Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas

Phase 1

Completed

Brief Summary: 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.

Detailed Description: 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.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Locations (15): Children's Hospital of Philadelphia
🇺🇸
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
🇺🇸
Pittsburgh, Pennsylvania, United States
Children's National Medical Center
🇺🇸
Washington, District of Columbia, United States
University of Alabama at Birmingham Comprehensive Cancer Center
🇺🇸
Birmingham, Alabama, United States
Cancer Institute at Oregon Health and Science University
🇺🇸
Portland, Oregon, United States
Texas Children's Cancer Center
🇺🇸
Houston, Texas, United States
Mayo Clinic Cancer Center
🇺🇸
Rochester, Minnesota, United States
Washington University School of Medicine
🇺🇸
Saint Louis, Missouri, United States
Children's Memorial Hospital - Chicago
🇺🇸
Chicago, Illinois, United States
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
🇺🇸
Bethesda, Maryland, United States
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
🇺🇸
Baltimore, Maryland, United States
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute
🇺🇸
Boston, Massachusetts, 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
Cleveland Clinic Taussig Cancer Center
🇺🇸
Cleveland, Ohio, United States