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.