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)
- Conditions
- Gastrointestinal stromal tumorsarcomas of soft tissue in the gastrointestinal tract10017991
- Registration Number
- NL-OMON48964
- Lead Sponsor
- Blueprint Medicines Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 15
1. Patients who are * 18 years of age.
2. Patients who have GIST, which is histologically confirmed metastatic and/or
unresectable (confirmed to be unresectable by a qualified surgeon).
3. Patients who received imatinib and 1 or 2 other TKIs for the treatment of
GIST, including TKIs used for adjuvant therapy. Each different TKI is counted
once regardless of how often it was used, and if two different TKIs are used in
combination, both TKIs are counted. Patients must have disease progression
prior to enrollment. Prior use of other systemic and local therapies is not
restricted.
4. Patients who have an ECOG PS of 0 to 1.
5. Patients, or legal guardian if permitted by local regulatory authorities,
who provides informed consent to participate in the study.
1. Patients who have received prior treatment with avapritinib or regorafenib.
2. Patients who have received more than 3 different TKIs for the treatment of
GIST, including TKIs used for adjuvant therapy. Each different TKI is counted
once regardless of how often it was used, and if two different TKIs are used in
combination, both TKIs are counted.
3. Patients who are known to be both KIT and PDGRF* wild type.
4. Patients who received any systemic anticancer therapy within 1 week before
the first dose of study drug. Prior radiotherapy (including stereotactic
radiotherapy) to major organs within 2 weeks of the first dose of study drug,
or focal radiotherapy, (including stereotactic radiotherapy), such as to
bones, limbs, or other areas not involving major organs, within 3 days.
5. Patients who have clinically significant, uncontrolled, cardiovascular
disease, including congestive heart failure Grades II, III or IV according to
the New York Heart Association classification, myocardial infarction or
unstable angina within the previous 6 months, or uncontrolled
hypertension.
6. Patients who have experienced arterial thrombotic or embolic events such as
cerebrovascular accident within 6 months before the first dose of study drug,
or venous thrombotic events such as pulmonary embolism within the 14 days
before the first dose of study drug or deep vein thrombosis within 14 days
before the first dose of study drug. Patients with venous thrombotic events
such as pulmonary embolism or deep vein thrombosis * 14 days before the first
dose of study drug are not excluded provided they are on stabel dosis of
anticoagulation, or have completed the planned coagulation regimen.
7. 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.
8. Patients who have a known risk of intracranial bleeding, such as a brain
aneurysm that has not been removed or repaired, or history of intracranial
bleeding within one year prior to randomization.
9. Patients who have a symptomatic non-healing wound, ulcer, gastrointestinal
perforation, or bone fracture.
10. Patients who have poor organ function as defined by one or more laboratory
parameters, as described in the protocol.
11. Patients who have received neutrophil growth factor support within 14 days
of the first dose of study drug.
12. Patients who require therapy with a concomitant medication that is a strong
inhibitor or strong or moderate inducer of CYP3A4.
13. Patients who have had a major surgical procedure within 14 days of the
first dose of study drug. Patient has significant traumatic injury within 28
days before the first dose of study drug.
14. Patients who have a history of another primary malignancy that has been
diagnosed or required therapy within 3 years before the first dose of study
drug. The following prior malignancies are not exclusionary: completely
resected basal cell and squamous cell skin cancer, curatively treated localized
prostate cancer, and completely resected carcinoma in situ of any site.
Patients with a prior or concurrent malignancy whose natural history or
treatment does not have the potential to interfere with the safety or efficacy
assessment of the investigational agent may be included after approval by
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint is PFS, based on central radiological assessment per<br /><br>mRECIST, version 1.1, in patients with advanced GIST. Progression-free survival<br /><br>is defined as time from randomization to disease progression, or death due to<br /><br>any cause, whichever occurs first.</p><br>
- Secondary Outcome Measures
Name Time Method <p>The key secondary endpoints are:<br /><br>* Objective response rate defined as the percentage of patients whose best<br /><br>response is CR or PR as assessed by central radiology using mRECIST, version<br /><br>1.1.<br /><br>* Overall survival defined as the time from date of randomization to death due<br /><br>to any cause.</p><br>