Gastrointestinal Stromal Tumors
Conditions
Brief summary
This is an open-label extension study to provide long term safety data for GIST patients who are deriving clinical benefit from avapritinib upon the completion of avapritinib clinical trials.
Interventions
DRUGAvapritinib
avapritinib tablet
Sponsors
Blueprint Medicines Corporation
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patient has histologically confirmed metastatic or unresectable GIST as established by entry in a previous avapritinib clinical trial and has been receiving treatment with avapritinib on one of these trials. 2. Patient continues to receive clinical benefit from avapritinib treatment, as assessed by the investigator. 3. Patient or legal guardian, if permitted by local regulatory authorities, provides informed consent.
Exclusion criteria
1. Patient requires therapy with a concomitant medication that is a strong inhibitor or strong inducer of cytochrome P450 (CYP) 3A4 2. Patient has a history of intracranial bleeding either prior to or during avapritinib treatment 3. Patients who have poor organ function, defined as Adverse Events of NCI CTCAE version 5.0 Grade 3 or higher at the time of enrollment must delay start of treatment until symptoms return to Grade 2 or baseline, or the start of treatment has been approved by the Sponsor. 4. Patients who have ongoing cognitive or mood effects Adverse Events of NCI CTCAE version 5.0 higher than Grade 1 must delay start of treatment until symptoms return to Grade 1 or baseline. 5. Women who are unwilling, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of enrollment and for at least 30 days after the last dose of avapritinib. Men who are unwilling, if not surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of first dose and for at least 90 days after the last dose of avapritinib. 6. Women who are pregnant. 7. Women who are breast feeding.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events of Special Interest (AESIs) | From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months) | AESIs for avapritinib are, regardless of grade or causality: * cognitive effects which include the following terms: memory impairment, cognitive disorder, confusional state and encephalopathy. * intracranial bleeding including haemorrhage intracranial, cerebral haemorrhage , and subdural haematoma. |
| Number of Participants With Serious Adverse Events (SAEs) | From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months) | An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. SAEs were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of all serious AEs, regardless of causality is located in Reported AE section. |
Countries
France
Participant flow
Pre-assignment details
Two participants (one from BLU-285-1101 (NCT02508532) and one from BLU-285-1303 (NCT03465722)) were continuing to receive avapritinib and were enrolled in this extension study.
Participants by arm
| Arm | Count |
|---|---|
| Avapritinib Participants with GIST who were deriving clinical benefit from avapritinib upon the completion of avapritinib clinical studies received avapritinib QD at the specified dose of the prior avapritinib protocol. | 2 |
| Total | 2 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Commercial treatment became available | 1 |
| Overall Study | Disease progression | 1 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 2 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 1 / 2 |
Outcome results
Primary
Number of Participants With Adverse Events of Special Interest (AESIs)
AESIs for avapritinib are, regardless of grade or causality: * cognitive effects which include the following terms: memory impairment, cognitive disorder, confusional state and encephalopathy. * intracranial bleeding including haemorrhage intracranial, cerebral haemorrhage , and subdural haematoma.
Time frame: From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
Population: The Safety Population was defined as all participants who received at least one dose of avapritinib.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Avapritinib | Number of Participants With Adverse Events of Special Interest (AESIs) | 0 Participants |
Primary
Number of Participants With Serious Adverse Events (SAEs)
An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. SAEs were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of all serious AEs, regardless of causality is located in Reported AE section.
Time frame: From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
Population: The Safety Population was defined as all participants who received at least one dose of avapritinib.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Avapritinib | Number of Participants With Serious Adverse Events (SAEs) | 1 Participants |