Clinical Trial: Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 Summary:

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.


Sponsor: Children's Hospital of Philadelphia

Current Primary Outcome: Time to disease progression [ Time Frame: 6 months ]

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.


Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Children's Hospital of Philadelphia

Dates:
Date Received: February 14, 2002
Date Started: February 2001
Date Completion:
Last Updated: June 1, 2016
Last Verified: June 2016