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    Clinical Trial Results:
    Assessing the Pharmacokinetics, Safety, Tolerability and Efficacy of Continuous Oral Levodopa via the DopaFuse Delivery System in Parkinson’s Disease Patients

    Summary
    EudraCT number
    		 2020-003372-41
    Trial protocol
    		 ES   IT   LU  
    Global end of trial date
    02 Aug 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    22 Sep 2023
    First version publication date
    22 Sep 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    TP-0007
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    SynAgile Corporation
    Sponsor organisation address
    628 river oaks parkway, San Jose, United States, CA
    Public contact
    Clinical Affairs, SynAgile Corporation, clinical@synagile.com
    Scientific contact
    Clinical Affairs, SynAgile Corporation, clinical@synagile.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
    29 Jun 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    02 Aug 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Aug 2022
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Pharmacokinetics - To compare plasma levodopa variability between intermittent vs. DopaFuse delivery system Safety & Tolerability - To assess the safety and tolerability of DopaFuse delivery system Efficacy - To assess the effect of DopaFuse delivery system on OFF time
    Protection of trial subjects
    The Investigator could not deviate from the protocol without a formal protocol amendment having been established and approved by an appropriate IRB/IEC, except when necessary to eliminate immediate hazards to the patient or when the change involved only logistical or administrative aspects of the study. Rescue medication in the form of rescue doses of apomorphine (Days 1-3) or additional standard oral LD/CD, inhaled LD/CD, or other medications routinely used by the patient to treat clinically significant OFF (Days 4-15), could be introduced at the judgment of the Investigator.
    Background therapy
    		 For this study, a patient’s standard LD/CD regimen was considered background treatment.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    12 Oct 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
    Italy: 6
    Country: Number of subjects enrolled
    Luxembourg: 7
    Country: Number of subjects enrolled
    Spain: 4
    Worldwide total number of subjects
    17
    EEA total number of subjects
    17
    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)
    11
    From 65 to 84 years
    6
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Patients with Parkinson's Disease on stable LD/CD treatment who had experienced at least one hour of OFF time per day were invited to participate in the study. A qualified dentist assessed the patients’ oral health and fit the retainers, which were worn without medication at home for 1-2 days to confirm tolerability prior to receiving IMP.

    Pre-assignment
    Screening details
    		 During screening, patients were evaluated for eligibility according to the protocol defined inclusion and exclusion criteria. Eligibility had to be approved by an Enrollment Authorization Committee prior to enrollment.

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

    Arms
    Arm title
    		 Arm 1 - Single group study
    Arm description
    		 Single group study
    Arm type
    		 Experimental

    Investigational medicinal product name
    DopaFuse Delivery System
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral paste
    Routes of administration
    Oral use
    Dosage and administration details
    DopaFuse treatment regimens were individualized in order to closely align with the patient’s usual levodopa/carbidopa (LD/CD) dosing quantities and timetable. LD/CD was given at a 4:1 ratio using one of two flow rates, which were converted to reflect usual oral LD dosing for the 12-hour study window.

    Number of subjects in period 1 [1]
    		Arm 1 - Single group study
    Started
    16
    Completed
    16
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: One patient was enrolled in the study but not treated due to an adverse event (vomiting during the wearing of the retainer) and was not included in the baseline period or subsequent analysis.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    		 Patients who were dosed at least once during the study

    Reporting group values
    		 Overall Study Total
    Number of subjects
    		 16 16
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 10 10
        From 65-84 years
    		 6 6
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 63.2 ± 9.1 -
    Gender categorical
    Units: Subjects
        Female
    		 7 7
        Male
    		 9 9
    Cognitive status:
    Units: Subjects
        Mini–Mental State Examination 30
    		 8 8
        Mini–Mental State Examination 29
    		 4 4
        Mini–Mental State Examination 28
    		 4 4
        Mini–Mental State Examination 27
    		 0 0
        Mini–Mental State Examination 26
    		 0 0
        Mini–Mental State Examination < 26
    		 0 0
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 1 1
        Not Hispanic or Latino
    		 12 12
        Not reported / Unknown
    		 3 3
    Race
    Units: Subjects
        White
    		 16 16
    Weight
    Units: kg
        arithmetic mean (standard deviation)
    		 70.9 ± 14.8 -
    Body Mass Index
    Units: kg/m2
        arithmetic mean (standard deviation)
    		 25.2 ± 4.3 -
    Height
    Units: cm
        arithmetic mean (standard deviation)
    		 167.5 ± 8.8 -

    End points

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    End points reporting groups
    Reporting group title
    Arm 1 - Single group study
    Reporting group description
    		 Single group study

    Primary: Variability in plasma concentration of levodopa

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    End point title
    		 Variability in plasma concentration of levodopa [1]
    End point description
    Variability in levodopa concentrations assessed by levodopa fluctuation index (FI) at 4-12 hours (steady state) with evaluations between Day 2 (DopaFuse alone) and Day 1 (oral LD/CD)
    End point type
    Primary
    End point timeframe
    4-12 hours (steady state) for Day 2 (DopaFuse alone) vs. Day 1 (oral LD/CD)
    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: Single arm study - statistical analysis not reported in accordance with EudraCT & EU CTR Frequently asked questions (EMA/370102/2016) - Question 83
    End point values
    		 Arm 1 - Single group study
    Number of subjects analysed
    16
    Units: Levodopa Fluctuation Index (FI)
    arithmetic mean (standard deviation)
        Day 2 (DopaFuse alone)
    1.50 ± 0.55
        Day 1 (Oral LD/CD)
    2.15 ± 0.59
    No statistical analyses for this end point

    Primary: Variability in plasma concentration of levodopa

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    End point title
    		 Variability in plasma concentration of levodopa [2]
    End point description
    Variability in levodopa concentrations assessed by levodopa fluctuation index (FI) at 4-12 hours (steady state) with evaluations between Day 3 (oral LD/CD morning dose + DopaFuse) and Day 1 (oral LD/CD)
    End point type
    Primary
    End point timeframe
    At 4-12 hours (steady state) for Day 3 (oral LD/CD morning dose + DopaFuse) vs. Day 1 (oral LD/CD)
    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: Single arm study - statistical analysis not reported in accordance with EudraCT & EU CTR Frequently asked questions (EMA/370102/2016) - Question 83
    End point values
    		 Arm 1 - Single group study
    Number of subjects analysed
    16
    Units: Levodopa Fluctuation index (FI)
    arithmetic mean (standard deviation)
        Day 3 (DopaFuse + LD/CD morning dose)
    1.03 ± 0.53
        Day 1 (Oral LD/CD)
    1.84 ± 0.63
    No statistical analyses for this end point

    Primary: Safety and Tolerability

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    End point title
    		 Safety and Tolerability [3]
    End point description
    Number of Treatment-emergent adverse events (TEAEs)
    End point type
    Primary
    End point timeframe
    Day 1 to Day 29
    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: Single arm study - statistical analysis not reported in accordance with EudraCT & EU CTR Frequently asked questions (EMA/370102/2016) - Question 83
    End point values
    		 Arm 1 - Single group study
    Number of subjects analysed
    16
    Units: Treatment Emergent Adverse Events (TEAE)
    19
    No statistical analyses for this end point

    Primary: Safety and Tolerability

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    End point title
    		 Safety and Tolerability [4]
    End point description
    Number of Serious Adverse Events (SAEs)
    End point type
    Primary
    End point timeframe
    Day 1 to Day 29
    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: Single arm study - statistical analysis not reported in accordance with EudraCT & EU CTR Frequently asked questions (EMA/370102/2016) - Question 83
    End point values
    		 Arm 1 - Single group study
    Number of subjects analysed
    16
    Units: Serious adverse events (SAEs)
    0
    No statistical analyses for this end point

    Primary: Safety and Tolerability

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    End point title
    		 Safety and Tolerability [5]
    End point description
    Number of TEAEs leading to study discontinuation
    End point type
    Primary
    End point timeframe
    Day 1 to Day 29
    Notes
    [5] - 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: Single arm study - statistical analysis not reported in accordance with EudraCT & EU CTR Frequently asked questions (EMA/370102/2016) - Question 83
    End point values
    		 Arm 1 - Single group study
    Number of subjects analysed
    16
    Units: TEAEs leading to discontinuation
    0
    No statistical analyses for this end point

    Primary: Safety and Tolerability

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    End point title
    		 Safety and Tolerability [6]
    End point description
    % of patients that completed study
    End point type
    Primary
    End point timeframe
    Day 1 to Day 29
    Notes
    [6] - 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: Single arm study - statistical analysis not reported in accordance with EudraCT & EU CTR Frequently asked questions (EMA/370102/2016) - Question 83
    End point values
    		 Arm 1 - Single group study
    Number of subjects analysed
    16
    Units: % patients completing study
    100
    No statistical analyses for this end point

    Primary: Efficacy Endpoint - OFF Time

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    End point title
    		 Efficacy Endpoint - OFF Time [7]
    End point description
    OFF time based on in-person investigator ratings for Day 15 (oral LD/CD morning dose + DopaFuse) vs. Day 1 (oral LD/CD)
    End point type
    Primary
    End point timeframe
    Day 1 to Day 15
    Notes
    [7] - 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: Single arm study - statistical analysis not reported in accordance with EudraCT & EU CTR Frequently asked questions (EMA/370102/2016) - Question 83
    End point values
    		 Arm 1 - Single group study
    Number of subjects analysed
    16
    Units: OFF time (Hours)
    arithmetic mean (standard deviation)
        Day 1 - Oral LD/CD
    3.23 ± 2.18
        Day 2 - DopaFuse alone
    3.07 ± 2.52
        Day 3 - DopaFuse + morning LD/CD
    1.60 ± 1.48
        Day 15 - DopaFuse + morning LD/CD
    1.51 ± 1.44
    No statistical analyses for this end point

    Secondary: Variability in plasma concentration of levodopa

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    End point title
    		 Variability in plasma concentration of levodopa
    End point description
    Variability in levodopa concentrations assessed by levodopa fluctuation index (FI) at 0-12 hours for Day 3 vs. and Day 1
    End point type
    Secondary
    End point timeframe
    0-12 hours for Day 3 vs. Day 1
    End point values
    		 Arm 1 - Single group study
    Number of subjects analysed
    16
    Units: Levodopa Fluctuation Index (FI)
    arithmetic mean (standard deviation)
        Day 3 (DopaFuse + LD/CD morning dose)
    2.06 ± 0.44
        Day 1 (Oral LD/CD)
    2.47 ± 0.47
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs were recorded from Day 1 until Day 29
    Adverse event reporting additional description
    AEs and ADEs were collected at each clinic or telephone visit with a non-leading question, as well as by reporting those events directly observed and spontaneously reported by the patient.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Arm 1 - Single group study
    Reporting group description
    		 Single arm study

    Serious adverse events
    		 Arm 1 - Single group study
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Arm 1 - Single group study
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 16 (50.00%)
    Vascular disorders
    Orthostatic hypotension
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    General disorders and administration site conditions
    Complication associated with device
         subjects affected / exposed
    5 / 16 (31.25%)
         occurrences all number
    9
    Vessel puncture site haematoma
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Vomiting
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Dry mouth
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Oral disorder
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1

    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
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    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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