NCT01048723

Study of RAD001 in Soft Tissue Extremity and/or Retroperitoneal Sarcomas

A Single-arm, Open Label Phase II Study of RAD001 in Soft Tissue Extremity and/or Retroperitoneal Sarcomas

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: H. Lee Moffitt Cancer Center and Research Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical research study is to learn if the study drug RAD001 can stop or slow the growth of resectable soft tissue sarcoma. The patient's physical state, their symptoms, changes in the size of the tumor, and laboratory findings obtained while on-study will help the research team decide if RAD001 is safe and effective in patients with this condition. The study drug, RAD001, is made by Novartis Pharmaceuticals Corporation.

Detailed description

A single-arm, open label, proof of principle phase II study exploring the efficacy of RAD001 in resectable soft tissue sarcomas either in the extremities, trunk or retroperitoneum. Patients with resectable sarcomas as detailed below were to have a core biopsy for molecular markers prior to therapy with RAD001 10mg PO daily x 2 weeks. Within 7-14 days of the end of therapy with RAD001, the patients were to be brought to surgery for definitive resection or, should they be candidates for neoadjuvant radiation, would have 6 16 gauge core biopsies taken percutaneously or using image guidance. Pharmacodynamic markers as detailed in the objectives were to be assessed in the laboratory. Patients were to be numbered sequentially from 1 to 40, or more if there were patients that dropped out due to drug toxicity, with the goal of achieving 40 patients in accrual for evaluation of the pre- and post-treatment tumor samples for Pharmacodynamic assays.

Conditions

• Sarcoma

Interventions

Type	Name	Description
DRUG	RAD001	Patients were instructed to take RAD001 in the morning, at the same time each day.

Timeline

• Start date: 2010-01-01
• Primary completion: 2011-10-01
• Completion: 2011-10-01
• First posted: 2010-01-14
• Last updated: 2013-12-16
• Results posted: 2012-08-17

Locations

1 site across 1 country: United States