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. 2016 Jan 10;18(6):764–778. doi: 10.1093/neuonc/nov264

Table 2.

Phase I/II trials recruiting patients with AT/RT

Phase Title Status IND Target/Comment
I Simvastatin with Topotecan and Cyclophosphamide in Relapsed and/or Refractory Pediatric Solid and CNS Tumors (Aflac ST1402) Recruiting Simvastatin HMG CoA reductase
I p28 in Treating Younger Patients with Recurrent or Progressive Central Nervous System Tumors Recruiting p28 Azurin-derived cell-penetrating peptide p28
I/II Molecular-Guided Therapy for Childhood Cancer Recruiting n.a. Guided therapy
I/II Crizotinib in Treating Young Patients with Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma Recruiting Crizotinib ALK-, MET-, ROS1-tyrosine kinases
II Phase 2 Study of Alisertib Therapy for Rhabdoid Tumors Recruiting Alisertib Aurora kinase A
II Iodine I 131 Monoclonal Antibody 3F8 in Treating Patients with Central Nervous System Cancer or Leptomeningeal Cancer Recruiting 3F8-I131 Gd2
III Risk-Adapted Therapy for Young Children with Embryonal Brain Tumors, Choroid Plexus Carcinoma, High Grade Glioma or Ependymoma Recruiting n.a. Feasibility study
I Study of Safety and Efficacy in Patients with Malignant Rhabdoid Tumors (MRT) and Neuroblastoma Active, not recruiting Ribociclib Cyclin D1/CDK4, 6
I Aflac ST0901 CHOANOME Sirolimus in Solid Tumors Active, not recruiting Sirolimus mTOR
I AZD2171 in Treating Young Patients with Recurrent, Progressive, or Refractory Primary CNS Tumors Active, not recruiting Cediranib VEGFR 1–3
I/II Methotrexate Infusion into the Fourth Ventricle in Children with Malignant Fourth Ventricular Brain Tumors: A Pilot Study Active, not recruiting Methotrexate DHFR inhibition
I/II Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients with Metastatic or Recurrent Malignant Solid Tumors Active, not recruiting Dasatinib BCR/ABL and SRC-TK
I ABT-888 and Temozolomide in Treating Young Patients with Recurrent or Refractory CNS Tumors Completed Veliparib PARP inhibitor; DNA repair inhibition
I Vorinostat with or without Isotretinoin in Treating Young Patients with Recurrent or Refractory Solid Tumors, Lymphoma, or Leukemia Completed Vorinostat HDAC
I Vorinostat and Temozolomide in Treating Young Patients with Relapsed or Refractory Primary Brain Tumors or Spinal Cord Tumors Completed Vorinostat HDAC
I Lenalidomide in Treating Young Patients with Recurrent, Progressive, or Refractory CNS Tumors Completed Lenalidomide Immune modulation
I Talabostat Combined with Temozolomide or Carboplatin in Treating Young Patients with Relapsed or Refractory Brain Tumors or Other Solid Tumors Completed Talabostat Inhibitor of dipeptidyl peptidase IV
I SCH 66336 in Treating Children with Recurrent or Progressive Brain Tumors Completed Lonafarnib Farnesyl transferase
I Temozolomide, Vincristine, and Irinotecan in Treating Young Patients with Refractory Solid Tumors Completed Irinotecan Conventional chemotherapy
II Oxaliplatin in Treating Children with Recurrent or Refractory Medulloblastoma, Supratentorial Primitive Neuroectodermal Tumor, or Atypical Teratoid Rhabdoid Tumor Completed Oxaliplatin Cytostatic
I Gamma-Secretase Inhibitor RO4929097 in Treating Young Patients with Relapsed or Refractory Solid Tumors, CNS Tumors, Lymphoma, or T-Cell Leukemia Terminated RO4929097 γ-Secretase
I MK0752 in Treating Young Patients with Recurrent or Refractory CNS Cancer Terminated MK0752 γ-Secretase inhibitor, which reduces Aβ40 production
Trials involving combinations of conventional chemotherapy (incl. HDCT)
III Combination Chemotherapy, Radiation Therapy, and an Autologous Peripheral Blood Stem Cell Transplant in Treating Young Patients with Atypical Teratoid/Rhabdoid Tumor of the Central Nervous System Ongoing, not recruiting NCT00653068
III Treatment of Patients with Newly Diagnosed Medulloblastoma, Supratentorial Primitive Neuroectodermal Tumor, or Atypical Teratoid Rhabdoid Tumor Ongoing, not recruiting NCT00085202
I Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Infants with Malignant Brain or Spinal Cord Tumors Completed NCT00003141
I Chemotherapy and Stem Cell Transplantation in Treating Children with Central Nervous System Cancer Completed NCT00053118
III Combination Chemotherapy Followed by Second-Look Surgery and Radiation Therapy in Treating Children with Nonmetastatic Medulloblastoma or Primitive Neuroectodermal Tumor Completed NCT00006461
III Combination Chemotherapy with or without Etoposide Followed by an Autologous Stem Cell Transplant in Treating Young Patients with Previously Untreated Malignant Brain Tumors Unknown Head Start III NCT00392886
III Intrathecal and Systemic Chemotherapy Combined with Radiation Therapy in Treating Young Patients with Newly Diagnosed Central Nervous System Atypical Teratoid/Rhabdoid Tumors Unknown NCT00084838
Chi et al, JCO 2009

Abbreviations: IND, investigational new drug; HMG CoA, 3-hydroxy-3-methylglutaryl coenzyme A; ALK, anaplastic lymphoma kinase; VEGFR, vascular endothelial growth factor receptor; DHFR, dihydrofolate reductase; PARP, poly(ADP-ribose) polymerase; DNA, desoxyribonucleic acid; n.a., depending on the identified target; BCR/ABL and SRC-TK, BCR/ABL and SRC tyrosine kinases.