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Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors

Primary Purpose

Brain and Central Nervous System Tumors

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
phenylacetate
Sponsored by
Texas Children's Cancer Center
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Brain and Central Nervous System Tumors focused on measuring 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

Eligibility Criteria

2 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Hospital Los Angeles
  • UCSF Cancer Center and Cancer Research Institute
  • Children's National Medical Center
  • Pediatric Oncology Branch
  • Cleveland Clinic Cancer Center
  • Texas Children's Cancer Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
November 1, 1999
Last Updated
June 25, 2013
Sponsor
Texas Children's Cancer Center
Collaborators
National Cancer Institute (NCI)
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1. Study Identification

Unique Protocol Identification Number
NCT00003241
Brief Title
Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors
Official Title
Phase II Study of Phenylacetate in Pediatric Patients With Central Nervous System Tumors
Study Type
Interventional

2. Study Status

Record Verification Date
April 2000
Overall Recruitment Status
Completed
Study Start Date
May 1998 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
September 2004 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Texas Children's Cancer Center
Collaborators
National Cancer Institute (NCI)

4. Oversight

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Brain and Central Nervous System Tumors
Keywords
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
phenylacetate

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lisa Bomgaars, MD
Organizational Affiliation
Texas Children's Cancer Center
Official's Role
Study Chair
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027-0700
Country
United States
Facility Name
UCSF Cancer Center and Cancer Research Institute
City
San Francisco
State/Province
California
ZIP/Postal Code
94115-0128
Country
United States
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010-2970
Country
United States
Facility Name
Pediatric Oncology Branch
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Cleveland Clinic Cancer Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Texas Children's Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030-2399
Country
United States

12. IPD Sharing Statement

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Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors

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