(PBMTC ONC-032) High Dose Temozolomide, Thiotepa and Carboplatin with Autologous Stem Cell Rescue (ASCR) Followed by Continuation Therapy with 13-cis-Retinoic Acid in Patients with Recurrent/Refractory Malignant Brain Tumors

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Title (PBMTC ONC-032) High Dose Temozolomide, Thiotepa and Carboplatin with Autologous Stem Cell Rescue (ASCR) Followed by Continuation Therapy with 13-cis-Retinoic Acid in Patients with Recurrent/Refractory Malignant Brain Tumors
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Principal Investigator Barbara Bambach, MD
Study Number 63705
Phase2
Summary The primary aim of this protocol is to determine the event-free survival (EFS) and overall survival (OS) of patients with recurrent or refractory medullobastoma/primitive neuroectodermal tumors (PNET) with minimal residual disease treated with high dose temozolomide, thiotepa and carboplatin with autologous stem cell rescue (ASCR) followed by continuation therapy with 13-cis-retinoic acid. It will also evaluate the toxicity of high dose therapy with ASCR when performed in a multi-institutional setting; determine the inter-individual variability in plasma pharmacokinetics of 13-cis-retinoic acid and assess single CSF to determine if it is a potential indicator of CNS penetration of the drug; and also will investigate genetic variation in enzymes and genetic variations to clinical response through pharmacokinetic tests.
Eligibility
  • Patients with recurrent or refractory medullobastoma/PNET,CNS germ cell tumors, ependymomas, AT/RT, high grade glioma and other malignant brain tumors.
  • Must have recurrent or refractory disease following at least one prior course of therapy.
  • Karnofsky performance status or Lansky performance score of greater than or equal to 70%.
  • Life expectancy of greater than 12 weeks
  • Adequate hematologic, renal, liver and cardiac functions as demonstrated by lab values performed within 21 days, inclusive, prior to administration of temozolomide
  • Must have adequate number of autologous stem cell available defined as a minimum of 2 x 10 (6) CD 34+cells/kg and preferable at least 5 x 10 (6) CD 34+ cells/kg.
  • Age greater than 6 months and less than 21 years of age.
  • Must have recovered from any effects of major surgery, chemotherapy or radiation.
  • Patients may have received stereotactic radiation at the time of recurrence as part of their salvage treatment
  • Written informed consent
For more information about the clinical study go to Clinicaltrial.Gov