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
Autologous Gene Therapy for Artemis-Deficient SCID	ART-SCID	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 Multicenter Study Evaluating the Safety and Tolerability of GCC19CART for Subjects With Relapsed or Refractory Metastatic Colorectal Cancer	Colorectal Cancer	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
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
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
A Phase I/II Study to Evaluate AZD5851 in GPC3+ Advanced/Recurrent Hepatocellular Carcinoma	Hepatocellular Carcinoma, Liver Cancer	Recruiting
Pancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Diabetes in the Intra-Muscular Site	Human pancreatic islets in intramuscular site	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, Multicenter, Open-Label Study of CC-95266 in Subjects With Relapsed and/or Refractory Multiple Myeloma	Multiple Myeloma	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
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 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
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
Transplantation of CRISPR-Cas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease	Sickle Cell Disease	Active, not 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
Muscle Cell Mediated Therapy for Tongue Dysphagia: An Investigation of Cook MyoSite Autologous Muscle Derived Cells	Tongue Dysphagia	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