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Clinical Trials/NCT03465722
NCT03465722
Completed
Phase 3

An International, Multicenter, Open-label, Randomized, Phase 3 Study of BLU-285 vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic Gastrointestinal Stromal Tumor (GIST)

Blueprint Medicines Corporation112 sites in 6 countries476 target enrollmentMarch 26, 2018

Overview

Phase
Phase 3
Intervention
avapritinib
Conditions
GIST
Sponsor
Blueprint Medicines Corporation
Enrollment
476
Locations
112
Primary Endpoint
Efficacy of Avapritinib Based on Progression-free Survival (PFS) Determined by Central Radiological Assessment Per Modified Response Evaluation Criteria in Solid Tumors (mRECIST), Version 1.1
Status
Completed
Last Updated
3 years ago

Overview

Brief Summary

This is an open-label, randomized, Phase 3 study in patients with locally advanced unresectable or metastatic GIST (advanced GIST) of avapritinib (also known as BLU-285) versus regorafenib in patients previously treated with imatinib and 1 or 2 other TKIs.

Registry
clinicaltrials.gov
Start Date
March 26, 2018
End Date
September 15, 2021
Last Updated
3 years ago
Study Type
Interventional
Study Design
Parallel
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Patients who are ≥ 18 years of age.
  • Patients who have histologically confirmed metastatic or unresectable GIST.
  • Patients who received imatinib and 1 or 2 other TKIs as prior treatment regimens. Patients who experienced intolerance to prior therapies must have objective disease progression prior to enrollment onto BLU-285-1303 study.
  • Patients who have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 1.

Exclusion Criteria

  • Patients who have received prior treatment with avapritinib or regorafenib.
  • Patients who have previously received more than 3 different TKI treatment regimens.
  • Patients who are known to be both V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog (KIT) and platelet-derived growth factor receptor alpha (PDGRFα) wild type.
  • Patients who received any systemic anticancer therapy within 1 week before the first dose of study drug.
  • Patients who have clinically significant cardiovascular disease
  • Patients have experienced arterial thrombotic or embolic events within 6 months before the first dose of study drug, or venous thrombotic events within 14 days of the first dose of study drug
  • Patients who have experienced any hemorrhage or bleeding event NCI CTCAE version 5.0 Grade 3 or higher within 4 weeks before the first dose of study drug
  • Patients who have a known risk of intracranial bleeding, or a history of intracranial bleeding within 1 year prior to the first dose of study drug
  • Patients who have a symptomatic non-healing wound, ulcer, gastrointestinal perforation, or bone fracture.
  • Patients who have poor organ function as defined by laboratory parameters specified in the protocol.

Arms & Interventions

avapritinib

300 mg PO QD

Intervention: avapritinib

regorafenib

160 mg PO QD

Intervention: regorafenib

Outcomes

Primary Outcomes

Efficacy of Avapritinib Based on Progression-free Survival (PFS) Determined by Central Radiological Assessment Per Modified Response Evaluation Criteria in Solid Tumors (mRECIST), Version 1.1

Time Frame: 24 Months

To demonstrate the efficacy of avapritinib based on progression-free survival (PFS) determined by central radiological assessment per modified Response Evaluation Criteria in Solid Tumors (mRECIST), version 1.1 in patients with advanced GIST following 2 or 3 regimens of prior treatment with a tyrosine kinase inhibitor (TKI), including imatinib, compared to patients treated with regorafenib. A progressively growing tumor must meet the following criteria: a) the target lesions must be greater or equal to 2cm in size and be a new GIST active lesion or b) the target lesions must be expanding on at least 2 sequential imaging studies.

Secondary Outcomes

  • Objective Response Rate (ORR) Determined by Central Radiology Assessment Per mRECIST, Version 1.1(24 Months)
  • Overall Survival (OS) in Patients With Advanced GIST Treated With Avapritinib Compared to Patients Treated With Regorafenib(24 Months)
  • European Organisation for Research and Treatment of Cancer Quality of Life (EORTC-QLQ-30). Change in Individual Scores in Patients With Advanced GIST Treated With Avapritinib Compared to Patients Treated With Regorafenib(Difference between baseline and week 12 of treatment)

Study Sites (112)

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