Nctid:
NCT00001565
Payload:
{"hasResults"=>false, "derivedSection"=>{"miscInfoModule"=>{"versionHolder"=>"2024-09-05"}, "conditionBrowseModule"=>{"meshes"=>[{"id"=>"D000009447", "term"=>"Neuroblastoma"}, {"id"=>"D000001932", "term"=>"Brain Neoplasms"}], "ancestors"=>[{"id"=>"D000009369", "term"=>"Neoplasms"}, {"id"=>"D000018241", "term"=>"Neuroectodermal Tumors, Primitive, Peripheral"}, {"id"=>"D000018242", "term"=>"Neuroectodermal Tumors, Primitive"}, {"id"=>"D000018302", "term"=>"Neoplasms, Neuroepithelial"}, {"id"=>"D000017599", "term"=>"Neuroectodermal Tumors"}, {"id"=>"D000009373", "term"=>"Neoplasms, Germ Cell and Embryonal"}, {"id"=>"D000009370", "term"=>"Neoplasms by Histologic Type"}, {"id"=>"D000009375", "term"=>"Neoplasms, Glandular and Epithelial"}, {"id"=>"D000009380", "term"=>"Neoplasms, Nerve Tissue"}, {"id"=>"D000016543", "term"=>"Central Nervous System Neoplasms"}, {"id"=>"D000009423", "term"=>"Nervous System Neoplasms"}, {"id"=>"D000009371", "term"=>"Neoplasms by Site"}, {"id"=>"D000001927", "term"=>"Brain Diseases"}, {"id"=>"D000002493", "term"=>"Central Nervous System Diseases"}, {"id"=>"D000009422", "term"=>"Nervous System Diseases"}], "browseLeaves"=>[{"id"=>"M5209", "name"=>"Brain Neoplasms", "asFound"=>"Brain Neoplasms", "relevance"=>"HIGH"}, {"id"=>"M12391", "name"=>"Neuroblastoma", "asFound"=>"Neuroblastoma", "relevance"=>"HIGH"}, {"id"=>"M20387", "name"=>"Neuroectodermal Tumors, Primitive, Peripheral", "relevance"=>"LOW"}, {"id"=>"M19845", "name"=>"Neuroectodermal Tumors", "relevance"=>"LOW"}, {"id"=>"M20388", "name"=>"Neuroectodermal Tumors, Primitive", "relevance"=>"LOW"}, {"id"=>"M20446", "name"=>"Neoplasms, Neuroepithelial", "relevance"=>"LOW"}, {"id"=>"M12318", "name"=>"Neoplasms, Germ Cell and Embryonal", "relevance"=>"LOW"}, {"id"=>"M12315", "name"=>"Neoplasms by Histologic Type", "relevance"=>"LOW"}, {"id"=>"M12320", "name"=>"Neoplasms, Glandular and Epithelial", "relevance"=>"LOW"}, {"id"=>"M12325", "name"=>"Neoplasms, Nerve Tissue", "relevance"=>"LOW"}, {"id"=>"M12367", "name"=>"Nervous System Neoplasms", "relevance"=>"LOW"}, {"id"=>"M18937", "name"=>"Central Nervous System Neoplasms", "relevance"=>"LOW"}, {"id"=>"M5204", "name"=>"Brain Diseases", "relevance"=>"LOW"}, {"id"=>"M5742", "name"=>"Central Nervous System Diseases", "relevance"=>"LOW"}, {"id"=>"T4085", "name"=>"Neuroblastoma", "asFound"=>"Neuroblastoma", "relevance"=>"HIGH"}, {"id"=>"T4092", "name"=>"Neuroepithelioma", "relevance"=>"LOW"}], "browseBranches"=>[{"name"=>"Neoplasms", "abbrev"=>"BC04"}, {"name"=>"Nervous System Diseases", "abbrev"=>"BC10"}, {"name"=>"All Conditions", "abbrev"=>"All"}, {"name"=>"Rare Diseases", "abbrev"=>"Rare"}]}, "interventionBrowseModule"=>{"meshes"=>[{"id"=>"C000075773", "term"=>"4-phenylbutyric acid"}], "ancestors"=>[{"id"=>"D000000970", "term"=>"Antineoplastic Agents"}], "browseLeaves"=>[{"id"=>"M245537", "name"=>"4-phenylbutyric acid", "asFound"=>"L3", "relevance"=>"HIGH"}, {"id"=>"M221795", "name"=>"Phenylacetic acid", "relevance"=>"LOW"}], "browseBranches"=>[{"name"=>"Antineoplastic Agents", "abbrev"=>"ANeo"}, {"name"=>"All Drugs and Chemicals", "abbrev"=>"All"}]}}, "protocolSection"=>{"designModule"=>{"phases"=>["PHASE1"], "studyType"=>"INTERVENTIONAL", "designInfo"=>{"primaryPurpose"=>"TREATMENT"}, "enrollmentInfo"=>{"count"=>35}}, "statusModule"=>{"overallStatus"=>"COMPLETED", "startDateStruct"=>{"date"=>"1996-12"}, "expandedAccessInfo"=>{"hasExpandedAccess"=>false}, "statusVerifiedDate"=>"1999-11", "completionDateStruct"=>{"date"=>"2000-10"}, "lastUpdateSubmitDate"=>"2008-03-03", "studyFirstSubmitDate"=>"1999-11-03", "studyFirstSubmitQcDate"=>"2002-12-09", "lastUpdatePostDateStruct"=>{"date"=>"2008-03-04", "type"=>"ESTIMATED"}, "studyFirstPostDateStruct"=>{"date"=>"2002-12-10", "type"=>"ESTIMATED"}}, "conditionsModule"=>{"keywords"=>["Brain Tumors", "Differentiation", "Maximally Tolerated Dose", "Neuroblastoma", "Phenylacetate"], "conditions"=>["Brain Neoplasms", "Neuroblastoma"]}, "referencesModule"=>{"references"=>[{"pmid"=>"8486788", "type"=>"BACKGROUND", "citation"=>"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."}, {"pmid"=>"8313377", "type"=>"BACKGROUND", "citation"=>"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."}, {"pmid"=>"1372534", "type"=>"BACKGROUND", "citation"=>"Samid D, Shack S, Sherman LT. Phenylacetate: a novel nontoxic inducer of tumor cell differentiation. Cancer Res. 1992 Apr 1;52(7):1988-92."}]}, "descriptionModule"=>{"briefSummary"=>"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.\n\nOnce 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.", "detailedDescription"=>"Phenylbutyrate is an aromatic fatty acid that is converted to phenylacetate in vivo by mitochondrial beta-oxidation to phenylacetate. Preclinical studies have shown that continuous exposure to phenylacetate or phenylbutyrate can induce tumor cytostasis and differentiation in a wide variety of cell lines including malignant gliomas and neuroblastomas. However, phenylbutyrate has been shown to be a more potent differentiating agent than phenylacetate in a variety of tumor cell lines. In addition, phenylbutyrate appears to have molecular activities that are distinct from phenylacetate. The objective of this trial is to determine the maximum tolerated dose and the toxicities of phenylbutyrate administered as a continuous intravenous infusion for 28 days. In addition, the pharmacokinetics of phenylbutyrate and its metabolite, phenylacetate, will be studied using both model-dependent and model-independent parameters."}, "eligibilityModule"=>{"sex"=>"ALL", "stdAges"=>["CHILD", "ADULT", "OLDER_ADULT"], "healthyVolunteers"=>false, "eligibilityCriteria"=>"Disease Characteristics:\n\nHistologically proven cancer that is refractory to standard therapy.\n\nPatients with neurofibromatosis having progressive inoperable plexiform neurofibromas with potential to cause significant morbidity are eligible.\n\nPatients with brainstem gliomas histology may have histology requirements waived.\n\nPatients without prior therapy are eligible if they have diseases with no available standard therapy.\n\nPatients 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.\n\nPatients with greater than grade 2 neurocortical toxicity will be excluded.\n\nPRIOR/CONCURRENT THERAPY:\n\nBiologic Therapy: No concurrent hematopoietic growth factor.\n\nChemotherapy: No chemotherapy within 3 weeks of study.\n\nNo nitrosoursea within 6 weeks of study.\n\nNo concurrent chemotherapy allowed.\n\nMust be on stable or decreasing dose of dexamethasone within 2 weeks of study.\n\nEndocrine Therapy: Not specified.\n\nRadiotherapy: No radiotherapy within 6 weeks of study.\n\nSurgery: Not specified.\n\nOther:\n\nPatient must be recovered from toxic effects of all prior therapy.\n\nConcurrent antibiotic therapy when appropriate.\n\nPatient Characteristics:\n\nAge: 2 to 21.\n\nPerformance Status: ECOG 0-2.\n\nLife Expectancy: At least 8 weeks.\n\nHematopoietic (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):\n\nAbsolute granulocyte count (AGC) at least 1500/mm3.\n\nPlatelet count at least 100,000/mm3.\n\nHemoglobin at least 8 g/dL.\n\nHepatic:\n\nBilirubin no greater than 2 mg/Dl.\n\nSGPT less than 2 times normal.\n\nRenal:\n\nCreatinine no greater than 1.5 mg/Dl OR\n\nCreatinine clearance at least 60 Ml/min/square meter.\n\nOther:\n\nNo systemic illness.\n\nNot pregnant or nursing.\n\nNo amino acidurias or organic acidemias."}, "identificationModule"=>{"nctId"=>"NCT00001565", "briefTitle"=>"Phase I and Pharmacokinetic Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Refractory Malignancy", "organization"=>{"class"=>"NIH", "fullName"=>"National Institutes of Health Clinical Center (CC)"}, "officialTitle"=>"Phase I and Pharmacokinetic Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Refractory Malignancy", "orgStudyIdInfo"=>{"id"=>"970051"}, "secondaryIdInfos"=>[{"id"=>"97-C-0051"}]}, "armsInterventionsModule"=>{"interventions"=>[{"name"=>"phenylbutyrate", "type"=>"DRUG"}]}, "contactsLocationsModule"=>{"locations"=>[{"zip"=>"20892", "city"=>"Bethesda", "state"=>"Maryland", "country"=>"United States", "facility"=>"National Cancer Institute (NCI)", "geoPoint"=>{"lat"=>38.98067, "lon"=>-77.10026}}]}, "sponsorCollaboratorsModule"=>{"leadSponsor"=>{"name"=>"National Cancer Institute (NCI)", "class"=>"NIH"}}}}
