Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors

Brain and Central Nervous System Tumors Clinical Trial

Official title:

Phase II Study of Phenylacetate in Pediatric Patients With Central Nervous System Tumors

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00003241
• Other study ID #: TCCC-H-5057
• Secondary ID: CDR0000066116NCI
• Status: Completed
• Phase: Phase 2
• First received: November 1, 1999
• Last updated: June 25, 2013
• Start date: May 1998
• Est. completion date: September 2004

Study information

• Verified date: April 2000
• Source: National Cancer Institute (NCI)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. completion date: September 2004
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years to 21 Years

Eligibility

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

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Brain and Central Nervous System Tumors
• Central Nervous System Neoplasms
• Nervous System Neoplasms

Intervention

Drug:
• phenylacetate

Locations

Country	Name	City	State
United States	Pediatric Oncology Branch	Bethesda	Maryland
United States	Cleveland Clinic Cancer Center	Cleveland	Ohio
United States	Texas Children's Cancer Center	Houston	Texas
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	UCSF Cancer Center and Cancer Research Institute	San Francisco	California
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (2)

Lead Sponsor	Collaborator
Texas Children's Cancer Center	National Cancer Institute (NCI)

Country where clinical trial is conducted

United States,