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Brief Title: Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas
Official Title: Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study
Study ID: NCT00030264
Brief 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.
Minimum Age:
Eligible Ages: CHILD, ADULT
Sex: ALL
Healthy Volunteers: No
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
Name: Jean B. Belasco, MD
Affiliation: Children's Hospital of Philadelphia
Role: PRINCIPAL_INVESTIGATOR