The UCSF Children’s Cancer Program offers a wide range of clinical trials for children and young adults with cancer in the San Francisco Bay Area, Northern California, and western United States. We are a designated Children's Oncology Group (COG) Phase 1 center. We also have studies from New Approaches to Neuroblastoma Therapy (NANT), Pacific Pediatric Neuro-Oncology Consortium (PNOC), Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL), Sarcoma Alliance for Research Through Collaboration (SARC), pharmaceutical industry, and university institutions. Many of our trials are available for patients up to 30 years of age.

For patients with newly diagnosed cancer, most clinical trials are offered through our participation in the national Children’s Oncology Group. A list of current open trials appropriate for newly diagnosed patients can be found here.

For patients with relapsed or refractory cancer, we strive to provide patients access to the newest treatment options in the form of phase 1 and phase 2 clinical trials. Our current open phase 1 and 2 clinical trials are listed below, according to disease category.

If you are interested in learning more about any of these trials, please contact us at 415-476-3831 or cancerclinicaltrials@peds.ucsf.edu.

ADVL1312: A Phase I/II Study of MK-1775 in Combination with Oral Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumors

Principal Investigator: Katherine Matthay, MD

 

ADVL1414: A Phase 1 Study of Selinexor (KPT-330, IND #125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, including CNS Tumors

Principal Investigator: Katherine Matthay, MD

 

ADVL1416: A Phase 1 Study of Ramucirumab, a Human Monoclonal Antibody Against the Vascular Endothelial Growth Factor-2 (VEGFR-2) Receptor in Children With Refractory Solid Tumors, Including CNS Tumors

Principal Investigator: Katherine Matthay, MD

 

ACNS1022: A Phase II Randomized Trial of Lenalidomide in Pediatric Patients with Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytoma and Optic Pathway Gliomas

Principal Investigator: Anuradha Banerjee, MD, MPH

 

ACNS1123: Phase 2 Trial of Response-Based Radiation Therapy for Patients with Localized Central Nervous System Germ Cell Tumors (CNS GCT)

Principal Investigator: Anuradha Banerjee, MD, MPH

 

ACNS1221: A Phase II Study For The Treatment Of Non-Metastatic Nodular Desmoplastic Medulloblastoma In Children Less Than 4 Years Of Age

Principal Investigator: Sabine Mueller, MD, PhD

 

GSK Trametinib: An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects with Cancer or Plexiform Neurofibromas and Trametinib in Combination with Dabrafenib in Children and Adolescents with Cancers Harboring V600 mutation

Principal Investigator: Sabine Mueller, MD, PhD

 

PBTC-029: A Phase I and Pharmacokinetic Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma

Principal Investigator: Anuradha Banerjee, MD

 

PNOC-001: Phase II Study of everolimus for recurrent or progressive low-grade gliomas in children

Principal Investigator: Sabine Mueller, MD, PhD

 

PNOC-002: Safety, Phase 0, and Pilot Efficacy Study of vemurafenib, an oral inhibitor of BRAFV600E, in Children with Recurrent/Refractory BRAFV600E-mutant gliomas

Principal Investigator: Theodore Nicolaides, MD

 

PNOC-003: A Pilot Trial Testing the Clinical Benefits of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children and Young Adults with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)

Principal Investigator: Sabine Mueller, MD, PhD

 

EZH102: A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects with Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

Principal Investigator: Kieuhoa Vo, MD, MAS

 

Ignyta Entrectinib: A Phase 1/1b. Open-Label, Dose-Escalation and Expannsion Study of Entrctinib in Children and Adolescents with Recurrent/Refactory Solid Tumors and Primary CNS Tumor

Principal Investigator: Amit Sabnis, MD

 

ADVL1212: A Phase I Study of Crizotinib in Combination with Conventional Chemotherapy for Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma (ALCL)

Principal Investigator: Katherine Matthay, MD

 

ADVL1314: A Phase I Study of Eribulin Mesylate (E7389, Ind #116,292), a Novel Microtubule Targeting Chemotherapeutic Agent in Children with Refractory or Recurrent Solid Tumors (excluding CNS), including Lymphomas

Principal Investigator: Katherine Matthay, MD

 

ANHL12P1: A Randomized Phase II Trial of Brentuximab Vedotin (SGN35, NSC# 749710), or Crizotinib
(NSC#749005, commercially labeled) in Combination with Chemotherapy for Newly Diagnosed Patients with Anaplastic Large Cell Lymphoma (ALCL) IND # 117117

Principal Investigator: Michelle Hermiston, MD, PhD

 

DFCI RAD001: A Feasibility Trial of Everolimus (RAD001), an mTOR Inhibitor, Given in Combination with Multiagent Reinduction Chemotherapy in Pediatric Patients with Relapsed Acute Lymphoblastic Leukemia

Principal Investigator: Mignon Loh, MD

 

DFCI #13-563: Phase I trial of the selective inhibitor of nuclear export, KPT-330, in relapsed childhood ALL and AML

Principal Investigator: Elliot Stieglitz, MD

 

TACL 2014-001: A Phase 1 Trial of Temsirolimus (CCI-779, Pfizer, Inc.) in Combination with Etoposide and Cyclophosophamide in Children with Relapsed Acute Lymphoblastic Leukemia and Non-Hodgkins Lymphoma

Principal Investigator: Michelle Hermiston, MD, PhD

 

 

ADVL1212: A Phase I Study of Crizotinib in Combination with Conventional Chemotherapy for Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma (ALCL)

Principal Investigator: Katherine Matthay, MD

 

ADVL1314: A Phase I Study of Eribulin Mesylate, a Novel Microtubule Targeting Chemotherapeutic Agent in Children with Refractory Or Recurrent Solid Tumors (excluding CNS), including Lymphomas

Principal Investigator: Katherine Matthay, MD

 

ADVL1315: A Phase I Study of the VEGF Receptor Tyrosine Kinase Inhibitor Axitinib  in Children with Recurrent or Refractory Solid Tumors

Principal Investigator: Katherine Matthay, MD

 

ADVL1412: A Phase 1/2 Study of Nivolumab in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with Ipilimumab

Principal Investigator: Katherine Matthay, MD

 

ADVL1522: A Phase 2 Study of IMGN901 (Lorvotuzumab Mertansine; IND 126953,NSC 783609) in Children with Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Periperal Nerve Sheath Tumor (MPNST) and Synovial Sarcoma

Principal Investigator: Katherine Matthay, MD

 

Compassionate Use MIBG Therapy: 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Refractory Neuroblastoma: A Compassionate Use Protocol

Principal Investigator: Katherine Matthay, MD

 

Compassionate Use Pheo Therapy: 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Patients with Malignant Pheochromocytoma & Related Tumors: A Compassionate Use Protocol

Principal Investigator: Paul Fitzgerald, MD

 

GSK Trametinib: An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects with Cancer or Plexiform Neurofibromas and Trametinib in Combination with Dabrafenib in Children and Adolescents with Cancers Harboring V600 mutation

Principal Investigator: Sabine Mueller, MD, PhD

 

NANT 2011-01: A Randomized Phase II study of 131I-MIBG VS. 131I-MIBG with Vincristine and Irinotecan vs 131I-MIBG with Vorinostat for Resistant/Relapsed Neuroblastoma

Principal Investigator: Katherine Matthay, MD

 

NANT 2011-04: A Phase I Study of Lenalidomide and ch14.18 With or Without Isotretinoin in Treating Younger Patients With Refractory or Recurrent Neuroblastoma

Principal Investigator: Katherine Matthay, MD

 

NANT 2012-01: Phase I Study of Difluoromethylornithine (DFMO) and Celecoxib Cyclophosphamide/Topotecan for Patients with Relapsed or Refractory neuroblastoma

Principal Investigator: Katherine Matthay, MD

 

NANT 2013-02: A Phase I Study of Sorafenib and Cyclophosphamide/Topotecan in Patients with Relapsed and Refractory Neuroblastoma

Principal Investigator: Katherine Matthay, MD

 

NANT 2014-01: Phase I Study Of SF1126 for Patients with Relapsed or Refractory Neuroblastoma

Principal Investigator: Katherine Matthay, MD

 

Ignyta Entrectinib: A Phase 1/1b. Open-Label, Dose-Escalation and Expannsion Study of Entrctinib in Children and Adolescents with Recurrent/Refactory Solid Tumors and Primary CNS Tumor

Principal Investigator: Amit Sabnis, MD

 

ADVL1212: A Phase I Study of Crizotinib in Combination with Conventional Chemotherapy for Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma (ALCL)

Principal Investigator: Katherine Matthay, MD

 

ADVL1312: A Phase I/II Study of MK-1775 in Combination with Oral Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumors

Principal Investigator: Katherine Matthay, MD

 

ADVL1314: A Phase I Study of Eribulin Mesylate (E7389, Ind #116,292), a Novel Microtubule Targeting Chemotherapeutic Agent in Children with Refractory or Recurrent Solid Tumors (excluding CNS), including Lymphomas

Principal Investigator: Katherine Matthay, MD

 

ADVL1315: A Phase I Study of the VEGF Receptor Tyrosine Kinase Inhibitor Axitinib  in Children with Recurrent or Refractory Solid Tumors

Principal Investigator: Katherine Matthay, MD

 

ADVL1322: A Phase II Study of Pazopanib in Children, Adolescents and Young Adults with Refractory Solid Tumors

Principal Investigator: Amit Sabnis, MD, MS

 

ADVL1412: A Phase 1/2 Study of Nivolumab in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with Ipilimumab

Principal Investigator: Katherine Matthay, MD

 

ADVL1414: A Phase 1 Study of Selinexor (KPT-330, IND #125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, including CNS Tumors

Principal Investigator: Katherine Matthay, MD

 

ADVL1416: A Phase 1 Study of Ramucirumab, a Human Monoclonal Antibody Against the Vascular Endothelial Growth Factor-2 (VEGFR-2) Receptor in Children With Refractory Solid Tumors, Including CNS Tumors

Principal Investigator: Katherine Matthay, MD

 

ADVL1522: A Phase 2 Study of IMGN901 (Lorvotuzumab Mertansine; IND 126953,NSC 783609) in Children with Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Periperal Nerve Sheath Tumor (MPNST) and Synovial Sarcoma

Principal Investigator: Katherine Matthay, MD

 

ARET12P1: A Multi-Institutional Feasibililty Study of Intra-Arterial Chemotherapy Given in the Ophthalmic Artery of Children with Retinoblastoma

Principal Investigator: Katherine Matthay, MD

 

AOST1321: A Phase 2 Study of Denosumab (IND#127430, NSC#744010), a RANK Ligand Antibody, for Recurrent or Refractory Osteosarcoma

Principal Investigator: Robert Goldsby, MD

 

AOST 1521: A Phase 2 Study of GPNMB-targeted Antibody-Drug Conjugate, CDX-011 (Glembatumumab Vedotin, CR011-vcMMAE; IND#128248, NSC#763737), in Recurrent or Refractory Osteosarcoma

Principal Investigator: Robert Goldsby, MD

 

GSK Trametinib: An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects with Cancer or Plexiform Neurofibromas and Trametinib in Combination with Dabrafenib in Children and Adolescents with Cancers Harboring V600 mutation

Principal Investigator: Sabine Mueller, MD, PhD

 

IA Melphalan and Chemo: A Pilot Study Evaluating the Safety of Alternating Systemic Chemotherapy and Intra-Arterial Melphalan Chemotherapy in Children With Newly Diagnosed Advanced Intra-Ocular Retinoblastoma

Principal Investigator: Anuradha Banerjee, MD, MPH

 

Ignyta Entrectinib: A Phase 1/1b. Open-Label, Dose-Escalation and Expannsion Study of Entrctinib in Children and Adolescents with Recurrent/Refactory Solid Tumors and Primary CNS Tumor

Principal Investigator: Amit Sabnis, MD

 

AEWS1221: Randomized Phase II Trial Evaluating the Addition of the IGF-1R Monoclonal Antibody Ganitumab (AMG479, NSC 750008, IND 120449) to Multiagent Chemotherapy for Patients with Newly Diagnosed Metastatic Ewing Sarcoma

Principal Investigator: Robert Goldsby, MD

 

EZH102: A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects with Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

Principal Investigator: Kieuhoa Vo, MD