Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas
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.
Neurofibromatosis Type 1
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study|
- Time to disease progression [ Time Frame: 6 months ] [ Designated as safety issue: No ]Tumor response will be assessed by magnetic resonance imaging (MRI) measurement whenever possible or computed tomography (CT) scan and/or tumor measurement during physical examination of palpable lesions.
|Study Start Date:||February 2001|
|Estimated Study Completion Date:||December 2015|
|Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Experimental: Methotrexate & Vinblastine
Methotrexate and Vinblastine will be given once a week for the first 26 weeks and then every two weeks for the next 26 weeks or until disease progression (whichever occurs first).
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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00030264
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Jean B. Belasco, MD||Children's Hospital of Philadelphia|