Ignite Creation Date:
2024-05-05 @ 11:29 AM
Last Modification Date:
2024-10-26 @ 9:08 AM
Study NCT ID:
NCT00053937
Status:
COMPLETED
Last Update Posted:
2015-04-30
First Post:
2003-02-05
Brief Title:
Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
Sponsor:
National Cancer Institute NCI
Organization:
National Cancer Institute NCI
Study Overview
Official Title:
Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas
Status:
COMPLETED
Status Verified Date:
2004-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| NCI-03-C-0058A | None | None | None |