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 Multi-Center, Phase 2 Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell (GRASP, BMT CTN 2001)
Sickle Cell Disease
Recruiting
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
Sickle Cell Disease
Recruiting
Transplantation of CRISPR-Cas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
Sickle Cell Disease
Active, not recruiting
Phase I Study of Autologous CD8+ and CD4+ Engineered T Cell Receptor T Cells in Subjects With Advanced or Metastatic Solid Tumor
Solid tumors with KRAS G12V mutation
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
A Phase 2, adaptive, randomized, open-label, assessor-blinded active-controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus Standard of Care in patients suffering from systemic lupus erythematosus (SLE) with active, refract
Systemic Lupus Erythematosus (SLE) with Lupus Nephritis (LN)
Recruiting
Muscle Cell Mediated Therapy for Tongue Dysphagia: An Investigation of Cook MyoSite Autologous Muscle Derived Cells
Tongue Dysphagia
Recruiting
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
Transfusion-Dependent Beta Thalassemia (TDT)
Recruiting