Trial Outcomes & Findings for Study for Patients Previously Treated in Avapritinib Clinical Trials (NCT NCT04825574)

NCT ID: NCT04825574

Last Updated: 2024-11-26

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

2 participants

Primary outcome timeframe

From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)

Results posted on

2024-11-26

Participant Flow

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.

Participant milestones

Participant milestones
Measure
Avapritinib
Participants with gastrointestinal stromal tumor (GIST) who were deriving clinical benefit from avapritinib upon the completion of avapritinib clinical studies received avapritinib once daily (QD) at the specified dose of the prior avapritinib protocol.
Overall Study
STARTED
2
Overall Study
Received at Least 1 Dose of Study Drug
2
Overall Study
COMPLETED
0
Overall Study
NOT COMPLETED
2

Reasons for withdrawal

Reasons for withdrawal
Measure
Avapritinib
Participants with gastrointestinal stromal tumor (GIST) who were deriving clinical benefit from avapritinib upon the completion of avapritinib clinical studies received avapritinib once daily (QD) at the specified dose of the prior avapritinib protocol.
Overall Study
Commercial treatment became available
1
Overall Study
Disease progression
1

Baseline Characteristics

Study for Patients Previously Treated in Avapritinib Clinical Trials

Baseline characteristics by cohort

Baseline data not reported

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
Avapritinib
n=2 Participants
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.
Number of Participants With Adverse Events of Special Interest (AESIs)
0 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
Avapritinib
n=2 Participants
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.
Number of Participants With Serious Adverse Events (SAEs)
1 Participants

Adverse Events

Avapritinib

Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Avapritinib
n=2 participants at risk
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.
Gastrointestinal disorders
Gastrointestinal haemorrhage
50.0%
1/2 • Number of events 1 • From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
The Safety Population was defined as all participants who received at least one dose of avapritinib. As specified in the protocol, only SAEs and AESIs were collected. Non-serious adverse event data was not collected.
Musculoskeletal and connective tissue disorders
Arthralgia
50.0%
1/2 • Number of events 1 • From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
The Safety Population was defined as all participants who received at least one dose of avapritinib. As specified in the protocol, only SAEs and AESIs were collected. Non-serious adverse event data was not collected.
Gastrointestinal disorders
Abdominal pain
50.0%
1/2 • Number of events 2 • From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
The Safety Population was defined as all participants who received at least one dose of avapritinib. As specified in the protocol, only SAEs and AESIs were collected. Non-serious adverse event data was not collected.
Gastrointestinal disorders
Vomiting
50.0%
1/2 • Number of events 1 • From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
The Safety Population was defined as all participants who received at least one dose of avapritinib. As specified in the protocol, only SAEs and AESIs were collected. Non-serious adverse event data was not collected.

Other adverse events

Adverse event data not reported

Additional Information

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Blueprint Medicines

Phone: 1-888-258-7768

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: OTHER