Early-Phase Clinical Trials at UCSF: Pediatric Cancer

Standard treatments are effective for many childhood cancers, but some require extra attention or innovation. At UCSF Benioff Children’s Hospitals, we offer our young patients easy access to early-phase clinical trials, which are studies of promising new therapies.

Enrolling in a clinical trial can provide access to drugs or other treatments that aren’t available anywhere else, offering families hope when their children's previous treatments were unsuccessful. Patients who participate also contribute to scientific research that can help others in the future.

Many of our doctors lead national research groups working to develop new therapies for brain tumors, leukemia, lymphoma and other cancers that affect children. A dedicated team of nurses and coordinators oversees our clinical trials, ensuring that the data we collect is robust and reliable. What's more, we are the only Northern California hospital to be designated an early-phase clinical trials core site for the Children’s Oncology Group, an organization funded by the National Cancer Institute and dedicated to developing better treatments for pediatric cancers.

Learn more about the comprehensive care we offer through our Pediatric Cancer Program.

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For more information about a clinical trial, use the links below to express your interest.

Active early-phase trials

Brain Tumors

NCT04323046: Immunotherapy Before and After Surgery for Treatment of Recurrent or Progressive High Grade Glioma in Children and Young Adults

NCT04485559: Trametinib and Everolimus for Treatment of Pediatric and Young Adult Patients With Recurrent Gliomas

NCT04684368: Study of a New Way to Treat Children and Young Adults with a Brain Tumor Called NGGCT

NCT04696029: DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

NCT04732065: ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors

NCT04870944: CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma

NCT05009992: Combination Therapy for the Treatment of Diffuse Midline Gliomas

NCT05057702: Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma

NCT05465174: Nivolumab and DAY101 for Treatment of Craniopharyngioma in Children and Young Adults

NCT05887882: IntrTumoral Injections of Natural Killer Cells for Recurrent Malignant Pediatric Brain Tumors

NCT06368817: Study of Lower Radiotherapy Dose to Treat Children With CNS Germinoma

NCT06861244: Embryonal Tumor With Multilayered Rosettes

NCT06894979: Testing the Addition of an Anti-Cancer Drug, AZD1390, During Radiation Therapy for Newly Diagnosed High-Grade Glioma, Diffuse Midline Glioma, or Diffuse Intrinsic Pontine Glioma

NCT07226453: Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependymoma

NCT07447076: Study of Novel Therapies for Young People With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)

NCT07506239: Efficacy of Laser Interstitial Thermal Therapy in Young Persons With Low-grade Glioma

Leukemia, Lymphoma, Myeloproliferative, and Histiocytic Disorders

NCT02981628: Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory CD22 Positive B Acute Lymphoblastic Leukemia

NCT03719105: Chemoimmunotherapy and Allogeneic Stem Cell Transplant for NK T-cell Leukemia/Lymphoma

NCT04079179: Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders

NCT04546399: Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)

NCT04811560: Phase 1/2 Study of Bleximenib in Participants with Acute Leukemia (cAMeLot-1)

NCT04851119: Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors

NCT04870944: CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma

NCT04972942: Targeted Immunotherapy After Myeloablative TBI-Based Conditioning & AlloHCT in CAYA with High Risk T-Cell ALL & Lymphoma

NCT05082519: Caloric Restriction and Activity to Reduce Chemoresistance in B-ALL

NCT05292664: Venetoclax Basket Trial for High Risk Hematologic Malignancies

NCT05533775: Study to Evaluate Glofitamab Monotherapy and Glofitamab + Chemoimmunotherapy in Pediatric and Young Adult Participants with Relapsed/Refractory Mature B-Cell Non-Hodgkin Lymphoma

NCT05761171: Study of Revumenib in Combination With Chemotherapy for Patients Diagnosed With Relapsed or Refractory Leukemia

NCT05828069: Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

NCT05849662: Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

NCT06247787: Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy

NCT06317662: Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly

Neuroblastoma

NCT02559778: Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

NCT02679144: Neuroblastoma Maintenance Therapy Trial

NCT03561259: Study of Therapeutic Iobenguane (131-I) and Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects

NCT04301843: Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

NCT04775485: Study to Evaluate DAY101 in Pediatric and Young Adult Patients with Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors

NCT04851119: Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors

NCT04870944: CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma

NCT05489887: Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

NCT06620302: Testing the Addition of an Anti-cancer Drug, DT2216, to the Usual Chemotherapy Treatment for Relapsed or Refractory Solid Tumors and Fibrolamellar Carcinoma

Sarcomas and other solid tumors

NCT04134559: Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma

NCT04322318: Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT

NCT04337177: Flavored, Oral Irinotecan VAL-413 (Orotecan®) Given With Temozolomide for Treatment of Recurrent Pediatric Solid Tumors

NCT04616560: Trastuzumab Deruxtecan for the Treatment of HER2+ Newly Diagnosed or Recurrent Osteosarcoma

NCT04634357: ET140203 T Cells in Pediatric Subjects With Hepatoblastoma, HCN-NOS, or Hepatocellular Carcinoma

NCT04775485: Study to Evaluate DAY101 in Pediatric and Young Adult Patients with Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors

NCT04851119: Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors

NCT04870944: CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma

NCT05783323: Larotrectinib to Enhance RAI Avidity in Differentiated Thyroid Cancer

NCT06064097: Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)

NCT06156410: Cabozantinib With Ifosfamide in Relapsed/Refractory Sarcoma

NCT06541262: Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid Tumors

NCT06620302: Testing the Addition of an Anti-cancer Drug, DT2216, to the Usual Chemotherapy Treatment for Relapsed or Refractory Solid Tumors and Fibrolamellar Carcinoma