Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
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.
|Neurofibromatosis Type 1 Precancerous Condition||Drug: pirfenidone||Phase 1|
|Study Design:||Masking: Open Label
Primary Purpose: Prevention
|Official Title:||Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas|
|Study Start Date:||December 2002|
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00053937
|United States, Alabama|
|University of Alabama at Birmingham Comprehensive Cancer Center|
|Birmingham, Alabama, United States, 35294-3300|
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010-2970|
|United States, Illinois|
|Children's Memorial Hospital - Chicago|
|Chicago, Illinois, United States, 60614|
|United States, Maryland|
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins|
|Baltimore, Maryland, United States, 21231|
|Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support|
|Bethesda, Maryland, United States, 20892|
|United States, Massachusetts|
|Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute|
|Boston, Massachusetts, United States, 02115|
|United States, Minnesota|
|Mayo Clinic Cancer Center|
|Rochester, Minnesota, United States, 55905|
|United States, Missouri|
|Washington University School of Medicine|
|Saint Louis, Missouri, United States, 63110|
|United States, New York|
|Beth Israel Medical Center - Singer Division|
|New York, New York, United States, 10128|
|University Hospital at State University of New York - Upstate Medical University|
|Syracuse, New York, United States, 13210|
|United States, Ohio|
|Cleveland Clinic Taussig Cancer Center|
|Cleveland, Ohio, United States, 44195|
|United States, Oregon|
|Cancer Institute at Oregon Health and Science University|
|Portland, Oregon, United States, 97239-3098|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Children's Hospital of Pittsburgh|
|Pittsburgh, Pennsylvania, United States, 15213|
|United States, Texas|
|Texas Children's Cancer Center|
|Houston, Texas, United States, 77030-2399|
|Study Chair:||Brigitte C. Widemann, MD||National Cancer Institute (NCI)|