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    Clinical Trial Results:
    Open-Label Extension Study to Evaluate the Safety of Long-Term Treatment with Avapritinib for Patients Previously Involved in an Avapritinib Study

    Summary
    EudraCT number
    		 2020-005751-21
    Trial protocol
    		 FR  
    Global end of trial date
    23 Nov 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    01 Dec 2024
    First version publication date
    01 Dec 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    BLU-285-1408
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04825574
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Blueprint Medicines
    Sponsor organisation address
    45 Sidney Street, Cambridge, United States, 02139
    Public contact
    Blueprint Medicines Medical Information, Blueprint Medicines, 1 18882587768, medinfo@blueprintmedicines.com
    Scientific contact
    Blueprint Medicines Medical Information, Blueprint Medicines, 1 18882587768, medinfo@blueprintmedicines.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    23 Nov 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Nov 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This was an open-label extension study to provide long term safety data for GIST patients who were deriving clinical benefit from avapritinib upon the completion of avapritinib clinical trials.
    Protection of trial subjects
    The study was conducted in accordance with ethical principles founded in the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    21 May 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 2
    Worldwide total number of subjects
    2
    EEA total number of subjects
    2
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    2
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening 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.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Avapritinib
    Arm description
    		 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.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Avapritinib
    Investigational medicinal product code
    Other name
    BLU-285
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Participants received avapritinib QD at the specified dose of the prior avapritinib protocol.

    Number of subjects in period 1
    		Avapritinib
    Started
    2
    Received at least 1 dose of study drug
    2
    Completed
    0
    Not completed
    2
         Disease progression
    1
         Commercial treatment became available
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Avapritinib
    Reporting group description
    		 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.

    Reporting group values
    		 Avapritinib Total
    Number of subjects
    		 2 2
    Age Categorical
    Units: participants
        <=18 years
    		 0 0
        Between 18 and 65 years
    		 2 2
        >=65 years
    		 0 0
    Sex: Female, Male
    Units: participants
        Not Reported for Participant Privacy
    		 2 2
    Race (NIH/OMB)
    Units: Subjects
        Not Reported for Participant Privacy
    		 2 2

    End points

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    End points reporting groups
    Reporting group title
    Avapritinib
    Reporting group description
    		 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.

    Primary: Number of Participants with Adverse Events of Special Interest (AESIs)

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    End point title
    		 Number of Participants with Adverse Events of Special Interest (AESIs) [1]
    End point description
    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. The Safety Population was defined as all participants who received at least one dose of avapritinib.
    End point type
    Primary
    End point timeframe
    From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only summary statistics were planned for this outcome measure.
    End point values
    		 Avapritinib
    Number of subjects analysed
    2
    Units: participants
    0
    No statistical analyses for this end point

    Primary: Number of Participants with Serious Adverse Events (SAEs)

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    End point title
    		 Number of Participants with Serious Adverse Events (SAEs) [2]
    End point description
    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. The Safety Population was defined as all participants who received at least one dose of avapritinib.
    End point type
    Primary
    End point timeframe
    From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only summary statistics were planned for this outcome measure.
    End point values
    		 Avapritinib
    Number of subjects analysed
    2
    Units: participants
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    From date of first avapritinib dose through 30 days after the last avapritinib dose (up to approximately 31 months)
    Adverse event reporting additional description
    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 were not collected.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Avapritinib
    Reporting group description
    		 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.

    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: As specified in the protocol, only SAEs and AESIs were collected. Non-serious adverse event data was not collected.
    Serious adverse events
    		 Avapritinib
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 2 (50.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Gastrointestinal disorders
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Abdominal pain
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Avapritinib
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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