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    Clinical Trial Results:
    A Double-blind, Placebo-controlled, Randomized, 18-month Phase 2a Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Oral UCB0599 in Study Participants with Early Parkinson’s Disease

    Summary
    EudraCT number
    		 2020-003265-19
    Trial protocol
    		 DE   FR   PL   ES   NL   IT  
    Global end of trial date
    06 Sep 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    23 Oct 2025
    First version publication date
    23 Oct 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    PD0053
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04658186
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    UCB Biopharma SRL
    Sponsor organisation address
    Allée de la Recherche 60, Brussels, Belgium, 1070
    Public contact
    UCB BIOSCIENCES GmbH, Clin Trial Reg & Results Disclosure, clinicaltrials@ucb.com
    Scientific contact
    UCB BIOSCIENCES GmbH, Clin Trial Reg & Results Disclosure, clinicaltrials@ucb.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
    24 Oct 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    06 Sep 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Sep 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this study is to demonstrate the superiority of UCB0599 over placebo with regard to clinical symptoms of disease progression over 12 and 18 months in participants diagnosed with early-stage Parkinson’s Disease (PD)
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored. DAT-SPECT related: Each study participant received a maximum of 3 injections of 123I-Ioflupane during the study, according to the approved label. The target single injection dose of 185MBq is estimated to result in a radiation burden of 4.63mSv and the total effective dose for the study will be 13.89mSv. This is categorized as a category III substantial risk level (ICR62), which is balanced by the substantial societal benefit from the results of this clinical study. A dose of a thyroid blocking agent was given before the radiotracer dose.
    Background therapy
    		 Background therapy as permitted in the protocol.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Dec 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 21
    Country: Number of subjects enrolled
    France: 66
    Country: Number of subjects enrolled
    Germany: 58
    Country: Number of subjects enrolled
    Italy: 55
    Country: Number of subjects enrolled
    Netherlands: 22
    Country: Number of subjects enrolled
    Poland: 84
    Country: Number of subjects enrolled
    Spain: 28
    Country: Number of subjects enrolled
    United Kingdom: 32
    Country: Number of subjects enrolled
    United States: 130
    Worldwide total number of subjects
    496
    EEA total number of subjects
    313
    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)
    313
    From 65 to 84 years
    183
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study started to enroll participants in December 2020 and concluded in September 2024.

    Pre-assignment
    Screening details
    		 The Participant Flow refers to the Randomized Set.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Participants received UCB0599 matching-placebo capsules, orally, from Day 1 up to 18 months during treatment period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    UCB0599 matching-placebo capsules
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    UCB0599 matching-placebo capsules were administered orally, from Day 1 to Month 18

    Arm title
    		 UCB0599 Low Dose Arm
    Arm description
    		 Participants received UCB0599 at low dose as capsules, orally, from Day 1 up to 18 months during treatment period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    UCB0599
    Investigational medicinal product code
    Other name
    Minzasolmin
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    UCB0599 low dose capsules were administered orally, from Day 1 to Month 18.

    Arm title
    		 UCB0599 High Dose Arm
    Arm description
    		 Participants received UCB0599 at high dose as capsules, orally, from Day 1 up to 18 months during treatment period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    UCB0599
    Investigational medicinal product code
    Other name
    Minzasolmin
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    UCB0599 high dose capsules were administered orally, from Day 1 to Month 18.

    Number of subjects in period 1
    		Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Started
    165
    166
    165
    Completed
    154
    138
    139
    Not completed
    11
    28
    26
         Participant is Moving in Another Province
    -
    1
    -
         Non-Compliance
    -
    1
    1
         Adverse event, non-fatal
    4
    18
    15
         Dropout on the Promotor's Decision
    -
    -
    1
         Worsening Symptoms; Participant Withdrew
    -
    -
    1
         Site Closure; Participant declined transfer
    -
    1
    -
         Lost to follow-up
    -
    -
    2
         Participant Not Eligible. Randomized In Error
    1
    -
    1
         Consent Withdrawn by Participant (not due to AE)
    5
    6
    3
         Lack of efficacy
    1
    1
    -
         Protocol deviation
    -
    -
    1
         PI Decision due to Participant Safety
    -
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received UCB0599 matching-placebo capsules, orally, from Day 1 up to 18 months during treatment period.

    Reporting group title
    UCB0599 Low Dose Arm
    Reporting group description
    		 Participants received UCB0599 at low dose as capsules, orally, from Day 1 up to 18 months during treatment period.

    Reporting group title
    UCB0599 High Dose Arm
    Reporting group description
    		 Participants received UCB0599 at high dose as capsules, orally, from Day 1 up to 18 months during treatment period.

    Reporting group values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm Total
    Number of subjects
    		 165 166 165 496
    Age Categorical
    Units: participants
        18 years to less than (<) 65 years
    		 98 105 110 313
        65 years to <85 years
    		 67 61 55 183
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 61.2 ( 8.3 ) 61.4 ( 7.8 ) 59.9 ( 8.4 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 65 66 65 196
        Male
    		 100 100 100 300

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received UCB0599 matching-placebo capsules, orally, from Day 1 up to 18 months during treatment period.

    Reporting group title
    UCB0599 Low Dose Arm
    Reporting group description
    		 Participants received UCB0599 at low dose as capsules, orally, from Day 1 up to 18 months during treatment period.

    Reporting group title
    UCB0599 High Dose Arm
    Reporting group description
    		 Participants received UCB0599 at high dose as capsules, orally, from Day 1 up to 18 months during treatment period.

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Day 0

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Day 0 [1]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. Full analysis set (FAS) included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Day 0
    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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    156
    159
    159
    Units: score on a scale
        arithmetic mean (standard deviation)
    34.0 ( 13.2 )
    33.9 ( 16.4 )
    30.6 ( 13.4 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 6

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 6 [2]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 6
    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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    154
    147
    146
    Units: score on a scale
        arithmetic mean (standard deviation)
    37.9 ( 16.0 )
    36.6 ( 20.0 )
    32.9 ( 15.7 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 2

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 2 [3]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 2
    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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    153
    156
    144
    Units: score on a scale
        arithmetic mean (standard deviation)
    34.7 ( 13.9 )
    34.8 ( 18.0 )
    30.3 ( 14.6 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 4

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 4 [4]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 4
    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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    153
    148
    143
    Units: score on a scale
        arithmetic mean (standard deviation)
    34.7 ( 14.9 )
    34.3 ( 18.6 )
    31.4 ( 15.0 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 8

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 8 [5]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 8
    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: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    155
    148
    143
    Units: score on a scale
        arithmetic mean (standard deviation)
    35.6 ( 14.3 )
    34.6 ( 19.7 )
    32.9 ( 15.5 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 10

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 10 [6]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 10
    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: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    156
    141
    140
    Units: score on a scale
        arithmetic mean (standard deviation)
    35.6 ( 13.6 )
    33.7 ( 18.4 )
    33.2 ( 15.6 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 14

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 14 [7]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 14
    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: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    154
    137
    141
    Units: score on a scale
        arithmetic mean (standard deviation)
    37.1 ( 14.6 )
    34.8 ( 18.8 )
    33.9 ( 16.9 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 16

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 16 [8]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 16
    Notes
    [8] - 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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    152
    139
    137
    Units: score on a scale
        arithmetic mean (standard deviation)
    37.2 ( 15.6 )
    35.9 ( 19.8 )
    33.6 ( 15.4 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 12

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 12 [9]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 12
    Notes
    [9] - 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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    152
    142
    140
    Units: score on a scale
        arithmetic mean (standard deviation)
    37.3 ( 15.3 )
    36.5 ( 19.2 )
    34.5 ( 15.4 )
    No statistical analyses for this end point

    Primary: Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 18

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    End point title
    		 Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 18 [10]
    End point description
    MDS-UPDRS is multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0–52 possible points). Part IA: 6 questions and assessed by examiner (0–24 possible points). Part IB: 7 questions on non-motor experiences of daily living completed (0–28 possible points). Part II assessed motor experiences of daily living (0–52 possible points) and includes 13 questions completed. Part III assessed motor signs of PD and was administered by rater (0–52 possible points). Part III: 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III(Score: 0–236). Higher score = more severe PD symptoms. FAS included all randomized participants who received at least partial study dose and had >=1 post-Baseline assessment. Number analyzed includes those evaluable for this assessment.
    End point type
    Primary
    End point timeframe
    Month 18
    Notes
    [10] - 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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    152
    138
    137
    Units: score on a scale
        arithmetic mean (standard deviation)
    37.9 ( 15.5 )
    35.6 ( 18.1 )
    34.9 ( 16.4 )
    No statistical analyses for this end point

    Secondary: MDS-UPDRS Part III Subscale

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    End point title
    		 MDS-UPDRS Part III Subscale
    End point description
    MDS-UPDRS part III includes motor items assessing speech, facial expression, rigidity, finger tapping, hand movements, pronation supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement (body bradykinesia), postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, and constancy of rest tremor. It included 33 scores based on 18-items, each anchored with 5 responses: 0=normal,1=slight,2=mild,3=moderate, 4=severe. The scale range was from 0 to 132, with a lower score = better motor function and a higher score = more severe motor symptoms. FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, “N” included all participants who were evaluable for this assessment and number analyzed (n) signifies participants who were evaluable at specified timepoints.
    End point type
    Secondary
    End point timeframe
    Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    159
    162
    164
    Units: score on a scale
    arithmetic mean (standard deviation)
        Day 0 (n=159, 162, 164)
    22.6 ( 9.8 )
    21.9 ( 11.3 )
    20.4 ( 9.5 )
        Month 2 (n=159, 158, 151)
    22.9 ( 10.2 )
    22.7 ( 12.4 )
    20.0 ( 10.3 )
        Month 4 (n= 157, 151, 147)
    23.1 ( 10.5 )
    22.5 ( 12.7 )
    20.7 ( 10.6 )
        Month 6 (n=157, 147, 147)
    24.3 ( 10.9 )
    23.2 ( 12.4 )
    21.3 ( 10.8 )
        Month 8 (n=156, 148, 145)
    23.3 ( 10.0 )
    22.2 ( 12.6 )
    21.0 ( 10.7 )
        Month 10 (n=156, 141, 140)
    23.3 ( 9.2 )
    21.6 ( 12.1 )
    21.0 ( 11.0 )
        Month 12 (n=153, 143, 140)
    24.1 ( 10.0 )
    22.8 ( 12.0 )
    21.8 ( 10.5 )
        Month 14 (n= 154, 139, 141)
    24.1 ( 10.1 )
    22.2 ( 11.9 )
    21.8 ( 11.4 )
        Month 16 (n=153, 139, 137)
    24.3 ( 10.1 )
    22.8 ( 12.3 )
    21.7 ( 10.7 )
        Month 18 (n=153, 138, 138)
    24.2 ( 10.1 )
    23.0 ( 11.7 )
    22.2 ( 10.9 )
    No statistical analyses for this end point

    Secondary: MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items

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    End point title
    		 MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items
    End point description
    The early-stage Parkinson’s disease (ePD) subscore is derived from a 15-item subset of the MDS-UPDRS Part III (Motor Examination). It includes all rigidity assessments (neck, upper limbs [right/left], and lower limbs [right/left]) and bradykinesia-related tasks: finger tapping (right/left), hand movements (right/left), pronation-supination of hands (right/left), toe tapping (right/left), and leg agility (right/left). Each item is scored on a 5-point likert scale (0 = no problem to 4 = severe), resulting in a total ePD subscore range of 0 to 60. Higher scores indicate greater motor impairment, while lower scores reflect better motor function. FAS was used. “N”= all participants who were evaluable for this assessment; 'n'=participants who were evaluable at specified timepoints.
    End point type
    Secondary
    End point timeframe
    Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    159
    162
    164
    Units: score on a scale
    arithmetic mean (standard deviation)
        Day 0 (n= 159, 162, 164)
    13.3 ( 7.11 )
    12.6 ( 7.96 )
    11.8 ( 6.85 )
        Month 2 (n=159, 159, 151)
    13.2 ( 7.33 )
    13.2 ( 8.48 )
    11.6 ( 7.57 )
        Month 4 (n=157, 151, 147)
    13.4 ( 7.50 )
    13.1 ( 8.76 )
    11.8 ( 7.64 )
        Month 6 (157, 148, 147)
    13.9 ( 7.39 )
    13.4 ( 8.42 )
    12.4 ( 7.58 )
        Month 8 (157, 148, 146)
    13.4 ( 6.82 )
    12.7 ( 8.60 )
    11.8 ( 7.49 )
        Month 10 (n=156, 141, 141)
    13.4 ( 6.46 )
    12.1 ( 8.14 )
    11.9 ( 7.98 )
        Month 12 (n=153, 144, 142)
    13.7 ( 6.98 )
    12.7 ( 8.20 )
    12.9 ( 7.61 )
        Month 14 (n=154, 142, 141)
    13.8 ( 7.14 )
    12.7 ( 8.35 )
    12.5 ( 7.88 )
        Month 16 (n=153, 139, 138)
    13.8 ( 7.24 )
    12.9 ( 8.26 )
    12.4 ( 7.83 )
        Month 18 (n=153, 138, 138)
    13.8 ( 7.34 )
    12.9 ( 7.90 )
    12.6 ( 7.61 )
    No statistical analyses for this end point

    Secondary: MDS-UPDRS Part II Subscale

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    End point title
    		 MDS-UPDRS Part II Subscale
    End point description
    MDS-UPDRS part II includes motor items assessing speech, saliva and drooling, chewing and swallowing, eating tasks (cutting food and handling utensils), dressing, hygiene, handwriting, doing hobbies and other activities, turning in bed, tremor, getting out of bed, a car or a deep chair, walking and balance, and freezing. Each of the items in the UPDRS is measured on a scale. It included 13-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range for Part II was 0-52, with higher scores reflecting greater severity. FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment and 'n' signifies participants who were evaluable at specified timepoints.
    End point type
    Secondary
    End point timeframe
    Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    163
    163
    161
    Units: score on a scale
    arithmetic mean (standard deviation)
        Day 0 (n=163, 163, 161)
    6.0 ( 4.0 )
    6.3 ( 4.6 )
    5.7 ( 4.3 )
        Month 2 (n=158, 160, 149)
    6.5 ( 4.3 )
    6.9 ( 5.2 )
    6.1 ( 4.1 )
        Month 4 (156, 150, 147)
    6.8 ( 4.6 )
    7.1 ( 5.4 )
    6.4 ( 4.5 )
        Month 6 (n=156, 148, 147)
    7.7 ( 5.1 )
    7.7 ( 6.2 )
    6.7 ( 5.0 )
        Month 8 (n=158, 148, 145)
    7.0 ( 4.6 )
    7.1 ( 5.7 )
    6.9 ( 4.7 )
        Month 10 (n=156, 142, 143)
    6.9 ( 4.5 )
    6.7 ( 5.4 )
    7.1 ( 4.7 )
        Month 12 (153, 144, 143)
    6.9 ( 4.9 )
    7.3 ( 5.5 )
    7.3 ( 5.1 )
        Month 14 (n=154, 140, 142)
    7.1 ( 4.8 )
    7.2 ( 5.8 )
    7.0 ( 5.1 )
        Month 16 (152, 140, 138 )
    7.1 ( 5.1 )
    7.2 ( 5.7 )
    6.7 ( 4.5 )
        Month 18 (n=152, 138, 138)
    7.3 ( 5.0 )
    6.8 ( 5.2 )
    7.3 ( 5.3 )
    No statistical analyses for this end point

    Secondary: MDS-UPDRS Part I Subscale

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    End point title
    		 MDS-UPDRS Part I Subscale
    End point description
    MDS-UPDRS Part I includes several non-motor aspects of experiences of daily living including cognitive impairment,hallucinations and psychosis,depressed mood,anxious mood,apathy,features of dopamine dysregulation syndrome during Part IA; and sleep problems, daytime sleepiness,pain and other sensation,urinary problems,constipation problems,light headedness on standing, and fatigue during Part IB.Part I assessed non-motor experiences of daily living and has 2 components ranging: 0–52.Part IA contained 6 questions and was assessed by examiner ranging: 0–24. Part IB contained 7 questions on non-motor experiences of daily living and was completed by participant ranging: 0–28.Each item is measured on scale of 0 to 4, 0 =normal and 4=higher score =severe abnormalities/worse outcome. FAS. “N”=all participants who were evaluable for assessment and 'n'=participants who were evaluable at specified timepoints.
    End point type
    Secondary
    End point timeframe
    Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    158
    161
    158
    Units: score on a scale
    arithmetic mean (standard deviation)
        Day 0 (n= 158, 161, 158)
    5.4 ( 3.9 )
    5.5 ( 4.4 )
    4.7 ( 3.7 )
        Month 2 (n= 155, 160, 145)
    5.2 ( 4.2 )
    5.2 ( 3.8 )
    4.3 ( 3.5 )
        Month 4 (n=153, 149, 144)
    5.1 ( 4.4 )
    5.0 ( 4.0 )
    4.5 ( 3.8 )
        Month 6 (n=154, 148, 146)
    5.9 ( 4.8 )
    5.7 ( 4.5 )
    5.1 ( 3.9 )
        Month 8 (n=156, 148, 147)
    5.5 ( 4.4 )
    5.3 ( 4.2 )
    5.0 ( 3.9 )
        Month 10 (n=156, 142, 143)
    5.4 ( 4.2 )
    5.4 ( 4.1 )
    4.9 ( 4.1 )
        Month 12 (n=152, 143, 143)
    6.2 ( 5.1 )
    6.2 ( 4.8 )
    5.3 ( 4.4 )
        Month 14 (n=154, 141, 142)
    5.9 ( 4.6 )
    5.8 ( 5.0 )
    5.0 ( 4.2 )
        Month 16 (n= 153, 140, 138)
    5.9 ( 4.8 )
    5.9 ( 5.1 )
    5.1 ( 4.1 )
        Month 18 (n=152, 138, 138)
    6.4 ( 5.1 )
    5.9 ( 4.6 )
    5.3 ( 4.5 )
    No statistical analyses for this end point

    Secondary: Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II

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    End point title
    		 Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II
    End point description
    The participant was considered to have an emerging symptom for the item, if the change from Baseline for the item is greater than 0 for 2 consecutive visits. The magnitude of change from Baseline was not considered to determine the emerging symptom. Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part II includes motor items assessing speech, saliva and drooling, chewing and swallowing, eating tasks (cutting food and handling utensils), dressing, hygiene, handwriting, doing hobbies and other activities, turning in bed, tremor, getting out of bed, a car or a deep chair, walking and balance, and freezing. This included 13-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range for Part II was 0-52, with higher scores = greater severity. FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment.
    End point type
    Secondary
    End point timeframe
    Baseline to Month 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    163
    164
    164
    Units: participants
        Number of Emerging symptoms: 0
    28
    36
    38
        Number of Emerging symptoms: 1
    18
    41
    31
        Number of Emerging symptoms: 2
    35
    33
    23
        Number of Emerging symptoms: 3
    37
    15
    26
        Number of Emerging symptoms: 4
    15
    13
    15
        Number of Emerging symptoms: 5
    15
    13
    14
        Number of Emerging symptoms: 6
    4
    7
    10
        Number of Emerging symptoms: 7
    4
    4
    4
        Number of Emerging symptoms: 8
    4
    1
    3
        Number of Emerging symptoms: 9
    2
    1
    0
        Number of Emerging symptoms: 10
    1
    0
    0
        Number of Emerging symptoms: 11
    0
    0
    0
        Number of Emerging symptoms: 12
    0
    0
    0
        Number of Emerging symptoms: 13
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Time to Worsening of the Disease as Measured by MDS-UPDRS Part III

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    End point title
    		 Time to Worsening of the Disease as Measured by MDS-UPDRS Part III
    End point description
    Time to worsening of the disease on the MDS-UPDRS III scale as defined by a 5-point increase in MDS-UPDRS III, within the 18-month period. MDS-UPDRS part III includes motor items assessing speech,facial expression,rigidity,finger tapping,hand movements,pronation supination movements of hands,toe tapping,leg agility,arising from chair,gait,freezing of gait,postural stability,posture,global spontaneity of movement (body bradykinesia), postural tremor of hands,kinetic tremor of hands,rest tremor amplitude, and constancy of rest tremor. It included 33 scores based on 18-items, each anchored with 5 responses: 0=normal,1=slight,2=mild,3=moderate, 4=severe. Scale ranges: 0-132, with lower score = better motor function; higher score = more severe motor symptoms. FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment.
    End point type
    Secondary
    End point timeframe
    Baseline to Month 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    163
    164
    164
    Units: months
        number (not applicable)
    8.64
    8.95
    9.45
    Statistical analysis title
    		 Statistical Analysis 2
    Statistical analysis description
    Results obtained from generalized linear model for RMET, with gender and age at baseline as covariates and treatment group as effect of interest.
    Comparison groups
    Placebo v UCB0599 High Dose Arm
    Number of subjects included in analysis
    327
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.214
    Method
    		 Wald Chi-square
    Parameter type
    		 Difference in RMET
    Point estimate
    0.81
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.47
         upper limit
    		 2.09
    Statistical analysis title
    		 Statistical Analysis 1
    Statistical analysis description
    Results were obtained from a generalized linear model for the restricted mean event time (RMET), with gender and age at baseline as covariates and treatment group as the effect of interest.
    Comparison groups
    Placebo v UCB0599 Low Dose Arm
    Number of subjects included in analysis
    327
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.647
    Method
    		 Wald Chi-square
    Parameter type
    		 Difference in RMET
    Point estimate
    0.31
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1
         upper limit
    		 1.61

    Secondary: Montreal Cognitive Assessment (MoCA)

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    End point title
    		 Montreal Cognitive Assessment (MoCA)
    End point description
    The MoCA assesses different cognitive domains (visuospatial/executive, naming, memory, attention, language, abstraction, delayed recall, and orientation). Participants are assessed on a 30-point scale. A score of 26 or above is considered normal, a lower score indicates cognitive impairment. FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, 'n' signifies participants who were evaluable at each specified timepoints.
    End point type
    Secondary
    End point timeframe
    Screening, Month 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    163
    164
    164
    Units: score on a scale
    arithmetic mean (standard deviation)
        Screening (n=163, 164, 164)
    27.8 ( 1.8 )
    27.6 ( 2.0 )
    27.8 ( 1.7 )
        Month 18 (n= 152, 137, 136)
    27.4 ( 2.2 )
    27.3 ( 2.4 )
    27.3 ( 2.4 )
    No statistical analyses for this end point

    Secondary: Change from Screening in Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR)

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    End point title
    		 Change from Screening in Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR)
    End point description
    The change from screening in mean striatum specific binding ratios (SBR) was assessed by DaT-SPECT using 123I-Ioflupane as radiopharmaceutical. The whole striatum was calculated as the average of the SBR data values for the four following “small” regions: left caudate small, left putamen small, right caudate small and right putamen small. The SBR was calculated for each region with the occipital cortex as a reference region, where a lower SBR indicates worse disease. The following formula was used to calculate this: (Average [Small region] – Average [Occipital region])/ (Average [Occipital region]). FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment and 'n' signifies participants who were evaluable at each specified timepoints.
    End point type
    Secondary
    End point timeframe
    Screening, Months 12 and 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    140
    133
    130
    Units: specific binding ratio
    arithmetic mean (standard deviation)
        Whole striatum:Month (M) 12(n=133,130,111)
    -0.202 ( 0.215 )
    -0.141 ( 0.194 )
    -0.102 ( 0.254 )
        Whole striatum: M18 (n=140,124,130)
    -0.233 ( 0.226 )
    -0.182 ( 0.232 )
    -0.193 ( 0.254 )
        Ipsilateral striatum: M 12 (n=114,114,95)
    -0.237 ( 0.262 )
    -0.161 ( 0.246 )
    -0.093 ( 0.294 )
        Ipsilateral striatum: M 18 (n=123,109,114)
    -0.260 ( 0.263 )
    -0.211 ( 0.282 )
    -0.194 ( 0.318 )
        Contralateral striatum: M 12 (n=114,114,95)
    -0.161 ( 0.210 )
    -0.138 ( 0.209 )
    -0.099 ( 0.278 )
        Contralateral striatum: M 18 (n=123,109,114)
    -0.203 ( 0.214 )
    -0.169 ( 0.237 )
    -0.188 ( 0.259 )
        Ipsilateral caudate small: M 12 (n=114,114,95)
    -0.296 ( 0.380 )
    -0.196 ( 0.352 )
    -0.080 ( 0.485 )
        Ipsilateral caudate small: M 18 (n=123,109,114)
    -0.312 ( 0.383 )
    -0.273 ( 0.390 )
    -0.210 ( 0.439 )
        Ipsilateral putamen small: M 12 (n=114,114,95)
    -0.178 ( 0.260 )
    -0.125 ( 0.255 )
    -0.106 ( 0.219 )
        Ipsilateral putamen small: M 18 (n=123,109,114)
    -0.208 ( 0.252 )
    -0.150 ( 0.260 )
    -0.179 ( 0.270 )
        Contralateral caudate small: M 12 (n=114,114,95)
    -0.256 ( 0.328 )
    -0.183 ( 0.332 )
    -0.145 ( 0.363 )
        Contralateral caudate small: M 18 (n=123,109,114)
    -0.291 ( 0.330 )
    -0.263 ( 0.370 )
    -0.248 ( 0.398 )
        Contralateral putamen small: M 12 (114,114,95)
    -0.066 ( 0.190 )
    -0.094 ( 0.223 )
    -0.053 ( 0.304 )
        Contralateral putamen small: M 18 (n=123,109,114)
    -0.115 ( 0.190 )
    -0.076 ( 0.197 )
    -0.128 ( 0.209 )
    No statistical analyses for this end point

    Secondary: Time to Start of Symptomatic Treatment (ST)

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    End point title
    		 Time to Start of Symptomatic Treatment (ST)
    End point description
    Time to start of symptomatic treatment (ST) within the 18-month period. FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment.
    End point type
    Secondary
    End point timeframe
    Baseline to Month 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    163
    164
    164
    Units: month
        number (not applicable)
    10.58
    11.59
    11.80
    Statistical analysis title
    		 Statistical Analysis 2
    Statistical analysis description
    Results were obtained from a generalized linear model for the RMET, with gender and age at baseline as covariates and treatment group as the effect of interest.
    Comparison groups
    Placebo v UCB0599 High Dose Arm
    Number of subjects included in analysis
    327
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.064
    Method
    		 Wald Chi-sqaure
    Parameter type
    		 Difference in RMET
    Point estimate
    1.22
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.07
         upper limit
    		 2.51
    Statistical analysis title
    		 Statistical Analysis 1
    Statistical analysis description
    Results were obtained from a generalized linear model for the RMET, with gender and age at baseline as covariates and treatment group as the effect of interest.
    Comparison groups
    Placebo v UCB0599 Low Dose Arm
    Number of subjects included in analysis
    327
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.124
    Method
    		 Wald Chi-square
    Parameter type
    		 Difference in RMET
    Point estimate
    1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.27
         upper limit
    		 2.27

    Secondary: Symptomatic Treatment (ST) Intake

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    End point title
    		 Symptomatic Treatment (ST) Intake
    End point description
    Cumulative number of participants on symptomatic treatment (ST) at 18 months are reported. FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
    End point type
    Secondary
    End point timeframe
    Month 18
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    153
    139
    140
    Units: participants
    111
    89
    88
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)

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    End point title
    		 Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
    End point description
    An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE is defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment. Safety set included all randomized study participants who receive at least a partial dose of study medication.
    End point type
    Secondary
    End point timeframe
    From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    164
    165
    164
    Units: percentage of participants
        number (not applicable)
    87.8
    86.7
    87.2
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Serious TEAEs

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    End point title
    		 Percentage of Participants With Serious TEAEs
    End point description
    Serious adverse event: Death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. Important medical events that may not result in death, be life-threatening, or require hospitalization may be considered serious when, based upon appropriate medical judgment, they may jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the outcomes listed in this definition. Safety set included all randomized study participants who receive at least a partial dose of study medication.
    End point type
    Secondary
    End point timeframe
    From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    164
    165
    164
    Units: percentage of participants
        number (not applicable)
    5.5
    7.9
    8.5
    No statistical analyses for this end point

    Secondary: Percentage of Participants With TEAEs Leading to Participant Withdrawal

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    End point title
    		 Percentage of Participants With TEAEs Leading to Participant Withdrawal
    End point description
    An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE is defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment. Safety set included all randomized study participants who receive at least a partial dose of study medication.
    End point type
    Secondary
    End point timeframe
    From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
    End point values
    		 Placebo UCB0599 Low Dose Arm UCB0599 High Dose Arm
    Number of subjects analysed
    164
    165
    164
    Units: percentage of participants
        number (not applicable)
    2.4
    10.9
    9.8
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
    Adverse event reporting additional description
    Safety analysis set included all randomized study participants who received at least a partial dose of study medication.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received UCB0599 matching-placebo capsules, orally, (4 UCB0599 matching-placebo capsules) twice per day (BID) from Day 1 up to 18 months.

    Reporting group title
    UCB0599 High Dose Arm
    Reporting group description
    		 Participants received UCB0599 low dose, capsules, orally, (4 UCB0599 low dose capsules) BID from Day 1 up to 18 months.

    Reporting group title
    UCB0599 Low Dose Arm
    Reporting group description
    		 Participants received UCB0599 low dose, capsules, orally, (2 UCB0599 low dose capsules and 2 UCB0599 matching-placebo capsules) BID from Day 1 up to 18 months.

    Serious adverse events
    		 Placebo UCB0599 High Dose Arm UCB0599 Low Dose Arm
    Total subjects affected by serious adverse events
         subjects affected / exposed
    9 / 164 (5.49%)
    14 / 164 (8.54%)
    13 / 165 (7.88%)
         number of deaths (all causes)
    0
    1
    1
         number of deaths resulting from adverse events
    0
    1
    1
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Gastric cancer
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metastatic gastric cancer
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    Prostate cancer
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lung neoplasm malignant
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Malignant melanoma
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Postoperative ileus
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hip fracture
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Fall
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Jaw fracture
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Mitral valve prolapse
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Angina pectoris
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Acute coronary syndrome
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Palpitations
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac arrest
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    Atrial fibrillation
         subjects affected / exposed
    2 / 164 (1.22%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Vocal cord paralysis
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Transient ischaemic attack
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Drug hypersensitivity
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Intestinal polyp
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pancreatitis acute
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Rectal haemorrhage
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Inguinal hernia
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Diarrhoea
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Hepatic cytolysis
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Angioedema
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Rash
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Nephrolithiasis
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Acute kidney injury
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary bladder polyp
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal colic
         subjects affected / exposed
    0 / 164 (0.00%)
    0 / 164 (0.00%)
    1 / 165 (0.61%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Coronavirus infection
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia aspiration
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infectious pleural effusion
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Diverticulitis
         subjects affected / exposed
    0 / 164 (0.00%)
    1 / 164 (0.61%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urosepsis
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    1 / 164 (0.61%)
    0 / 164 (0.00%)
    0 / 165 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo UCB0599 High Dose Arm UCB0599 Low Dose Arm
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    102 / 164 (62.20%)
    89 / 164 (54.27%)
    97 / 165 (58.79%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    9 / 164 (5.49%)
    12 / 164 (7.32%)
    17 / 165 (10.30%)
         occurrences all number
    9
    14
    22
    Nervous system disorders
    Headache
         subjects affected / exposed
    16 / 164 (9.76%)
    16 / 164 (9.76%)
    11 / 165 (6.67%)
         occurrences all number
    17
    24
    25
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    12 / 164 (7.32%)
    7 / 164 (4.27%)
    13 / 165 (7.88%)
         occurrences all number
    14
    7
    15
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    14 / 164 (8.54%)
    8 / 164 (4.88%)
    7 / 165 (4.24%)
         occurrences all number
    14
    9
    7
    Diarrhoea
         subjects affected / exposed
    8 / 164 (4.88%)
    9 / 164 (5.49%)
    10 / 165 (6.06%)
         occurrences all number
    10
    10
    18
    Nausea
         subjects affected / exposed
    8 / 164 (4.88%)
    8 / 164 (4.88%)
    12 / 165 (7.27%)
         occurrences all number
    15
    8
    14
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    9 / 164 (5.49%)
    11 / 164 (6.71%)
    5 / 165 (3.03%)
         occurrences all number
    10
    11
    5
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    17 / 164 (10.37%)
    12 / 164 (7.32%)
    15 / 165 (9.09%)
         occurrences all number
    17
    14
    19
    Arthralgia
         subjects affected / exposed
    19 / 164 (11.59%)
    4 / 164 (2.44%)
    14 / 165 (8.48%)
         occurrences all number
    22
    4
    14
    Infections and infestations
    Influenza
         subjects affected / exposed
    10 / 164 (6.10%)
    14 / 164 (8.54%)
    6 / 165 (3.64%)
         occurrences all number
    10
    15
    7
    Urinary tract infection
         subjects affected / exposed
    15 / 164 (9.15%)
    12 / 164 (7.32%)
    18 / 165 (10.91%)
         occurrences all number
    20
    14
    26
    COVID-19
         subjects affected / exposed
    35 / 164 (21.34%)
    32 / 164 (19.51%)
    22 / 165 (13.33%)
         occurrences all number
    38
    33
    23
    Nasopharyngitis
         subjects affected / exposed
    19 / 164 (11.59%)
    14 / 164 (8.54%)
    14 / 165 (8.48%)
         occurrences all number
    20
    15
    16

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    29 Oct 2020
    Protocol Amendment 1 was dated 29 Oct 2020. The purpose of this substantial amendment was to remove planned video recording of participants during the MDS-UPDRS Part III assessment due to operational limitations (example, procedures for de-identification, video processing, video data handling, access, and storage).
    21 May 2021
    Protocol Amendment 2 was dated 21 May 2021. The purpose of this substantial amendment was to increase the upper age limit for participants’ inclusion in the study to 75 years, to reflect the ePD population. This population did not present additional risk compared to the population initially included in the study.
    04 Oct 2021
    Protocol Amendment 3 was dated 04 Oct 2021. The purpose of this substantial amendment was to add a treatment group with a lower dose of UCB0599, allowed the use of historical symptomatic treatments, and harmonized feedback from regulatory authorities and ethic review boards received during the initial Clinical Trial Applications.
    24 Feb 2022
    Protocol Amendment 4 was dated 24 Feb 2022. The purpose of this substantial amendment was to amend exclusion and discontinuation criteria regarding renal function, diabetes, and electrocardiograph (ECG) abnormalities to better reflect the parameters in the targeted study population.
    30 Jun 2023
    Protocol Amendment 5 was dated 30 Jun 2023. The purpose of this substantial amendment was to incorporate additional laboratory tests at Day 150 in order to allow for close monitoring of liver function test parameters.
    21 Mar 2024
    Protocol Amendment 6 was dated 21 Mar 2024. The purpose of this substantial amendment was to provided clarification around blinding and the timing of the Month 12 data analysis, and remove analyses that were no longer needed. In addition, the list of secondary and exploratory efficacy endpoints was updated.

    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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