NCT01402817

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

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Indiana University · Academic / Other
• Sex: All
• Age: 3 Years – 65 Years
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Neurofibromatosis
• NF1
• Plexiform Neurofibromas

Interventions

Type	Name	Description
DRUG	Sutent®/Sunitinib	Upon enrollment, subjects will receive Sutent® orally. Adults (Age \>18) will receive 25mg. Children will receive 10mg/m2/day. All subjects will take the daily dose for 28 days followed by a 14 day rest period. If subjects tolerate the initial dose, adults will be increased to 37.5mg and children will be increased to 15mg/m2/day. Again, subjects will take that dose for 28 days followed by a rest period of 14 days. Adults who tolerate the increase will go up to the maximum dose of 50mg. The maximum dose for children is 15mg/m2/day.

Timeline

• Start date: 2012-03-01
• Primary completion: 2015-10-01
• Completion: 2018-02-14
• First posted: 2011-07-26
• Last updated: 2018-03-14
• Results posted: 2016-05-04

Locations

1 site across 1 country: United States