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    Clinical Trial Results:
    A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Efficacy and Safety of BIIB122/DNL151 in Participants with Parkinson’s Disease and Pathogenic LRRK2 Variants

    Summary
    EudraCT number
    		 2022-000747-77
    Trial protocol
    		 DE   ES   FR   IT   BE  
    Global end of trial date
    27 Jul 2023

    Results information
    Results version number
    		 v2(current)
    This version publication date
    28 Jul 2024
    First version publication date
    14 Mar 2024
    Other versions
    		 v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    283PD302
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05418673
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Biogen
    Sponsor organisation address
    250 Binney Street, Cambridge, United States, 02142
    Public contact
    Biogen Study Medical Director, Biogen, clinicaltrials@biogen.com
    Scientific contact
    Biogen Study Medical Director, Biogen, clinicaltrials@biogen.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
    27 Jul 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Jul 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to evaluate the efficacy of BIIB122 225 milligrams (mg) compared with placebo, based on the time to confirmed worsening in Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III combined score.
    Protection of trial subjects
    Written informed consent was obtained from each subject or subject’s legally authorised representative (e.g., parent or legal guardian), as applicable, prior to evaluations performed for eligibility. Subjects or the subject’s legally authorised representative were given adequate time to review the information in the informed consent/assent and were allowed to ask, and have answered, questions concerning all portions of the conduct of the study.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    26 Aug 2022
    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
    United States: 6
    Country: Number of subjects enrolled
    United Kingdom: 1
    Worldwide total number of subjects
    7
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    2
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants took part in the study at multiple investigative sites in the United States and the United Kingdom from 26 Aug 2022 to 27 Jul 2023.

    Pre-assignment
    Screening details
    		 A total of 7 participants were enrolled and treated in the study, but none of the participants completed it. 

    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, Data analyst, Carer

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Participants received BIIB122-matching placebo tablets, orally, once daily (QD) for up to 290 days.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Administered as specified in the treatment arm.

    Arm title
    		 BIIB122 225 mg
    Arm description
    		 Participants received BIIB122 225 milligrams (mg) tablets, orally, QD for up to 290 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    BIIB122
    Investigational medicinal product code
    Other name
    DNL151
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Administered as specified in the treatment arm.

    Number of subjects in period 1
    		Placebo BIIB122 225 mg
    Started
    3
    4
    Completed
    0
    0
    Not completed
    3
    4
         Study Terminated by Sponsor
    2
    4
         Lost to follow-up
    1
    -

    Baseline characteristics

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    Subject analysis sets

    Subject analysis set title
    BIIB 122 or matching-placebo (Pooled)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants either received BIIB122 225 mg or BIIB122-matching placebo tablets, orally, QD for up to 290 days. To maintain blinding, pooled data are reported for baseline characteristics.

    Subject analysis sets values
    		 BIIB 122 or matching-placebo (Pooled)
    Number of subjects
    7
    Age Categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    66.9 ( 10.70 )
    Gender categorical
    Units: Subjects
        Male
    5
        Female
    2
    Ethnicity
    Units: Subjects
        Not Hispanic or Latino
    7
    Race
    Units: Subjects
        White
    7

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants received BIIB122-matching placebo tablets, orally, once daily (QD) for up to 290 days.

    Reporting group title
    BIIB122 225 mg
    Reporting group description
    		 Participants received BIIB122 225 milligrams (mg) tablets, orally, QD for up to 290 days.

    Subject analysis set title
    BIIB 122 or matching-placebo (Pooled)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants either received BIIB122 225 mg or BIIB122-matching placebo tablets, orally, QD for up to 290 days. To maintain blinding, pooled data are reported for baseline characteristics.

    Primary: Time to Confirmed Worsening in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III

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    End point title
    		 Time to Confirmed Worsening in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III [1]
    End point description
    Time to confirmed worsening is defined as a worsening event sustained over 2 consecutive assessments. MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. Part II assesses motor experiences of daily living (range 0-52). It contains 13 questions which are to be completed by the participant. Part III assesses the motor signs of PD and is administered by the rater (range 0-132). Part III contains 33 scores based on 18 items. For each question, a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Part II and III combined score equals the sum of Parts II and III (range 0-184). A higher score indicates more severe symptoms of PD. Based on the low enrolment number and to protect and maintain participant privacy/confidentiality, no data is reported, as only 1 participant completed post-baseline visit for clinical efficacy outcomes.
    End point type
    Primary
    End point timeframe
    From Week 96 to Week 180
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive analysis is planned to be analysed.
    End point values
    		 Placebo BIIB122 225 mg
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: Days
        median (full range (min-max))
    ( to )
    ( to )
    Notes
    [2] - Data were not reported for this endpoint as no participants were analyzed.
    [3] - Data were not reported for this endpoint as no participants were analyzed.
    No statistical analyses for this end point

    Secondary: Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

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    End point title
    		 Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
    End point description
    An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death; in the view of the investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or is a medically important event. Safety analysis set.
    End point type
    Secondary
    End point timeframe
    From the first dose (Day 1) of study drug up to end of follow-up (up to 336 days)
    End point values
    		 Placebo BIIB122 225 mg
    Number of subjects analysed
    3
    4
    Units: participants
        AEs
    2
    3
        SAEs
    0
    0
    No statistical analyses for this end point

    Secondary: Time to Confirmed Worsening in Modified Schwab and England Activities of Daily Living Scale (mSE-ADL) Score

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    End point title
    		 Time to Confirmed Worsening in Modified Schwab and England Activities of Daily Living Scale (mSE-ADL) Score
    End point description
    Time to confirmed worsening is defined as a worsening event sustained over 2 consecutive assessments. The mSE-ADL scale reflects the speed, ease, and independence with which an individual performs daily activities or personal chores with 100% indicating total independence, falling to 0%, which indicates a state of complete dependence. The individual is asked to rate his or her function using an 11-point scale (10% increments), from 100% (completely independent; able to do all chores without slowness, difficulty, or impairment; essentially normal; unaware of any difficulty) to 0% (vegetative functions such as swallowing, bladder and bowels are not functioning; bedridden). The lower the score, the worse the functional status. Based on the low enrolment number and to protect and maintain participant privacy/confidentiality, no data is reported, as only 1 participant completed post-baseline visit for clinical efficacy outcomes.
    End point type
    Secondary
    End point timeframe
    From Week 96 to Week 180
    End point values
    		 Placebo BIIB122 225 mg
    Number of subjects analysed
    0 [4]
    0 [5]
    Units: Days
        median (full range (min-max))
    ( to )
    ( to )
    Notes
    [4] - Data were not reported for this endpoint as no participants were analyzed.
    [5] - Data were not reported for this endpoint as no participants were analyzed.
    No statistical analyses for this end point

    Secondary: Change From Baseline in MDS-UPDRS Parts II and III Combined Score at Week 96

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    End point title
    		 Change From Baseline in MDS-UPDRS Parts II and III Combined Score at Week 96
    End point description
    MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. Part II assesses motor experiences of daily living (range 0-52). It contains 13 questions which are to be completed by the participant. Part III assesses the motor signs of PD and is administered by the rater (range 0-132). Part III contains 33 scores based on 18 items. For each question, a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Part II and III combined score equals the sum of Parts II and III (range 0-184). A higher score indicates more severe symptoms of PD. Due to the early termination of the study (at week 48), sufficient data were not collected for the outcome measure analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 96
    End point values
    		 Placebo BIIB122 225 mg
    Number of subjects analysed
    0 [6]
    0 [7]
    Units: Score on a scale
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [6] - Data were not reported for this endpoint as no participants were analyzed.
    [7] - Data were not reported for this endpoint as no participants were analyzed.
    No statistical analyses for this end point

    Secondary: Change From Baseline in MDS-UPDRS Parts I, II, and III Combined Score at Week 96

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    End point title
    		 Change From Baseline in MDS-UPDRS Parts I, II, and III Combined Score at Week 96
    End point description
    The MDS-UPDRS is a multimodal scale consisting of four parts. Part I assessed non-motor experiences of daily living and has 2 components (Range 0-52). Part IA contains 6 questions and are assessed by the examiner (Range 0-24). Part IB contains 7 questions on non-motor experiences of daily living which was completed by the participant (Range 0-28). Part II assessed motor experiences of daily living (Range 0-52). It contained 13 questions completed by the participant. Part III assessed the motor signs of PD and was administered by the rater (Range 0-132). Part III contained 33 scores based on 18 items. For each question a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. The MDS-UPDRS Total Score equals the sum of Parts I,II, and III (Range: 0-236). A higher score indicated more severe symptoms of PD. Due to the early termination of the study (at week 48), sufficient data were not collected for the outcome measure analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 96
    End point values
    		 Placebo BIIB122 225 mg
    Number of subjects analysed
    0 [8]
    0 [9]
    Units: Score on a scale
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [8] - Data were not reported for this endpoint as no participants were analyzed.
    [9] - Data were not reported for this endpoint as no participants were analyzed.
    No statistical analyses for this end point

    Secondary: Time to Confirmed Worsening in MDS-UPDRS Part II Score

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    End point title
    		 Time to Confirmed Worsening in MDS-UPDRS Part II Score
    End point description
    Time to confirmed worsening is defined as a worsening event sustained over 2 consecutive assessments. MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. Part II assesses motor experiences of daily living (range 0-52). It contains 13 questions which are to be completed by the participant. Part III assesses the motor signs of PD and is administered by the rater (range 0-132). Part III contains 33 scores based on 18 items. For each question, a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Part II and III combined score equals the sum of Parts II and III (range 0-184). A higher score indicates more severe symptoms of PD. Based on the low enrolment number and to protect and maintain participant privacy/confidentiality, no data is reported, as only 1 participant completed post-baseline visit for clinical efficacy outcomes.
    End point type
    Secondary
    End point timeframe
    From Week 96 to Week 180
    End point values
    		 Placebo BIIB122 225 mg
    Number of subjects analysed
    0 [10]
    0 [11]
    Units: Days
        median (full range (min-max))
    ( to )
    ( to )
    Notes
    [10] - Data were not reported for this endpoint as no participants were analyzed.
    [11] - Data were not reported for this endpoint as no participants were analyzed.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the first dose (Day 1) of the study drug up to the end of follow-up (up to 336 days)
    Adverse event reporting additional description
    SAS included all randomised subjects who received atleast 1 dose of study treatment. Low number of subjects were enrolled in study& some subjects have been rolled over to283PD201(NCT05348785),data was reported as pooled group & preferred terms are reported as system organ class to avoid identification of individual subjects & to maintain blinding.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26
    Reporting groups
    Reporting group title
    BIIB 122 or matching-placebo (Pooled)
    Reporting group description
    		 Participants either received BIIB122 225 mg or BIIB122-matching placebo tablets, orally, QD for up to 290 days.

    Serious adverse events
    		 BIIB 122 or matching-placebo (Pooled)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 7 (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
    		 BIIB 122 or matching-placebo (Pooled)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 7 (71.43%)
    Injury, poisoning and procedural complications
    Injury, Poisoning and Procedural Complications
         subjects affected / exposed
    2 / 7 (28.57%)
         occurrences all number
    2
    Nervous system disorders
    Nervous System Disorders
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Ear and Labyrinth Disorders
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Skin and Subcutaneous Tissue Disorders
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Psychiatric disorders
    Psychiatric Disorders
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Musculoskeletal and Connective Tissue Disorders
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1
    Infections and infestations
    Infections and Infestations
         subjects affected / exposed
    1 / 7 (14.29%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Apr 2023
    A category of medications was added to be used with caution and corresponding updates to exclusionary medications and disallowed concomitant medications.

    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.
    Sponsor decided to stop study based on operational & strategic considerations & not for reasons related to efficacy/safety.Due to early cessation of study before subjects reached timewindow for efficacy assessments,efficacy analysis was not possible.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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