Phenylbutyrate in Treating Children With Progressive or Recurrent Cancer of the Central Nervous System
Recruitment status was Active, not recruiting
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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 2 |
| Study Type: | Interventional |
| Study Design: | Primary Purpose: Treatment |
| Official Title: | Phase II Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Progressive or Recurrent CNS Malignancy |
| Study Start Date: | November 2000 |
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 |
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| 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 | |
| Study Chair: | Susan M. Blaney, MD | Texas Children's Cancer Center |
More Information
Additional Information:
No publications provided
| ClinicalTrials.gov Identifier: | NCT00006238 History of Changes |
| Other 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 |
| 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 |
Additional relevant MeSH terms:
|
Nervous System Neoplasms Central Nervous System Neoplasms Neoplasms by Site Neoplasms Nervous System Diseases |
4-phenylbutyric acid Antineoplastic Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 23, 2013