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Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors

Study 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 phenylacetate in treating children with recurrent or progressive brain tumors.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 2 Years - 21 Years
Gender All
More Inclusion & Exclusion 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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT00003241
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Texas Children's Cancer Center
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Lisa Bomgaars, MD
Principal Investigator Affiliation Texas Children's Cancer Center
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other, NIH
Overall Status Completed
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Brain and Central Nervous System Tumors
Additional Details

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.#46;brain stem glioma vs.#46;medulloblastoma and primitive neuroectodermal tumors vs.#46;ependymoma vs.#46;low grade glioma vs.#46;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.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Children's Hospital Los Angeles, Los Angeles, California

Status

Address

Children's Hospital Los Angeles

Los Angeles, California, 90027-0700

San Francisco, California

Status

Address

UCSF Cancer Center and Cancer Research Institute

San Francisco, California, 94115-0128

Children's National Medical Center, Washington, District of Columbia

Status

Address

Children's National Medical Center

Washington, District of Columbia, 20010-2970

Pediatric Oncology Branch, Bethesda, Maryland

Status

Address

Pediatric Oncology Branch

Bethesda, Maryland, 20892

Cleveland Clinic Cancer Center, Cleveland, Ohio

Status

Address

Cleveland Clinic Cancer Center

Cleveland, Ohio, 44195

Texas Children's Cancer Center, Houston, Texas

Status

Address

Texas Children's Cancer Center

Houston, Texas, 77030-2399