Search / Trial NCT00001565

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

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Trial Information

Current as of March 05, 2024

Completed

Keywords

Brain Tumors Differentiation Maximally Tolerated Dose Neuroblastoma Phenylacetate

Description

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.

Attachments

readout_NCT00001565_2024-03-05.pdf

4.5 MB

NCT00001565_study_protocol.pdf

4.5 MB

About company

The National Institute of Allergy and Infectious Diseases (NIAID, /ˈnaɪ.æd/) is one of the 27 institutes and centers that make up the National Institutes of Health (NIH), an agency of the United States Department of Health and Human Services (HHS). NIAID's mission is to conduct basic and applied research to better understand, treat, and prevent infectious, immunologic, and allergic diseases.

Contacts

JC

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

Reviews (48)

4.6

All reviews come from applied patients

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Leslie Alexander
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Michael Foster
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Dries Vincent
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Leslie Alexander
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

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