Phase II Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Progressive or Recurrent CNS Malignancy
- Determine the therapeutic efficacy of phenylbutyrate in terms of response rate and time
to progression in children with recurrent or progressive CNS malignancy.
- Determine the toxicity of this regimen in these patients.
- Determine the correlation between serum steady state phenylbutyrate levels and response
or toxicity in these patients.
OUTLINE: This is a multicenter study. Patients are stratified according to tumor histology
(high grade glioma (anaplastic astrocytoma or glioblastoma multiforme) vs brain stem glioma
vs medulloblastoma or primitive neuroectodermal tumors vs other).
Patients receive phenylbutyrate IV continuously on days 1-28. Treatment continues every 4
weeks for up to a maximum of 12 courses in the absence of disease progression or
Patients are followed at 1 week.
PROJECTED ACCRUAL: A maximum of 120 patients (approximately 9-24 evaluable patients per
stratum) will be accrued for this study within 2 years.
Primary Purpose: Treatment
Susan M. Blaney, MD
Texas Children's Cancer Center
United States: Federal Government
|Children's Hospital of Philadelphia
|Philadelphia, Pennsylvania 19104
|University of Minnesota Cancer Center
|Minneapolis, Minnesota 55455
|Children's National Medical Center
|Washington, District of Columbia 20010-2970
|Children's Hospital of Pittsburgh
|Pittsburgh, Pennsylvania 15213
|Texas Children's Cancer Center
|Houston, Texas 77030-2399
|UCSF Comprehensive Cancer Center
|San Francisco, California 94115