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Phenylbutyrate in Treating Children With Progressive or Recurrent Cancer of the Central Nervous System
This study is ongoing, but not recruiting participants.
First Received: September 11, 2000   Last Updated: July 23, 2008   History of Changes
Sponsors and Collaborators: Texas Children's Cancer Center
National Cancer Institute (NCI)
Information provided by: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00006238
  Purpose

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.

PURPOSE: Phase II trial to study the effectiveness of phenylbutyrate in treating children who have recurrent or progressive cancer of the central nervous system.


Condition Intervention Phase
Brain and Central Nervous System Tumors
Drug: sodium phenylbutyrate
Phase II

MedlinePlus related topics: Cancer
Drug Information available for: Sodium phenylbutyrate
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment
Official Title: Phase II Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Progressive or Recurrent CNS Malignancy

Further study details as provided by National Cancer Institute (NCI):

Study Start Date: November 2000
Detailed Description:

OBJECTIVES:

  • 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.

  Eligibility

Ages Eligible for Study:   2 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • 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 spectroscopy
  • Measurable disease by CT or MRI imaging that clearly demonstrates recurrent or progressive nature of the lesion
  • Histologic evidence of bone marrow involvement allowed

PATIENT CHARACTERISTICS:

Age:

  • 2 to 21

Performance status:

  • Lansky 50-100% (10 and under)
  • Karnofsky 50-100% (over 10)

Life expectancy:

  • At least 8 weeks

Hematopoietic:

  • 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

Hepatic:

  • Bilirubin no greater than 1.5 mg/dL
  • SGPT no greater than 2 times normal

Renal:

  • Creatinine normal for age OR
  • Creatinine clearance at least 70 mL/min

Other:

  • 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

PRIOR CONCURRENT THERAPY:

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

Chemotherapy:

  • 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 study

Radiotherapy:

  • 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

Surgery:

  • See Disease Characteristics
  • See Radiotherapy

Other:

  • No other concurrent investigational agents
  • Concurrent electrolyte supplements to maintain electrolyte levels allowed
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00006238

Locations
United States, California
UCSF Comprehensive Cancer Center
San Francisco, California, United States, 94143-0128
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010-2970
United States, Minnesota
University of Minnesota Cancer Center
Minneapolis, Minnesota, United States, 55455
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104-4318
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
Texas Children's Cancer Center
Houston, Texas, United States, 77030-2399
Sponsors and Collaborators
Texas Children's Cancer Center
Investigators
Study Chair: Susan M. Blaney, MD Texas Children's Cancer Center
  More Information

Additional Information:
No publications provided

Study ID Numbers: CDR0000068163, TCCC-H-6321, TCCC-GCRC-0692, NCI-O1-C-0028, NCI-T98-0042
Study First Received: September 11, 2000
Last Updated: July 23, 2008
ClinicalTrials.gov Identifier: NCT00006238     History of Changes
Health Authority: United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
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

Study placed in the following topic categories:
Choroid Plexus Neoplasms
Astrocytoma
4-phenylbutyric acid
Brain Tumor, Childhood
Central Nervous System Neoplasms
Ependymoma
Recurrence
Brain Neoplasms
Medulloblastoma
Craniopharyngioma
Oligodendroglioma
Meningioma
Glioma
Nervous System Neoplasms

Additional relevant MeSH terms:
Neoplasms
Neoplasms by Site
Antineoplastic Agents
4-phenylbutyric acid
Therapeutic Uses
Nervous System Diseases
Central Nervous System Neoplasms
Pharmacologic Actions
Nervous System Neoplasms

ClinicalTrials.gov processed this record on May 06, 2009