Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas
- Determine the maximum tolerated dose or "comparable dose" of pirfenidone in pediatric
patients with neurofibromatosis type 1 and inoperable, symptomatic plexiform
- 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.
Masking: Open Label, Primary Purpose: Prevention
Brigitte C. Widemann, MD
National Cancer Institute (NCI)
United States: Federal Government
|Children's Hospital of Philadelphia||Philadelphia, Pennsylvania 19104|
|Mayo Clinic Cancer Center||Rochester, Minnesota 55905|
|Washington University School of Medicine||Saint Louis, Missouri 63110|
|University of Alabama at Birmingham Comprehensive Cancer Center||Birmingham, Alabama 35294-3300|
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins||Baltimore, Maryland 21231-2410|
|Cleveland Clinic Taussig Cancer Center||Cleveland, Ohio 44195|
|Children's National Medical Center||Washington, District of Columbia 20010-2970|
|Children's Hospital of Pittsburgh||Pittsburgh, Pennsylvania 15213|
|Children's Memorial Hospital - Chicago||Chicago, Illinois 60614|
|Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute||Boston, Massachusetts 02115|
|Texas Children's Cancer Center||Houston, Texas 77030-2399|
|Cancer Institute at Oregon Health and Science University||Portland, Oregon 97201-3098|
|University Hospital at State University of New York - Upstate Medical University||Syracuse, New York 13210|
|Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support||Bethesda, Maryland 20892-1182|
|Beth Israel Medical Center - Singer Division||New York, New York 10128|