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
Primary Purpose: Treatment
Lisa Bomgaars, MD
Texas Children's Cancer Center
United States: Federal Government
|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|