NCT01271712

Study of Regorafenib as a 3rd-line or Beyond Treatment for Gastrointestinal Stromal Tumors (GIST)

A Randomized, Double-blind, Placebo-controlled Phase III Study of Regorafenib Plus Best Supportive Care Versus Placebo Plus Best Supportive Care for Subjects With Metastatic and/or Unresectable Gastrointestinal Stromal Tumors (GIST) Whose Disease Has Progressed Despite Prior Treatment With at Least Imatinib and Sunitinib

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 199 (actual)
• Sponsor: Bayer · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

A randomized, double-blind, placebo-controlled phase III study of regorafenib plus best supportive care versus placebo plus best supportive care for subjects with metastatic and/or unresectable gastrointestinal stromal tumors (GIST) whose disease has progressed despite prior treatment with at least imatinib and sunitinib. The study is composed of 3 periods: A Screening Period, a Treatment Period, and a Survival Follow up Period. Subjects randomized to be treated with regorafenib will receive 160 mg po od for 3 weeks of every 4 week (28 day) cycle (ie, 3 weeks on/1 week off). In addition subjects will receive best supportive care which excludes any disease specific anti cancer therapy such as any kinase inhibitor, chemotherapy, radiation therapy, or surgery. Tumor assessment will be every 4 weeks for the first 3 months, every 6 weeks for the next 3 months (through month 6), and every 8 weeks until the end of treatment, or more frequently if clinically indicated. Tumor assessments include CT or MRI and will be performed until tumor progression is seen in a central radiology review. Subjects receiving placebo who experience disease progression may be offered active treatment. Subjects who experience progression during regorafenib treatment may continue open label treatment. All subjects will enter the Survival Follow-up Period upon discontinuation of randomized study treatment.

Conditions

• Gastrointestinal Stromal Tumors

Interventions

Type	Name	Description
DRUG	Regorafenib (Stivarga, BAY73-4506)	160 mg po once daily (od), 3 weeks on/1 week off. Route of administration: oral
DRUG	Placebo	once daily (od), 3 weeks on/1 week off. Route of administration: oral
DRUG	Best supportive care	Best supportive care includes any method to preserve the comfort and dignity of the patients, and excludes any disease-specific anti-neoplastic therapy such as any kinase inhibitor, chemotherapy, radiation therapy, or surgical intervention.

Timeline

• Start date: 2011-01-04
• Primary completion: 2012-01-26
• Completion: 2019-04-15
• First posted: 2011-01-07
• Last updated: 2021-01-29
• Results posted: 2013-10-25

Locations

57 sites across 17 countries: United States, Austria, Belgium, Canada, China, Finland, France, Germany, Israel, Italy, Japan, Netherlands, Poland, Singapore, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study