RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Pirfenidone may slow the growth or prevent further development of plexiform neurofibromas. PURPOSE: Phase I trial to study the effectiveness of pirfenidone in treating young patients who have neurofibromatosis type 1 and plexiform neurofibroma.
OBJECTIVES: * Determine the maximum tolerated dose or "comparable dose" of pirfenidone in pediatric patients with neurofibromatosis type 1 and inoperable, symptomatic plexiform neurofibromas. * Determine the toxic effects of this drug in these patients. * Determine the plasma pharmacokinetics of this drug in these patients. * Determine, preliminarily, if this drug could be beneficial for pediatric patients with refractory solid tumors. * Assess the quality of life of patients treated with this drug. OUTLINE: This is an open-label, multicenter, dose-escalation study. Patients receive oral pirfenidone three times daily on days 1-28. Courses repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Cohorts of 3-6 patients receive escalating doses of pirfenidone until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity. Quality of life is assessed at baseline, before course 4, and then after every 6 courses. PROJECTED ACCRUAL: A total of 3-18 patients will be accrued for this study within 18 months.
Study Type
INTERVENTIONAL
Purpose
PREVENTION
Masking
NONE
University of Alabama at Birmingham Comprehensive Cancer Center
Birmingham, Alabama, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
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Children's Memorial Hospital - Chicago
Chicago, Illinois, United States
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
Bethesda, Maryland, United States
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute
Boston, Massachusetts, United States
Mayo Clinic Cancer Center
Rochester, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
Beth Israel Medical Center - Singer Division
New York, New York, United States
University Hospital at State University of New York - Upstate Medical University
Syracuse, New York, United States
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