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Phase II Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Progressive or Recurrent CNS Malignancy

Phase 2
2 Years
21 Years
Open (Enrolling)
Brain and Central Nervous System Tumors

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Trial Information

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
unacceptable toxicity.

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.

Inclusion Criteria


- Histologically confirmed recurrent or progressive brain tumor after radiotherapy,
chemotherapy, or bone marrow transplantation

- High grade glioma (anaplastic astrocytoma or glioblastoma multiforme)

- Brain stem glioma

- Medulloblastoma or primitive neuroectodermal tumors present in supratentorial or
posterior fossa

- Other

- Histological confirmation waived in brain stem tumors

- Patients previously treated with radiosurgery require a biopsy, PET scan or NMR

- Measurable disease by CT or MRI imaging that clearly demonstrates recurrent or
progressive nature of the lesion

- Histologic evidence of bone marrow involvement allowed



- 2 to 21

Performance status:

- Lansky 50-100% (10 and under)

- Karnofsky 50-100% (over 10)

Life expectancy:

- At least 8 weeks


- Absolute neutrophil count at least 1,000/mm^3*

- Platelet count at least 50,000/mm^3*

- Hemoglobin at least 8.0 g/dL* NOTE: *Transfusion allowed


- Bilirubin no greater than 1.5 mg/dL

- SGPT no greater than 2 times normal


- Creatinine normal for age OR

- Creatinine clearance at least 70 mL/min


- No other concurrent significant systemic illness (e.g., infection)

- No significant electrolyte abnormalities

- No amino acidurias or organic acidemias

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception during and for 6 months after study


Biologic therapy:

- See Disease Characteristics

- Recovered from toxic effects of prior immunotherapy

- Prior bone marrow transplantation allowed

- No concurrent prophylactic hematopoietic growth factors except for neutropenia or
documented infection


- See Disease Characteristics

- At least 3 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosoureas)

- Recovered from prior chemotherapy

- No other concurrent chemotherapy

Endocrine therapy:

- If receiving dexamethasone must be on stable or decreasing dose for 2 weeks prior to


- See Disease Characteristics

- Recovered from prior radiotherapy

- More than 8 weeks since prior radiotherapy to evaluable lesion

- More than 4 months since prior radiosurgery to evaluable lesion

- Prior extensive radiotherapy (i.e., craniospinal radiotherapy or field encompassing
region greater than hemipelvis) allowed


- See Disease Characteristics

- See Radiotherapy


- No other concurrent investigational agents

- Concurrent electrolyte supplements to maintain electrolyte levels allowed

Type of Study:


Study Design:

Primary Purpose: Treatment

Principal Investigator

Susan M. Blaney, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Texas Children's Cancer Center


United States: Federal Government

Study ID:




Start Date:

November 2000

Completion Date:

Related Keywords:

  • Brain and Central Nervous System Tumors
  • childhood infratentorial ependymoma
  • childhood supratentorial ependymoma
  • childhood craniopharyngioma
  • recurrent childhood brain tumor
  • childhood central nervous system germ cell tumor
  • childhood high-grade cerebral astrocytoma
  • childhood oligodendroglioma
  • childhood choroid plexus tumor
  • childhood grade I meningioma
  • childhood grade II meningioma
  • childhood grade III meningioma
  • recurrent childhood cerebellar astrocytoma
  • recurrent childhood cerebral astrocytoma
  • recurrent childhood medulloblastoma
  • recurrent childhood visual pathway and hypothalamic glioma
  • recurrent childhood ependymoma
  • Nervous System Neoplasms
  • Central Nervous System Neoplasms



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