Clinical Trials

99 enrolled 3 FDA

Neurofibromatosis 1, Pain, Neurofibroma, Plexiform

NCT04924075 2026-02-24

MK-6482-015

Phase 2 Recruiting

322 enrolled 1 FDA

CNSEndocrine

von Hippel-Lindau Disease, Adenoma, Islet Cell, Paraganglioma, Pheochromocytoma

NCT03363373 2026-02-19

Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow

Phase 2 Recruiting

122 enrolled 2 FDA

Phase 2 Active not recruiting

Neuroblastoma

NCT04711135 2026-02-12

NETTER-P

11 enrolled 14 charts 1 FDA

CNSEndocrineGastrointestinalNET

Gastro-enteropancreatic neuroendocrine tumor, Paraganglioma, Pheochromocytoma

NCT04924608 2026-01-12

KOMET

Phase 3 Active not recruiting

145 enrolled 17 charts 1 FDA

Neurofibromatosis 1, Neurofibroma, Plexiform

NCT01757626 2026-01-07

Combination Therapy of Antibody Hu3F8 With Granulocyte- Macrophage Colony Stimulating Factor (GM-CSF) in Patients With Relapsed/Refractory High-Risk Neuroblastoma

186 enrolled 2 FDA

Neuroblastoma

NCT03899792 2026-01-06

LIBRETTO-121

36 enrolled 13 charts 2 FDA

BreastCNSEndocrineGastrointestinalHead & NeckSarcomaThoracic

Thyroid Cancer, Papillary, Breast Neoplasms, Bronchial Neoplasms, Carcinoma, Bronchogenic, Carcinoma, Non-Small-Cell Lung, Colonic Neoplasms, Endocrine System Diseases, Endocrine Gland Neoplasms, Head and Neck Neoplasms, Lung Diseases, Lung Neoplasms, Neoplasms, Neoplasms by Site, Respiratory Tract Diseases, Respiratory Tract Neoplasms, Sarcoma, Thoracic Neoplasms, Thyroid Diseases, Thyroid Neoplasms, Central Nervous System Neoplasms, Myofibromatosis, Carcinoma, Medullary

NCT02650401 2025-12-24

STARTRK-NG

CNSEndocrineGenitourinaryHead & NeckSarcoma

69 enrolled 1 FDA

Thyroid Cancer, Papillary, Brain Neoplasms, Glioma, Medulloblastoma, Neoplasm Metastasis, Wilms Tumor, Neuroblastoma, Recurrence, Salivary Gland Neoplasms, Sarcoma, Sarcoma, Ewing, Central Nervous System Neoplasms, Nephroma, Mesoblastic

NCT03157128 2025-10-20

LIBRETTO-001

BreastCNSEndocrineGastrointestinalHead & NeckThoracic

857 enrolled 3 FDA

Thyroid Cancer, Papillary, Breast Neoplasms, Bronchial Neoplasms, Carcinoma, Bronchogenic, Carcinoma, Non-Small-Cell Lung, Colonic Neoplasms, Endocrine System Diseases, Endocrine Gland Neoplasms, Head and Neck Neoplasms, Lung Diseases, Lung Neoplasms, Neoplasms, Neoplasms by Site, Respiratory Tract Diseases, Respiratory Tract Neoplasms, Thoracic Neoplasms, Thyroid Diseases, Thyroid Neoplasms, Central Nervous System Neoplasms, Carcinoma, Medullary

NCT03962543 2025-08-07

ReNeu

Phase 2 Active not recruiting

114 enrolled 25 charts 1 FDA

73 enrolled 13 charts 1 FDA

Neurofibroma, Neurofibromatosis 1, Neurofibromatoses, Neurofibroma, Plexiform

NCT03295396 2025-07-28

ONC201 in Adults With Recurrent H3 K27M-mutant Glioma

Phase 2 Terminated

Phase 3 Active not recruiting

Glioma

NCT04775485 2025-04-10

FIREFLY-1

Phase 2 Recruiting

141 enrolled 1 FDA

CNSOther solid neoplasm

Low-grade Glioma

NCT04164901 2025-04-03

Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

331 enrolled 25 charts 1 FDA

Glioma

NCT03416530 2025-02-14

ONC201 in Pediatric H3 K27M Gliomas

Phase 1 Terminated

134 enrolled 1 FDA

44 enrolled 22 charts 1 FDA

Diffuse Intrinsic Pontine Glioma, Glioma

NCT01121588 2025-01-23

Studying An Investigational Drug Crizotinib (PF-02341066) In Non Non-Small Cell Lung Cancer Tumors That Are Positive For Anaplastic Lymphoma Kinase (ALK)

Phase 1 Terminated

CNSLymphoid

Lymphoma, Neoplasms, Neuroblastoma

NCT02525692 2024-12-24

Oral ONC201 in Adult Recurrent Glioblastoma

Phase 2 Terminated

84 enrolled 14 charts 1 FDA

Glioblastoma

NCT02395666 2024-08-06

Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission

140 enrolled 14 charts 1 FDA

Neuroblastoma

NCT02684058 2023-12-13

Study of Efficacy and Safety of Dabrafenib in Combination With Trametinib in Pediatric Patients With BRAF V600 Mutation Positive LGG or Relapsed or Refractory HGG Tumors

151 enrolled 45 charts 1 FDA

Astrocytoma, Ganglioneuroma, Glioblastoma, Glioma, Oligodendroglioma, Ganglioglioma, Neurocytoma

NCT02034110 2023-08-21

Efficacy and Safety of the Combination Therapy of Dabrafenib and Trametinib in Subjects With BRAF V600E- Mutated Rare Cancers

206 enrolled 40 charts 2 FDA

CNSEndocrineGastrointestinalHead & NeckOther hematologic neoplasmPlasma cellSarcoma

Nonseminomatous germ cell tumor, Biliary Tract Neoplasms, Glioma, Leukemia, Hairy Cell, Multiple Myeloma, Neoplasms, Gastrointestinal Stromal Tumors, Thyroid Carcinoma, Anaplastic

NCT02614794 2023-08-14

HER2CLIMB