Clinical Trial: Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas

Study Status: Suspended
Recruit Status: Suspended
Study Type: Interventional

Official Title: A Pilot Study of Sutent®/Sunitinib (SU11248), an Oral Multi-Targeted Tyrosine Kinase Inhibitor in Subjects With NF-1 Plexiform Neurofibromas

Brief Summary: This is a pilot study to determine if adults and children with neurofibromatosis type 1 who have plexiform tumors given Sutent® respond to this drug therapy.

Detailed Summary: This is an open-label pilot study to evaluate the efficacy of Sutent® in individuals with NF1 who have clinically significant plexiform tumors. A secondary goal of this study will be to seek to improve on current and novel tools to evaluate tumor response of plexiform tumors. The rationale for this study arises from the response of human and murine NF1 cells to Sutent® in vitro and the clinical response of individuals with NF1 using a similar drug,Gleevec®. Following enrollment adult subjects will start receiving Sutent® by month at 25mg once a day for 28 days. Subjects will then have 14 days without taking any Sutent®. If tolerated the dose will be increased to 37.5mg and 50mg with the same regimen (28 days of taking medication followed by 14 without). Children will be started on a dose of 10mg/m2/day once a day for 28 days. They will then have 14 days without taking any Sutent®. If tolerated the dose will be increased to 15mg/m2/day.
Sponsor: Indiana University

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

To estimate the disease control rate (SD, PR, CR) with Sutent® in patients with neurofibromas (NF1). Tumor response criteria are determined by changes in size using all 3 dimensional measurements: width (W), transvers (T) , and length (L) measurements. Partial Response: ≥20% decrease in the sum of the products of the three perpendicular diameters of all target lesions (up to 5), taking as reference the initial baseline measurements.Stable Disease (SD): Neither sufficient decrease in the sum of the products of the three perpendicular diameters of all target lesions to qualify for PR (taking as reference the initial baseline measurements), nor sufficient increase in a single target lesions to qualify for PD, (taking as reference the smallest disease measurement since the treatment started).Progressive Disease (PD): 40% or more increase in the product of perpendicular diameters of ANY target lesion, taking as reference the smallest product observed.


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

To estimate the disease control rate (SD, PR, CR) with Sutent® in patients with neurofibromas (NF1).


Current Secondary Outcome: Volumetric Disease Evaluation [ Time Frame: 6 months ]

To determine the response rate with Sutent® in patients with plexiform neurofibromas using volumetric analysis of MRI scans


Original Secondary Outcome: Same as current

Information By: Indiana University

Dates:
Date Received: July 25, 2011
Date Started: March 2012
Date Completion: October 2017
Last Updated: January 27, 2017
Last Verified: January 2017