Phenylbutyrate to Treat Children With Progressive or Recurrent Brain Tumors
Status:
Completed
Trial end date:
2002-07-01
Target enrollment:
Participant gender:
Summary
This study will examine the safety and effectiveness of treating brain tumors in children
with a continuous infusion of phenylbutyrate. A breakdown product of this drug,
phenylacetate, is normally found in low concentrations in the blood. At much higher
concentrations, phenylbutyrate and phenylacetate are active against cancer in animals.
Patients between 2 and 21 years old with a brain tumor that has progressed or recurred after
radiation or chemotherapy, including bone marrow transplant, may be eligible for this study.
Candidates will be screened with a medical history and physical examination, blood tests,
magnetic resonance imaging (MRI) or computerized tomography (CT) of the head and, if needed,
a spinal fluid test and bone marrow test.
Study participants will have a continuous infusion of phenylbutyrate for two 28-day
cycles-every day, 24 hours a day, 7 days a week. The medicine will be infused through a thin
tube (catheter) placed in a large vein in the upper chest, delivered through a portable
infusion pump. Patients will be hospitalized for at least 3 days when the treatment begins.
If there are no side effects at that time, the infusions can continue on an outpatient basis.
The patient or care giver will receive the medicine in 4-day supplies and will be taught how
to change the bag and tubing daily for drug administration, as well as how to use the
infusion pump. Patients will be monitored with weekly blood tests to look for side effects
and measure blood levels of phenylbutyrate. They will have a physical examination at least
once a week. At the end of the second 28-day cycle, patients will have a CT or MRI scan to
evaluate the tumor's response to treatment. Patients whose tumor has grown will stop
treatment and come off the study. Those whose tumor has remained stable or shrunk may
continue phenylbutyrate as long as the treatment is beneficial and there are no serious side
effects. CT or MRI scans will be done after every 2 cycles (or sooner if needed) to evaluate
the treatment.
Patients with certain tumor types (medulloblastoma, PNET, ependymoma, malignant germ cell
tumor and pineoblastoma) or who have symptoms that indicate there might be tumor along the
spinal cord may have a spinal tap. For this procedure, the patient lies on the side and a
needle is inserted between two vertebrae (bones of the spine) in the lower back, into the
cerebrospinal fluid space. A sample of fluid is drawn for testing for cancer cells. If the
tumor has spread through the spinal fluid, a spinal tap will be done every other cycle (every
2 months) to monitor the effects of therapy.