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Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors

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ClinicalTrials.gov Identifier: NCT00003241
Recruitment Status : Completed
First Posted : September 10, 2004
Last Update Posted : June 26, 2013
National Cancer Institute (NCI)
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

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 phenylacetate in treating children with recurrent or progressive brain tumors.

Condition or disease Intervention/treatment Phase
Brain and Central Nervous System Tumors Drug: phenylacetate Phase 2

Detailed Description:

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 years.

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Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Official Title: Phase II Study of Phenylacetate in Pediatric Patients With Central Nervous System Tumors
Study Start Date : May 1998
Actual Study Completion Date : September 2004

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00003241

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United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027-0700
UCSF Cancer Center and Cancer Research Institute
San Francisco, California, United States, 94115-0128
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010-2970
United States, Maryland
Pediatric Oncology Branch
Bethesda, Maryland, United States, 20892
United States, Ohio
Cleveland Clinic Cancer Center
Cleveland, Ohio, United States, 44195
United States, Texas
Texas Children's Cancer Center
Houston, Texas, United States, 77030-2399
Sponsors and Collaborators
Texas Children's Cancer Center
National Cancer Institute (NCI)
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Study Chair: Lisa Bomgaars, MD Texas Children's Cancer Center
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ClinicalTrials.gov Identifier: NCT00003241    
Other Study ID Numbers: TCCC-H-5057
CDR0000066116 ( Registry Identifier: PDQ (Physician Data Query) )
First Posted: September 10, 2004    Key Record Dates
Last Update Posted: June 26, 2013
Last Verified: April 2000
Keywords provided by National Cancer Institute (NCI):
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
Additional relevant MeSH terms:
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Nervous System Neoplasms
Central Nervous System Neoplasms
Neoplasms by Site
Nervous System Diseases
Phenylacetic acid
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents