- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00001565
Phase I and Pharmacokinetic Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Refractory Malignancy
This is a pharmacokinetic trial. Patients receive phenylbutyrate through a central venous catheter for each 28 day cycle. The first several days of drug administration should be inpatient. Cycles may be repeated if there is no tumor progression or dose limiting toxicities (DLT). There are no breaks between cycles.
Once a minimum of 3 patients have completed at least 4 weeks of therapy without DLT, new patients will be entered at the next dose level.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment
Phase
- Phase 1
Contacts and Locations
Study Locations
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Maryland
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Bethesda, Maryland, United States, 20892
- National Cancer Institute (NCI)
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Disease Characteristics:
Histologically proven cancer that is refractory to standard therapy.
Patients with neurofibromatosis having progressive inoperable plexiform neurofibromas with potential to cause significant morbidity are eligible.
Patients with brainstem gliomas histology may have histology requirements waived.
Patients without prior therapy are eligible if they have diseases with no available standard therapy.
Patients with evidence of bone marrow involvement by tumor, or a history of either bone marrow transplantation or extensive radiotherapy will be eligible, but inevaluable for hematologic toxicities.
Patients with greater than grade 2 neurocortical toxicity will be excluded.
PRIOR/CONCURRENT THERAPY:
Biologic Therapy: No concurrent hematopoietic growth factor.
Chemotherapy: No chemotherapy within 3 weeks of study.
No nitrosoursea within 6 weeks of study.
No concurrent chemotherapy allowed.
Must be on stable or decreasing dose of dexamethasone within 2 weeks of study.
Endocrine Therapy: Not specified.
Radiotherapy: No radiotherapy within 6 weeks of study.
Surgery: Not specified.
Other:
Patient must be recovered from toxic effects of all prior therapy.
Concurrent antibiotic therapy when appropriate.
Patient Characteristics:
Age: 2 to 21.
Performance Status: ECOG 0-2.
Life Expectancy: At least 8 weeks.
Hematopoietic (hematologic requirements below do not apply to patients with histologically confirmed bone marrow involvement or history of either bone marrow transplantation or extensive radiotherapy; these patients are inevaluable for hematologic toxicity):
Absolute granulocyte count (AGC) at least 1500/mm3.
Platelet count at least 100,000/mm3.
Hemoglobin at least 8 g/dL.
Hepatic:
Bilirubin no greater than 2 mg/Dl.
SGPT less than 2 times normal.
Renal:
Creatinine no greater than 1.5 mg/Dl OR
Creatinine clearance at least 60 Ml/min/square meter.
Other:
No systemic illness.
Not pregnant or nursing.
No amino acidurias or organic acidemias.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Sponsor
Publications and helpful links
General Publications
- Samid D, Shack S, Myers CE. Selective growth arrest and phenotypic reversion of prostate cancer cells in vitro by nontoxic pharmacological concentrations of phenylacetate. J Clin Invest. 1993 May;91(5):2288-95. doi: 10.1172/JCI116457.
- Samid D, Ram Z, Hudgins WR, Shack S, Liu L, Walbridge S, Oldfield EH, Myers CE. Selective activity of phenylacetate against malignant gliomas: resemblance to fetal brain damage in phenylketonuria. Cancer Res. 1994 Feb 15;54(4):891-5.
- Samid D, Shack S, Sherman LT. Phenylacetate: a novel nontoxic inducer of tumor cell differentiation. Cancer Res. 1992 Apr 1;52(7):1988-92.
Study record dates
Study Major Dates
Study Start
Study Completion
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Neoplasms by Site
- Neoplasms, Glandular and Epithelial
- Neoplasms, Neuroepithelial
- Neuroectodermal Tumors
- Neoplasms, Germ Cell and Embryonal
- Neoplasms, Nerve Tissue
- Central Nervous System Neoplasms
- Nervous System Neoplasms
- Neuroectodermal Tumors, Primitive
- Neuroectodermal Tumors, Primitive, Peripheral
- Brain Neoplasms
- Neuroblastoma
- Antineoplastic Agents
- 4-phenylbutyric acid
Other Study ID Numbers
- 970051
- 97-C-0051
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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