All Recurrent/Progressive Tumors

• PBTC031
  Phase I and Pharmacokinetics Study of PTC299 in Pediatric Patients with Refractory and Recurrent CNS Tumors
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  Title Phase I and Pharmacokinetics Study of PTC299 in Pediatric Patients with Refractory and Recurrent CNS Tumors Tumor Type any recurrent brain tumor Description This is a Phase I study of PTC299 in children with recurrent or refractory brain tumors. PTC299 is a new oral anti-angiogenesis medicine, which targets vascular endothelial growth factor (VEGF) to prevent tumor growth and metastasis, and by interrupting cell division. Antiangiogenesis has been shown to be an effective strategy at controlling tumor growth in many cancers. Major Eligbility Age: >3 years and < 21 years old Must be able to swallow capsules and have recovered from previous therapy Clinicaltrials.gov ID NCT01158300

• ADVL1111 A Phase I study of the c-met inhibitor, tivantinib, in children with relapsed or refractory solid tumors More Details
  
  

  Title A Phase I study of the c-met inhibitor, tivantinib, in children with relapsed or refractory solid tumors Tumor Type all relapsed/recurrent CNS tumors Description clinical and adult clinical studies using this strategy. This trial is a Phase I trial looking to determine the optimum dose in children. Medication is oral (capsules) and given daily. Major Eligbility Age: >12months to <21yrs Must have measurable disease. Exclusion: any known arrhythmia

• ADVL1013
  A Phase 1 Study of MK-2206, an AKT inhibitor, in Pediatric Patients with Recurrent or Refractory Solid Tumors or Leukemia
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  Title A Phase 1 Study of MK-2206, an AKT inhibitor, in Pediatric Patients with Recurrent or Refractory Solid Tumors or Leukemia Tumor Type Recurrent/refractory solid tumors (including CNS tumors) or leukemia Description MK-2206 is an allosteric AKT inhibitor and will be taken orally every other day (schedule 1) or once weekly (schedule 2). Major Eligbility > 12 months and < 21 years of age Adequate bone marrow, kidney, liver, neurologic Patients receiving insulin or growth hormone therapy or known type I or II diabetes mellitus are not eligible Patients must be able to swallow whole tablets  Clinicaltrials.gov ID NCT01231919

Medulloblastoma – see also ‘All Recurrent/Progressive Tumors’

• PBTC036
  A Molecular Biology and Phase II Study of Imetelstat (GRN163L) in Children with Recurrent High-Grade Glioma, Ependymoma, Medulloblastoma/Primitive Neuroectodermal Tumor and Diffuse Intrinsic Pontine Glioma
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  Title A Molecular Biology and Phase II Study of Imetelstat (GRN163L) in Children with Recurrent High-Grade Glioma, Ependymoma, Medulloblastoma/Primitive Neuroectodermal Tumor and Diffuse Intrinsic Pontine Glioma Tumor Type recurrent ependymoma recurrent medulloblastoma recurrent DIPG recurrent high-grade glioma Description This Phase 2 and biology study seeks to evaluate the efficacy of imetelstat in a variety of aggressive pediatric brain tumors. Imetelstat attacks an enzyme called telomerase, which is present in a very high proportion of pediatric brain tumors and helps to immortalize brain tumor cells. Imetelstat prevents telomerase from working well. It is hoped that by preventing this function, tumor cells will be unable to propagate themselves indefinitely. Major Eligbility Age: >12months to <21yrs Tumor: recurrent/refractory medulloblastoma, ependymoma, high-grade glioma, or diffuse intrinsic pontine glioma (DIPG) Performance status: >50% Exclusion: no known coagulopathy or use of systemic anticoagulant medications, for the biology portion of the study, patients need to have frozen tumor tissue available. For the Phase 2 portion of the study, patients need to have measurable disease

• PBTC025B
  A Phase II Clinical Trial Evaluating the Efficacy and Safety
  of GDC-0449 in Adults with Recurrent or Refractory Medulloblastoma.
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  Title A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Adults with Recurrent or Refractory Medulloblastoma. Tumor Type recurrent medulloblastoma Description This is a Phase II study of GDC-0449, which is an oral drug that interferes with signaling of the sonic hedgehog pathway. This pathway is known to drive the growth of some medulloblastomas, and inhibition of this pathway has shown early promise in the treatment of patients with recurrent medulloblastoma Major Eligbility Age >= 22 years old Histologically confirmed recurrent medulloblastoma Neurologic deficits stable for at least 1 week  Clinicaltrials.gov ID NCT00939484

• PBTC032
  A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC0449 in Children with Recurrent and Refractory Medulloblastoma
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  Title A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC0449 in Children with Recurrent and Refractory Medulloblastoma Tumor Type recurrent medulloblastoma Description This is a Phase II study of GDC-0449, which is an oral drug that interferes with signaling of the sonic hedgehog pathway. This pathway is known to drive the growth of some medulloblastomas, and inhibition of this pathway has shown early promise in the treatment of patients with recurrent medulloblastoma. Major Eligbility Age: > 3 years and < 21 years old must have adequate tumor tissue available and measureable disease on MRI  Clinicaltrials.gov ID NCT01239316

• ACNS0821
  Temozolomide with Irinotecan Versus Temozolomide, Irinotecan plus Bevacizumab for Recurrent/Refractory Medulloblastoma/CNS PNET of Childhood, A COG Randomized Phase II Screening Trial
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  Title Temozolomide with Irinotecan Versus Temozolomide, Irinotecan plus Bevacizumab for Recurrent/Refractory Medulloblastoma/CNS PNET of Childhood, A COG Randomized Phase II Screening Trial Tumor Type recurrent/refractory medulloblastoma/PNET Description Major Eligbility Age: >21 yrs old Measureable residual disease Exclusion: serious/non-healing wound, known coagulopathy  Clinicaltrials.gov ID NCT01217437

Ependymoma – see also ‘All Recurrent/Progressive Tumors’

Low-grade glioma – see also ‘All Recurrent/Progressive Tumors’

• ACNS1022
  Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma
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  Title Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma Tumor Type Either optic pathway glioma OR biopsy-proven recurrent or progressive pilocytic astrocytoma; must have disease that is measureable on MRI. Patients with NF1 are eligible. Description This study is testing the benefit of lenalidomide in the treatment of children with either optic pathway gliomas or pilocytic astrocytomas. Lenalidomide is an oral agent with both anti-angiogenesis and immunomodulatory effect. It has been used in both adults and children and has been Major Eligbility Age < 22 years Tumor: either optic pathway glioma OR biopsy-proven recurrent or progressive pilocytic astrocytoma; must have disease that is measureable on MRI. Patients with NF1 are eligible. As this is an oral pill, patients must be able to tolerate oral medications. Clinicaltrials.gov ID NCT01553149

• PBTC029
  Phase I and Pharmacokinetic Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma
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  Title Phase I and Pharmacokinetic Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma Tumor Type refractory or recurrent low-grade glioma Description This is a Phase I study of AZD6244 in children with recurrent or refractory low-grade glioma. AZD6244 is an oral medication that inhibits MEK1 and MEK2, which are proteins that have recently been shown to be abnormal and encourage proliferation in low-grade gliomas. Inhibition of these proteins is hoped to cause the tumor to regress. Major Eligbility Age: >12 years and <21 years Recurrent or refractory low-grade glioma MUST have had radiation therapy (at least 3 months previously) and be able to swallow capsules  Clinicaltrials.gov ID NCT01089101

• CNMC VRL
  Weekly Vinorelbine for children with progressive or recurrent low-grade glioma
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  Title Weekly Vinorelbine for children with progressive or recurrent low-grade glioma Tumor Type recurrent low-grade glioma Description This is a Phase II study seeking to measure the efficacy and tolerability of Vinorelbine in children with recurrent/progressive low-grade glioma. Vinorelbine is a vinca alkaloid, a cousin of Vincristine. Vinorelbine has potentially less toxicity and has recently been shown to have potential efficacy in children with these tumors, stabilizing and reducing tumor size. Major Eligbility Age: < 18 years Must have histologic verification of low-grade glioma, except for brain stem or optic pathway gliomas May have neurofibromatosis

• RAD001
  A Phase II study of Everolimus (RAD001) for children with neurofibromatosis type 1 and chemotherapy-refractory radiographically progressive low-grade gliomas
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  Title A Phase II study of Everolimus (RAD001) for children with neurofibromatosis type 1 and chemotherapy-refractory radiographically progressive low-grade gliomas Tumor Type recurrent low-grade glioma (NF1+) Description This is a Phase II study of Everolimus (RAD001) in children with neurofibromatosis who have recurrent/refractory low-grade glioma. Everolimus targets the MTOR pathway, which has been implicated in development and growth of low-grade gliomas in children with NF. Major Eligbility Age: > 1 year and < 21 years Must have NF1 and progressive low-grade glioma that is measureable on MRI Must have failed carboplatin and be recovered from any side effects Exclusion: cannot be on enzyme-inducing anti-seizure medication

Recurrent DIPG

• PBTC036
  A Molecular Biology and Phase II Study of Imetelstat (GRN163L) in Children with Recurrent High-Grade Glioma, Ependymoma, Medulloblastoma/Primitive Neuroectodermal Tumor and Diffuse Intrinsic Pontine Glioma
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  Title A Molecular Biology and Phase II Study of Imetelstat (GRN163L) in Children with Recurrent High-Grade Glioma, Ependymoma, Medulloblastoma/Primitive Neuroectodermal Tumor and Diffuse Intrinsic Pontine Glioma Tumor Type recurrent ependymoma recurrent medulloblastoma recurrent DIPG recurrent high-grade glioma Description This Phase 2 and biology study seeks to evaluate the efficacy of imetelstat in a variety of aggressive pediatric brain tumors.  Imetelstat attacks an enzyme called telomerase, which is present in a very high proportion of pediatric brain tumors and helps to immortalize brain tumor cells.  Imetelstat prevents telomerase from working well.  It is hoped that by preventing this function, tumor cells will be unable to propagate themselves indefinitely. Major Eligbility Age: >12months to <21yrs Tumor: recurrent/refractory medulloblastoma, ependymoma, high-grade glioma, or diffuse intrinsic pontine glioma (DIPG) Performance status: >50% Exclusion: no known coagulopathy or use of systemic anticoagulant medications, for the biology portion of the study, patients need to have frozen tumor tissue available. For the Phase 2 portion of the study, patients need to have measurable disease