Phase III study of Regorafenib as a 3rd line or > treatment for GIST

• Research type

  Research Study

• Full title

  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

• IRAS ID

  66041

• Contact name

  Anne Thomas

• Sponsor organisation

  Bayer Healthcare AG

• Eudract number

  2009-017957-37

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research 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 TUMOURS (GIST) WHOSE DISEASE HAS PROGRESSED DESPITE PRIOR TREATMENT WITH AT LEAST IMATINIB AND SUNITINIB Imatinib, and upon imatinib failure, sunitinib are currently approved for the treatment of metastatic and/or unresectable GISTs. Eventually these therapies fail due development of resistance by the tumour. New agents like regorafenib may open new treatment avenues by potentially overcoming the mutation induced resistance through binding to structurally different mutant kinases. The primary objective of this phase III study is to compare the treatment groups in terms of Progression-Free Survival (PFS), per blinded central radiology review, according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Approximately 170 patients are planned to participate in this study in approximately 21 countries. Participants will be randomly assigned to either the regorafenib or placebo, in a 2:1 ratio. Participants randomised into the study will take four 40 mg (160mg) tablets of regorafenib or placebo once daily in the morning for three weeks and then have one week of rest. Participants who have received placebo may be offered open-label regorafenib after objective tumour progression. In addition to taking either regorafenib or the placebo the participants will have best supportive care, which includes any method to preserve the comfort and dignity of participants (excluding chemotherapy, radiotherapy and surgical intervention). General health and research related blood tests, urine tests, ECGs and scans will be performed in order to assess the participants?? health and the efficacy of the study drug. Tumour assessments will be performed regularly until objective tumour progression. Participants are expected to be in this study for approximately one year depending on how they respond to the treatment. This trial is being sponsored by Bayer Healthcare.

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  10/H0408/111

• Date of REC Opinion

  1 Mar 2011

• REC opinion

  Further Information Favourable Opinion