University of California San Francisco, Benioff Children's Hospital
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 22 Trials 
27 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Bankiewicz, Krystof
NCT02852213: A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients

Recruiting
1
42
US
AAV2-hAADC, Adeno Virus Human Aromatic L-Amino Acid Decarboxylase
Krzysztof Bankiewicz, National Institute of Neurological Disorders and Stroke (NINDS), University of California, San Francisco
AADC Deficiency
07/27
07/31
Gupta, Nalin
NCT02852213: A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients

Recruiting
1
42
US
AAV2-hAADC, Adeno Virus Human Aromatic L-Amino Acid Decarboxylase
Krzysztof Bankiewicz, National Institute of Neurological Disorders and Stroke (NINDS), University of California, San Francisco
AADC Deficiency
07/27
07/31
Mueller, Sabine
NCT01734512: PNOC 001: Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children

Completed
2
65
US
Everolimus
University of California, San Francisco, Novartis Pharmaceuticals, Pacific Pediatric Neuro-Oncology Consortium, The Pediatric Low Grade Astrocytoma (PLGA) Foundation
Pediatric Recurrent Progressive Low-grade Gliomas, Pediatric Progressive Low-grade Gliomas
01/20
07/24
NCT05123534: A Phase 2 Study of Sonodynamic Therapy Using SONALA-001 and Exablate 4000 Type 2.0 in Patients with DIPG

Suspended
2
15
US
SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS), Exablate Type 2.0
SonALAsense, Inc.
Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma
07/24
10/24
NCT05009992: Combination Therapy for the Treatment of Diffuse Midline Gliomas

Hourglass Jun 2024 - Jul 2024 : Follow up data for DMG
Recruiting
2
360
Europe, US, RoW
ONC201, TIC10, Antagonist of dopamine receptor D2 (DRD2) and caseinolytic protease proteolytic subunit (ClpP), Radiation Therapy, Radiotherapy, Paxalisib, Inhibitor of the PI3K/AKT/mTOR pathway, GDC-0084, GDC0084, PI3K Inhibitor GDC-0084
University of California, San Francisco, The Chad-Tough Defeat DIPG Foundation, Mithil Prasad Foundation, Storm the Heavens Fund, National Institute of Neurological Disorders and Stroke (NINDS)
Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Recurrent Diffuse Intrinsic Pontine Glioma, Recurrent Diffuse Midline Glioma, H3 K27M-Mutant, Recurrent WHO Grade III Glioma, WHO Grade III Glioma
12/25
06/29
PNOC029, NCT05465174: Nivolumab and Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults

Recruiting
2
56
US
Nivolumab, Opdivo, Tovorafenib, DAY101, TAK580, AMG-2112819, BSK1369
Sabine Mueller, MD, PhD, Bristol-Myers Squibb, Day One Biopharmaceuticals, Inc.
Craniopharyngioma, Child, Craniopharyngioma, Recurrent Craniopharyngioma
03/27
03/28
NCT03690869 / 2023-000604-19: REGN2810 in Pediatric Patients With Relapsed, Refractory Solid, or Central Nervous System (CNS) Tumors and Safety and Efficacy of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed or Recurrent Glioma

Calendar Jan 2025 - Dec 2025: From trial n pediatrics with solid tumors
Terminated
1/2
57
US
cemiplimab (monotherapy), REGN2810, Libtayo, cemiplimab (maintenance), Conventional or hypofractionated, Re-irradiation
Regeneron Pharmaceuticals, Pacific Pediatric Neuro-Oncology Consortium
Relapsed Solid Tumor, Refractory Solid Tumor, Relapsed Central Nervous System Tumor, Refractory Central Nervous System Tumor, Diffuse Intrinsic Pontine Glioma, High Grade Glioma
05/23
05/23
CA209-8TX, NCT02960230: H3.3K27M Peptide Vaccine With Nivolumab for Children With Newly Diagnosed DIPG and Other Gliomas

Completed
1/2
50
Europe, US
K27M peptide, Nivolumab
Sabine Mueller, MD, PhD, The V Foundation for Cancer Research, Pacific Pediatric Neuro-Oncology Consortium, Bristol-Myers Squibb
Diffuse Intrinsic Pontine Glioma, Glioma, Diffuse Midline Glioma, H3 K27M-Mutant
12/23
12/23
BLU-285-3101, NCT04773782 / 2020-005234-15: A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling

Active, not recruiting
1/2
37
Europe, Canada, US, RoW
avapritinib, BLU-285
Blueprint Medicines Corporation
Solid Tumor, Unspecified, Child, Relapsed Solid Neoplasm, CNS Tumor
02/25
12/25
NCT01748149: Vemurafenib in Children With Recurrent/Refractory BRAF Gene V600E (BRAFV600E)-Mutant Gliomas

Active, not recruiting
1
40
Canada, US
Vemurafenib, RO5185426, PLX4032
University of California, San Francisco, Genentech, Inc.
Pediatric Recurrent/Refractory BRAFV600E-mutant Gliomas
12/18
12/25
NCT01922076: Adavosertib and Local Radiation Therapy in Treating Children With Newly Diagnosed Diffuse Intrinsic Pontine Gliomas

Completed
1
46
Canada, US
Adavosertib, AZD-1775, AZD1775, MK-1775, MK1775, Laboratory Biomarker Analysis, Pharmacological Study, Radiation Therapy, Cancer Radiotherapy, Energy Type, ENERGY_TYPE, Irradiate, Irradiated, Irradiation, Radiation, Radiation Therapy, NOS, Radiotherapeutics, Radiotherapy, RT, Therapy, Radiation
National Cancer Institute (NCI)
Anaplastic Astrocytoma, Anaplastic Oligoastrocytoma, Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Glioblastoma, Gliosarcoma
12/20
09/22
PNOC016, NCT03893487: Fimepinostat in Treating Brain Tumors in Children and Young Adults

Active, not recruiting
1
30
Europe, US
Fimepinostat, CUDC-907, Therapeutic Conventional Surgery
Sabine Mueller, MD, PhD, Pediatric Neuro-Oncology Consortium, Cannonball Kids' Cancer Foundation, Curis, Inc.
Diffuse Intrinsic Pontine Glioma, Recurrent Anaplastic Astrocytoma, Recurrent Glioblastoma, Recurrent Malignant Glioma, Recurrent Medulloblastoma
10/22
08/27
NCT02962167: Modified Measles Virus (MV-NIS) for Children and Young Adults With Recurrent Medulloblastoma or Recurrent ATRT

Completed
1
34
US
Modified Measles Virus, MV-NIS, Modified Measles Virus Lumbar Puncture, MV-NIS LP
Sabine Mueller, MD, PhD, No More Kids With Cancer, The Matthew Larson Foundation for Pediatric Brain Tumors, Vyriad, Inc., Mayo Clinic
Medulloblastoma, Childhood, Recurrent, Atypical Teratoid/Rhabdoid Tumor, Medulloblastoma Recurrent
05/23
05/23
NCT04323046: Immunotherapy Before and After Surgery for Treatment of Recurrent or Progressive High Grade Glioma in Children and Young Adults

Recruiting
1
20
Europe, US, RoW
Nivolumab, BMS-936558, MDX-1106, NIVO, ONO-4538, Opdivo, Quality-of-Life Assessment, Quality of Life Assessment, Questionnaire Administration
Sabine Mueller, MD, PhD, Pediatric Neuro-Oncology Consortium
Glioblastoma, Malignant Glioma, Recurrent Glioblastoma, Recurrent Malignant Glioma, Recurrent Grade III Glioma, Grade III Glioma
03/26
03/29
PNOC017, NCT03749187: BGB-290 and Temozolomide in Treating Isocitrate Dehydrogenase (IDH)1/2-Mutant Grade I-IV Gliomas

Recruiting
1
78
US
PARP Inhibitor BGB-290, BGB-290, Temozolomide, CCRG-81045, Imidazo[5,1-d]-1,2,3,5-tetrazine-8-carboxamide, 3, 4-dihydro-3-methyl-4-oxo-, M & B 39831, M and B 39831, Methazolastone, RP-46161, SCH 52365, Temcad, Temodal, Temodar, Temomedac
University of California, San Francisco, BeiGene USA, Inc.
Glioblastoma, IDH1 Gene Mutation, IDH2 Gene Mutation, Low Grade Glioma, Malignant Glioma, Recurrent Glioblastoma, Recurrent WHO Grade II Glioma, Recurrent WHO Grade III Glioma, WHO Grade II Glioma, WHO Grade III Glioma
06/26
07/29
PNOC025, NCT05169944: Magrolimab in Children and Adults With Recurrent or Progressive Malignant Brain Tumors

Completed
1
13
US
Magrolimab, Anti-CD47 Monoclonal Antibody
University of California, San Francisco, Gilead Sciences
Brain Cancer, Malignant Brain Tumor, Recurrent Brain Tumor, Progressive Malignant Brain Tumor, Brain Tumor, Pediatric, Brain Tumor Adult
09/24
09/24
NCI-2021-00046, NCT04732065: ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors

Recruiting
1
208
Europe, US
ONC206, antagonist of dopamine receptor D2 (DRD2) /human mitochondrial caseinolytic protease P (ClpP)ClpP Agonist, DRD2 antagonist/ClpP agonist, Standard of Care Radiation Therapy, Radiation Therapy (RT), Cancer Radiotherapy, Optional Proton (1H) MR spectroscopy (MRS), MRS
Sabine Mueller, MD, PhD, Chimerix, Mithil Prasad Foundation, Storm the Heavens Fund, The ChadTough Defeat DIPG Foundation, National Cancer Institute (NCI), Dana-Farber Cancer Institute
Diffuse Midline Glioma (DMG), Glioblastoma, Recurrent Ependymoma, Recurrent Malignant Central Nervous System Neoplasm, Spinal Cord Glioma, World Health Organization (WHO) Grade III Glioma, CNS Tumor, Central Nervous System Tumor
05/27
07/27
PNOC028, NCT05887882: Intra-Tumoral Injections of Natural Killer Cells for Recurrent Malignant Pediatric Brain Tumors

Recruiting
1
24
US
Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, UD TGFβi NK cells, UD TGFβi NK cell product, Implantation, Magnetic Resonance Imaging (MRI), MRI, Quality-of-Life Assessment, QOL Assessment
Sabine Mueller, MD, PhD, Rally Foundation, Washington University School of Medicine, Nationwide Children's Hospital, CureSearch, Tommy Strong Foundation
Pediatric Brain Tumor, Recurrent Pediatric Brain Tumor, Pediatric Supratentorial Neoplasm
12/27
12/27
NCT04485559: Trametinib and Everolimus for Treatment of Pediatric and Young Adult Patients With Recurrent Gliomas (PNOC021)

Recruiting
1
50
US
Everolimus, 42-O-(2-Hydroxy)ethyl Rapamycin, Afinitor, Certican, RAD 001, RAD001, Votubia, Zortress, Trametinib, GSK1120212, JTP-74057, MEK Inhibitor GSK1120212, Mekinist
University of California, San Francisco, Novartis Pharmaceuticals, Pediatric Brain Tumor Foundation, The Lilabean Foundation for Pediatric Brain Cancer Research
Recurrent World Health Organization (WHO) Grade II Glioma, Low-grade Glioma, High Grade Glioma
12/27
12/27
PNOC018, NCT05478837: Genetically Modified Cells (KIND T Cells) for the Treatment of HLA-A*0201-Positive Patients With H3.3K27M-Mutated Glioma

Recruiting
1
12
US
Cyclophosphamide, Cytoxan, Cycloblastin, Fludarabine, Fludara, Autologous Anti-H3.3K27M TCR-expressing T-cells, KIND T cells
University of California, San Francisco, The V Foundation, Parker Institute for Cancer Immunotherapy, Alliance for Cancer Gene Therapy
Diffuse Midline Glioma, H3 K27M-Mutant
08/29
08/29
PNOC008, NCT03739372: Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan for Patients With High Grade Glioma

Active, not recruiting
N/A
44
US
Specialized tumor board recommendation
University of California, San Francisco, Pacific Pediatric Neuro-Oncology Consortium, The V Foundation for Cancer Research
Glioma, Glioma of Brain, Cancer, Pediatric Cancer, Pediatric Brain Tumor, Astrocytoma, Glioblastoma, Hemispheric
12/23
01/27
NCT05000905: Improving Cognition and Behavior in Pediatric Cancer Survivors Using a Novel Mindful Attention Training

Recruiting
N/A
70
US
Adaptive Attention Training, Questionnaires
University of California, San Francisco
Pediatric Cancer
05/25
05/25
PNOC027, NCT05057702: Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma

Recruiting
N/A
10
US
Specialized Tumor Board Treatment Plan, Individualized Treatment Plan, Specialized Treatment Plan
University of California, San Francisco, Washington University School of Medicine, St. Baldrick's Foundation, Ashion Analytics, University of Washington, Pediatric Neuro-Oncology Consortium
Medulloblastoma, Medulloblastoma, Childhood, Medulloblastoma Recurrent
12/25
01/28
NCT06000787: MCT for the Harvard/UCSF ROBIN Center

Recruiting
N/A
47
US
External beam radiotherapy, 131I-Metaiodobenzylguanidine (MIBG)
Brigham and Women's Hospital, Dana-Farber Cancer Institute, National Cancer Institute (NCI), University of California, San Francisco
Glioma, Childhood Brainstem, Neuroblastoma
08/28
08/28
Mahuvakar, Shivani
NCT02852213: A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients

Recruiting
1
42
US
AAV2-hAADC, Adeno Virus Human Aromatic L-Amino Acid Decarboxylase
Krzysztof Bankiewicz, National Institute of Neurological Disorders and Stroke (NINDS), University of California, San Francisco
AADC Deficiency
07/27
07/31
STEALTH, NCT05432986: Assessment of CSF Shunt Flow With Thermal Measurements B

Completed
N/A
141
US
Thermal Anisotropy Measurement Device, FlowSense
Rhaeos, Inc.
Hydrocephalus
11/23
11/23
Pen, Catalina
NCT02852213: A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients

Recruiting
1
42
US
AAV2-hAADC, Adeno Virus Human Aromatic L-Amino Acid Decarboxylase
Krzysztof Bankiewicz, National Institute of Neurological Disorders and Stroke (NINDS), University of California, San Francisco
AADC Deficiency
07/27
07/31

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