NCT00053937

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

Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: —
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 3 Years – 21 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Neurofibromatosis Type 1
• Precancerous Condition

Interventions

Type	Name	Description
DRUG	pirfenidone

Timeline

• Start date: 2002-12-01
• First posted: 2003-02-06
• Last updated: 2015-04-30

Locations

15 sites across 1 country: United States