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    Clinical Trial Results:
    A Phase 1, Randomized, Parallel-group, Open-label, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Vonoprazan (10 or 20 mg Once Daily) in Adolescents With Symptomatic Gastroesophageal Reflux Disease

    Summary
    EudraCT number
    		 2023-000179-10
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    13 Jun 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Dec 2023
    First version publication date
    25 Dec 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VPED-102
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05343364
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Phathom Pharmaceuticals, Inc.
    Sponsor organisation address
    2150 East Lake Cook Road, Suite 800, Buffalo Grove, United States, IL 60089
    Public contact
    Phathom Medical Information, Phathom Pharmaceuticals, Inc., 1 888-775-7428, medicalinformation@phathompharma.com
    Scientific contact
    Phathom Medical Information, Phathom Pharmaceuticals, Inc., 1 888-775-7428, medicalinformation@phathompharma.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002703-PIP01-19
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Jun 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Jun 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study is to evaluate the pharmacokinetic profile of vonoprazan in adolescent participants with symptomatic gastroesophageal reflux disease (GERD).
    Protection of trial subjects
    All aspects of the study were carefully monitored, by the sponsor or its designee, for compliance with applicable government regulation with respect to current good clinical practice and current standard operating procedures.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    09 May 2022
    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
    United States: 24
    Worldwide total number of subjects
    24
    EEA total number of subjects
    0
    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)
    24
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 24 participants were enrolled into this study in the United States between May 2022 and June 2023.

    Pre-assignment
    Screening details
    		 The total duration of the study was up to 8 weeks. The Screening Period was up to 4 weeks, Treatment Period was 2 weeks, and safety follow-up phone call was 2 weeks after last study drug administration.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Vonoprazan 10 mg QD
    Arm description
    		 Participants were randomized to receive vonoprazan 10 mg once daily (QD) from Day 1 to Day 14.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vonoprazan
    Investigational medicinal product code
    TAK-438
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet.

    Arm title
    		 Vonoprazan 20 mg QD
    Arm description
    		 Participants were randomized to receive vonoprazan 20 mg QD from Day 1 to Day 14.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vonoprazan
    Investigational medicinal product code
    TAK-438
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet.

    Number of subjects in period 1
    		Vonoprazan 10 mg QD Vonoprazan 20 mg QD
    Started
    12
    12
    Completed
    12
    11
    Not completed
    0
    1
         Miscellaneous
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Vonoprazan 10 mg QD
    Reporting group description
    		 Participants were randomized to receive vonoprazan 10 mg once daily (QD) from Day 1 to Day 14.

    Reporting group title
    Vonoprazan 20 mg QD
    Reporting group description
    		 Participants were randomized to receive vonoprazan 20 mg QD from Day 1 to Day 14.

    Reporting group values
    		 Vonoprazan 10 mg QD Vonoprazan 20 mg QD Total
    Number of subjects
    		 12 12 24
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 14.3 ± 1.78 15.2 ± 1.75 -
    Gender categorical
    Units: Subjects
        Female
    		 6 7 13
        Male
    		 6 5 11
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 4 2 6
        Not Hispanic or Latino
    		 6 10 16
        Unknown or Not Reported
    		 2 0 2
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 3 5 8
        White
    		 9 7 16
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    Vonoprazan 10 mg QD
    Reporting group description
    		 Participants were randomized to receive vonoprazan 10 mg once daily (QD) from Day 1 to Day 14.

    Reporting group title
    Vonoprazan 20 mg QD
    Reporting group description
    		 Participants were randomized to receive vonoprazan 20 mg QD from Day 1 to Day 14.

    Primary: Maximum Observed Drug Concentration at Steady State (Cmax-ss) of Vonoprazan

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    End point title
    		 Maximum Observed Drug Concentration at Steady State (Cmax-ss) of Vonoprazan [1]
    End point description
    Plasma pharmacokinetic (PK) parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. PK set: inclusive of all evaluable participants who had at least one measurable concentration result.
    End point type
    Primary
    End point timeframe
    Blood samples were collected predose, once between 0.5 and 2 hours, and once between 2.5 and 4 hours post-dose on Days 7 and 14
    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: No additional statistical analyses were pre-specified for this endpoint.
    End point values
    		 Vonoprazan 10 mg QD Vonoprazan 20 mg QD
    Number of subjects analysed
    9
    8
    Units: ng/mL
        arithmetic mean (full range (min-max))
    13.4 (6.57 to 25.6)
    26.7 (13.1 to 49.1)
    No statistical analyses for this end point

    Primary: Area Under the Plasma Concentration-time Curve During the Dosing Interval τ (AUCτ) of Vonoprazan

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    End point title
    		 Area Under the Plasma Concentration-time Curve During the Dosing Interval τ (AUCτ) of Vonoprazan [2]
    End point description
    PK parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. PK set: inclusive of all evaluable participants who had at least one measurable concentration result.
    End point type
    Primary
    End point timeframe
    Blood samples were collected predose, once between 0.5 and 2 hours, and once between 2.5 and 4 hours post-dose on Days 7 and 14
    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: No additional statistical analyses were pre-specified for this endpoint.
    End point values
    		 Vonoprazan 10 mg QD Vonoprazan 20 mg QD
    Number of subjects analysed
    9
    8
    Units: h*ng/mL
        arithmetic mean (full range (min-max))
    94.6 (56.0 to 165)
    208 (95.6 to 381)
    No statistical analyses for this end point

    Primary: Apparent Oral Clearance (CL) of Vonoprazan

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    End point title
    		 Apparent Oral Clearance (CL) of Vonoprazan [3]
    End point description
    PK parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. PK set: inclusive of all evaluable participants who had at least one measurable concentration result.
    End point type
    Primary
    End point timeframe
    Blood samples were collected predose, once between 0.5 and 2 hours, and once between 2.5 and 4 hours post-dose on Days 7 and 14
    Notes
    [3] - 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: No additional statistical analyses were pre-specified for this endpoint.
    End point values
    		 Vonoprazan 10 mg QD Vonoprazan 20 mg QD
    Number of subjects analysed
    9
    8
    Units: L/h
        arithmetic mean (full range (min-max))
    121 (49.5 to 182)
    133 (73.3 to 209)
    No statistical analyses for this end point

    Primary: Central Volume of Distribution (Vc) of Vonoprazan

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    End point title
    		 Central Volume of Distribution (Vc) of Vonoprazan [4]
    End point description
    PK parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. PK set: inclusive of all evaluable participants who had at least one measurable concentration result.
    End point type
    Primary
    End point timeframe
    Blood samples were collected predose, once between 0.5 and 2 hours, and once between 2.5 and 4 hours post-dose on Days 7 and 14
    Notes
    [4] - 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: No additional statistical analyses were pre-specified for this endpoint.
    End point values
    		 Vonoprazan 10 mg QD Vonoprazan 20 mg QD
    Number of subjects analysed
    9
    8
    Units: liters
        arithmetic mean (full range (min-max))
    661 (357 to 1040)
    799 (435 to 1110)
    No statistical analyses for this end point

    Secondary: Number of Participants Experiencing Adverse Events (AEs)

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    End point title
    		 Number of Participants Experiencing Adverse Events (AEs)
    End point description
    AE: any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug. Treatment-emergent adverse event (TEAE): any AE that occurred after the first dose of study drug or at baseline that worsens in either intensity or frequency after the first dose of study drug. Serious AE: any AE for which the following occurred: death, was life threatening, hopsitalization or prolongation of hospitalization, persistent or significant disability/incapacity, a congenital anomaly/birth defect, or the AE was deemed an important medical event. Related AE: any AE that follows a reasonable temporal sequence from administration of study drug, or for which possible involvement of the drug cannot be ruled out, although factors other than the study drug may also be responsible. Clinically significant changes from baseline in laboratory test values, (hematology, serum chemistry and urinalysis), electrocardiograms (ECG) and vital signs were reported as AEs.
    End point type
    Secondary
    End point timeframe
    Up to Day 28
    End point values
    		 Vonoprazan 10 mg QD Vonoprazan 20 mg QD
    Number of subjects analysed
    12 [5]
    12 [6]
    Units: participants
        Any TEAEs
    4
    1
        Any Serious TEAEs
    0
    0
        Any Study Drug-related TEAEs
    0
    0
        Any Serious Study Drug-related TEAEs
    0
    0
        Any TEAE Leading to Treatment Discontinuation
    0
    0
        Any TEAE Leading to Study Discontinuation
    0
    0
        Any AEs Leading to Death
    0
    0
    Notes
    [5] - Safety Set: inclusive of all participants who received at least 1 dose of study drug.
    [6] - Safety Set: inclusive of all participants who received at least 1 dose of study drug.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to Day 28
    Adverse event reporting additional description
    Safety Set: inclusive of all participants who received at least 1 dose of study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    Vonoprazan 10 mg QD
    Reporting group description
    		 Participants were randomized to receive vonoprazan 10 mg QD from Day 1 to Day 14.

    Reporting group title
    Vonoprazan 20 mg QD
    Reporting group description
    		 Participants were randomized to receive vonoprazan 20 mg QD from Day 1 to Day 14.

    Serious adverse events
    		 Vonoprazan 10 mg QD Vonoprazan 20 mg QD
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Vonoprazan 10 mg QD Vonoprazan 20 mg QD
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 12 (33.33%)
    1 / 12 (8.33%)
    Nervous system disorders
    Migraine
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Enterocolitis infectious
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 Apr 2022
    Reasons for amendment: - To clarify that participants must be aged 12 to 17 years, inclusive, at time of informed consent and throughout study participation. - To clarify that only select sites will perform gastric pH monitoring in participants deemed clinically indicated by the principal investigator. - To add that all study drug doses be taken on an empty stomach. - To add that the time and content of any meals consumed prior to taking study drug on Day 6 or Day 7 be recorded. - To add details for Day 7 dosing with respect to allowed water and meals. - To clarify that Day -1 assessments are only required for participants undergoing gastric pH monitoring. - To clarify that Day 6 is an optional visit for participants that check in to the clinic in the evening before starting Day 7 assessments. - Remove the Day 14 PK blood collection. - Remove morphology from electrocardiogram assessments.
    14 Sep 2022
    Reasons for amendment: - Increased number of sites from 5 to 12. - Updated information about vonoprazan. - Removed duplicate entry for sucralfate under excluded medications and treatments. - Removed gastrin and pepsinogen I and II assessments. - Removed optional Day 6 visit. - Modified strategy to estimate PK parameters to a population based approach; as a result adjusted PK sampling schedule on Day 7, added PK sampling on Day 14, provided information on when time of dosing and meals should be collected and remove Tmax,ss and t1/2z PK parameters. - Removed required overnight stay for PK sampling due to revised sampling schedule. - Added details to the 24-hour gastric pH monitoring. - Moved symptom assessments from Day 8 to Day 7. - Updated Schedule of Events and Study Design to reflect above changes.

    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
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