Protocol Number: 03-C-0058
Patients between 3 and 21 years of age with NF1 and plexiform neurofibromas may be eligible for this study. Patients whose tumors can easily be removed surgically may not participate in this study. Candidates will have a medical history and physical examination, blood and urine tests, and magnetic resonance imaging (MRI). Study participants will take pirfenidone by mouth 3 times a day every day for 28 days. This constitutes one 28-day treatment cycle. The drug dose will be gradually increased in successive groups of three to six patients. Each group of patients must complete a 28-day cycle with no severe side effects before the next group receives a higher dose. The dose will continue to be increased until an effective dose in adults is reached. Treatment may continue for up to 2 years as long as the tumor remains stable or shrinks and side effects are tolerated. Patients (or their parents) will keep a record of treatment side effects and other medications taken. Blood tests to monitor drug side effects will be done before cycles 2, 3, 4, 7, and 10, and at 6-cycle intervals after that. Physical examinations will be done at the same times, but at 3-cycle intervals after cycle 10. MRI scans will be done before treatment starts, again before cycle 4, and every six cycles after that to measure the size of the tumors. On the first day of treatment, patients will take only one dose of pirfenidone and will have a series of blood tests to measure blood levels of drug at various times. Blood samples of less than a teaspoon will be drawn at 1, 2, 3, 5, 8, 12, 24, and 24.5 hours after the pirfenidone dose. A small indwelling tube will be placed in the vein to avoid multiple needle sticks for the frequent draws. Tumor tissue samples may also be obtained from patients scheduled to have a biopsy (surgical removal of a sample of tumor tissue for examination) or surgery as part of their medical management. The samples will be analyzed for cell components of plexiform neurofibromas. Some of the tissue may be grown in culture to study what patients might be likely to benefit from treatment. Patients (or their parents) will also be asked to fill out questionnaires assessing quality of life before starting treatment with pirfenidone, before treatment cycle 4, and then at 6-cycle intervals.
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