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Phase II Study of Phenylacetate in Pediatric Patients With Central Nervous System Tumors

Phase 2
2 Years
21 Years
Not Enrolling
Brain and Central Nervous System Tumors

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Trial Information

Phase II Study of Phenylacetate in Pediatric Patients With Central Nervous System Tumors

OBJECTIVES: I. Determine the efficacy of phenylacetate in terms of response rate and time to
progression in children with recurrent or progressive brain tumors, or with previously
untreated poor prognosis brain tumors. II. Assess the toxicity of phenylacetate in these
patients treated at the maximum tolerated dose. III. Determine the correlation between serum
steady state phenylacetate levels and toxicity or response in these patients.

OUTLINE: Patients are stratified by histologic type (anaplastic astrocytoma and glioblastoma
multiforme vs brain stem glioma vs medulloblastoma and primitive neuroectodermal tumors vs
ependymoma vs low grade glioma vs others). Patients receive phenylacetate as a continuous
intravenous infusion on days 1-28. Courses of treatment are given continuously without rest.
Treatment continues in the absence of disease progression or unacceptable toxicity. Patients
are followed weekly.

PROJECTED ACCRUAL: A total of 9-30 patients per stratum will be accrued for this study in 2

Inclusion Criteria

DISEASE CHARACTERISTICS: Histologically confirmed recurrent or progressive brain tumors
including the following: Anaplastic astrocytoma and glioblastoma multiforme Brain stem
glioma Medulloblastoma or primitive neuroectodermal tumors present in supratentorial or
posterior fossa locations Ependymoma Low grade gliomas Other Measurable or evaluable
disease by CT or MRI OR Histologically confirmed previously untreated glial tumors
including: Brain stem glioma Glioblastoma multiforme Measurable disease after surgery

PATIENT CHARACTERISTICS: Age: 2 to 21 Performance status: Karnofsky 50-100% (over 10 years
of age) Lansky 50-100% (under 10 years of age) Life expectancy: At least 8 weeks
Hematopoietic: Absolute neutrophil count greater than 1,000/mm3 Platelet count greater
than 50,000/mm3 Hemoglobin greater than 7.0 g/dL Transfusion support allowed after bone
marrow transplantation or extensive radiation Hepatic: Bilirubin less than 1.5 mg/dL SGPT
less than 2 times normal Renal: Creatinine no greater than 1.5 mg/dL OR Creatinine
clearance greater than 60 mL/min Other: Not pregnant or nursing Fertile patients must use
effective contraception during and for 6 months after study No significant systemic
illness including infections No amino acidurias or organic acidemias

PRIOR CONCURRENT THERAPY: Biologic therapy: Recovered from all prior immunotherapy No
concurrent prophylactic hematopoietic growth factors Chemotherapy: At least 3 weeks since
prior myelosuppressive chemotherapy (6 weeks for nitrosourea) and recovered No other
concurrent cancer chemotherapy Endocrine therapy: Stable or decreasing dosage of
dexamethasone for intracranial pressure within 2 weeks of study entry No concurrent
dexamethasone used as an antiemetic Radiotherapy: At least 8 weeks since prior
radiotherapy to evaluable lesions and recovered Surgery: At least 4 months since prior
radiosurgery Other: No other concurrent investigational agents

Type of Study:


Study Design:

Primary Purpose: Treatment

Principal Investigator

Lisa Bomgaars, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Texas Children's Cancer Center


United States: Federal Government

Study ID:




Start Date:

May 1998

Completion Date:

September 2004

Related Keywords:

  • Brain and Central Nervous System Tumors
  • recurrent childhood brain tumor
  • childhood high-grade cerebral astrocytoma
  • untreated childhood brain stem glioma
  • recurrent childhood brain stem glioma
  • recurrent childhood supratentorial primitive neuroectodermal tumor
  • recurrent childhood cerebellar astrocytoma
  • recurrent childhood cerebral astrocytoma
  • recurrent childhood medulloblastoma
  • recurrent childhood ependymoma
  • Brain Neoplasms
  • Nervous System Neoplasms
  • Central Nervous System Neoplasms



Cleveland Clinic Cancer Center Cleveland, Ohio  44195
UCSF Cancer Center and Cancer Research Institute San Francisco, California  94115-0128
Children's Hospital Los Angeles Los Angeles, California  90027-0700
Children's National Medical Center Washington, District of Columbia  20010-2970
Texas Children's Cancer Center Houston, Texas  77030-2399
Pediatric Oncology Branch Bethesda, Maryland  20892