NCT00030264

Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas

Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 23 (actual)
• Sponsor: Children's Hospital of Philadelphia · Academic / Other
• Sex: All
• Age: 25 Years
• Healthy volunteers: Not accepted

Summary

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining methotrexate with vinblastine may be effective treatment for neurofibromatosis type 1 associated with progressive plexiform neurofibromas. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have neurofibromatosis type 1 associated with progressive plexiform neurofibromas.

Detailed description

OBJECTIVES: * Determine the effect of chronic vinblastine and methotrexate on time to disease progression in children or young adults with progressive plexiform neurofibroma associated with neurofibromatosis type 1. * Determine the objective response rate in patients treated with this regimen. * Determine the toxic effects of this regimen in these patients. * Determine the quality of life of patients treated with this regimen. OUTLINE: Patients are stratified according to tumor status (severely debilitating and/or life-threatening vs cosmetically disfiguring). Patients receive methotrexate and vinblastine IV weekly for 26 weeks and then every 2 weeks for 26 weeks in the absence of disease progression or unacceptable toxicity. Quality of life is assessed at baseline and then every 3 months during study participation. Patients are followed every 3 months until disease progression. PROJECTED ACCRUAL: A total of 35 patients will be accrued for this study within approximately 3 years.

Conditions

• Neurofibromatosis Type 1
• Precancerous Condition

Interventions

Type	Name	Description
DRUG	Methotrexate	Methotrexate will be given at a dose of 30mg/m2/week intramuscular (IM) or intravenous (IV) for the first 26 weeks, then every 2 weeks for the next 26 weeks or until disease progression (whichever occurs first)
DRUG	Vinblastine	Vinblastine will be given at a dose of 6mg/m2/week intravenous (IV) for for the first 26 weeks, then every 2 weeks for the next 26 weeks or until disease progression (whichever occurs first). Maximum actual dose may not exceed 10mg.

Timeline

• Start date: 2001-02-01
• Primary completion: 2013-12-01
• Completion: 2016-03-01
• First posted: 2003-01-27
• Last updated: 2018-08-08
• Results posted: 2018-08-08

Locations

1 site across 1 country: United States