Phase I and Pharmacokinetic Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Refractory Malignancy
Launched by NATIONAL CANCER INSTITUTE (NCI) · Dec 9, 2002
Trial Information
Current as of January 15, 2025
Completed
Keywords
ClinConnect Summary
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 obj...
Gender
ALL
Eligibility criteria
- 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.
About National Cancer Institute (Nci)
The National Cancer Institute (NCI) is a prominent component of the National Institutes of Health (NIH), dedicated to advancing cancer research and improving patient outcomes through innovative clinical trials. As a leading sponsor of cancer-related studies, NCI focuses on facilitating the development of new therapies, enhancing prevention strategies, and understanding the biology of cancer. The institute collaborates with academic institutions, healthcare providers, and industry partners to conduct rigorous clinical trials that aim to translate scientific discoveries into effective treatments. NCI’s commitment to fostering a robust research environment supports the mission to eliminate cancer as a major health problem.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Bethesda, Maryland, United States
People applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
Discussion 0
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