Clinical Trial: Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas

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

Official Title: Pilot Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas

Brief Summary: The purpose of this Pilot Study is to determine if NF1 patients with plexiform neurofibromas treated with Tasgina® respond to therapy.

Detailed Summary: This is an open-label Pilot Study to determine the efficacy of Tasigna® in adults with neurofibromatosis (NF1) and plexiform neurofibromas with the secondary goals of determining the toxicity, and tumor markers in this genetically defined population. The rationale for this study arises from the response of human and murine NF1 cells to Tasigna® in vitro and the clinical response in NF1 patients with plexiform neurofibromas using the similar drug, Gleevec®. Following enrollment each subject will initially receive Tasigna orally at 200 mg twice daily for two weeks. If tolerated, the dose will be increased to 300 mg twice daily after a minimum of two weeks and will be increase to a maximum dose of 400mg twice daily after an additional two weeks if tolerated. Subjects will have his/her dose increased as tolerated dose during the first three months of therapy. The maximum targeted dose is 400mg twice daily.
Sponsor: Indiana University

Current Primary Outcome: Disease Response [ Time Frame: 6 months ]

To estimate the disease control rate (PD,SD, PR, CR) with Tasigna® in patients with neurofibromas (NF1) using standard RECIST criteria. Complete Response (CR) is defined as; disappearance of all target lesions. Partial Response (PR) is defined as at least a 30% decrease in the sum of the longest diameter of target lesions, taking as a reference the baseline sum longest diameter. Stable Disease (SD) is defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum longest diameter since the treatment started. Disease Progression (PD) is defined as at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since the treatment started or the appearance of one or more new lesions.


Original Primary Outcome: Disease Response [ Time Frame: 6 months ]

To estimate the disease control rate (SD, PR, CR) with Tasigna® in patients with neurofibromas (NF1) using standard RECIST criteria.


Current Secondary Outcome:

Original Secondary Outcome: volumetric disease evaluation [ Time Frame: 6 months ]

To determine the response rate with Tasigna® in patients with plexiform neurofibromas (and NF1) using volumetric analysis of MRI scans


Information By: Indiana University

Dates:
Date Received: January 11, 2011
Date Started: January 2011
Date Completion:
Last Updated: April 11, 2017
Last Verified: April 2017