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.

ADVL1217: A Phase I Study of MK-1775 Concurrent with Local Radiation Therapy for the Treatment of Newly Diagnosed Children with Diffuse Intrinsic Pontine Gliomas

Principal Investigator: Sabine Mueller, MD, PhD

 

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

 

ADVL1411: A Phase I/II Study of BMN 673, an Oral Poly(ADP-ribose) Polymerase Inhibitor, Plus Temozolomide in Children with Refractory or Recurrent Malignancies

Principal Investigator: Katherine Matthay, MD

 

ACNS0821: Temozolomide with Irinotecan Versus Temozolomide, Irinotecan plus Bevacizumab for Recurrent/Refractory Medulloblastoma/CNS PNET of Childhood, A COG Randomized Phase II Screening Trial

Principal Investigator: Anuradha Banerjee, MD, MPH

 

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

 

DFCI #10-321: Phase II Trial of Molecularly Determined Treatment of Children and Young Adults with Newly Diagnosed Diffuse Intrinsic Pontine Gliomas

Principal Investigator: Anuradha Banerjee, MD, MPH

 

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 Feasibility of Using Molecular Profiling to Guide an Individualized Treatment Plan in Children and Young Adults with Newly Diagnosed DIPG

Principal Investigator: Sabine Mueller, 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 (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

 

AHOD1221: A Phase I/II Study of Brentuximab Vedotin in Combination with Gemcitabine for Pediatric and Young Adult Patients with Relapsed or Refractory Hodgkin Lymphoma

Principal Investigator: Michelle Hermiston, MD, PhD

 

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

 

ASCT1221: A Randomized Phase II Study Comparing Two Different Conditioning Regimens Prior to Allogeneic Hematopoietic Cell Transplantation (HCT) for Children with Juvenile Myelomonocytic Leukemia (JMML)

Principal Investigator: Christopher Dvorak, 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

 

Epizyme EPZ-5676: A Phase I Dose Escalation and Expanded Cohort Study of EPZ-5676 in the Treatment of Pediatric Patients with Relapsed/Refractory Leukemias Bearing a Rearrangement of the MLL Gene

Principal Investigator: Mignon Loh, MD

 

TACL 2008-002: A Phase I Trial of NECTAR Nelarabine, Etoposide and Cyclophosphamide in T-ALL Relapse)

Principal Investigator: Michelle Hermiston, MD, PhD

 

TACL 2009-003: A Pilot Study of Decitabine and Vorinostat with Chemotherapy for Relapsed ALL

Principal Investigator: Michelle Hermiston, MD, PhD

 

TACL 2009-012: A Phase I Dose Finding Study of Panobinostat In Children with Refractory Hematologic Malignancies

Principal Investigator: Michelle Hermiston, MD, PhD

 

ADVL0912: A Phase I/II Study of PF-02341066 (Crizotinib), an Oral Small Molecule Inhibitor of Anaplastic Lymphoma Kinase (ALK) and C-Met, in Children with Relapsed/Refractory Solid Tumors and Anaplastic Large Cell Lymphoma

Principal Investigator: Katherine Matthay, 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, 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

 

ADVL1411: A Phase I/II Study of BMN 673, an Oral Poly(ADP-ribose) Polymerase Inhibitor, Plus Temozolomide in Children with Refractory or Recurrent Malignancies

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

 

ANBL09P1: A COG Pilot Study of Intensive Induction Chemotherapy and 131I-MIBG Followed by Myeloablative Busulfan/Melphalan (Bu/Mel) for Newly Diagnosed High-Risk Neuroblastoma

Principal Investigator: Katherine Matthay, MD

 

ANBL1221: A Phase II Randomized Trial of Irinotecan/Temozolomide with Temsirolimus (NSC# 683864, IND# 61010) or Chimeric 14.18 Antibody (ch14.18) (NSC# 623408, IND# 4308) in Children with Refractory, Relapsed or Progressive Neuroblastoma

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

 

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

Principal Investigator: Katherine Matthay, 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 (INLYTA, IND# 123101) 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

 

ADVL1411: A Phase I/II Study of BMN 673, an Oral Poly(ADP-ribose) Polymerase Inhibitor, Plus Temozolomide in Children with Refractory or Recurrent Malignancies

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

 

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

Principal Investigator: Katherine Matthay, 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 Feasibility Study of Combination Intra-Arterial Melphalan Chemotherapy and Systemic Chemotherapy for the Treatment of Advanced Intra-Ocular Retinoblastoma

Principal Investigator: Katherine Matthay, MD