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    Clinical Trial Results:
    A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Subjects who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)

    Summary
    EudraCT number
    		 2017-002471-25
    Trial protocol
    		 GB   DE  
    Global end of trial date
    08 Oct 2019

    Results information
    Results version number
    		 v2(current)
    This version publication date
    17 Feb 2022
    First version publication date
    13 Nov 2020
    Other versions
    		 v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    BN40697
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03342053
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Hoffmann-La Roche
    Sponsor organisation address
    Grenzacherstrasse 124, Basel, Switzerland, CH-4070
    Public contact
    F. Hoffmann-La Roche AG, F. Hoffmann-La Roche AG, +41 616878333, global.trial_information@roche.com
    Scientific contact
    Medical Communications, Hoffmann-La Roche, +41 616878333, global.trial_information@roche.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
    11 Jul 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Oct 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This study tested the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7234292 administered intrathecally to adult patients with Huntington's Disease.
    Protection of trial subjects
    This study was conducted in accordance with the protocol and with the following: ● Consensus ethical principles derived from international guidelines including the Declaration of Helsinki and Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines ● Applicable ICH Good Clinical Practice (GCP) Guidelines ● Applicable laws and regulations
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Jan 2018
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 6
    Country: Number of subjects enrolled
    Germany: 19
    Country: Number of subjects enrolled
    United Kingdom: 21
    Worldwide total number of subjects
    46
    EEA total number of subjects
    19
    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)
    43
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participant eligibility for the study was determined within 4 weeks prior to participant entry into the Treatment Period.

    Pre-assignment
    Screening details
    		 This study is an OLE for patients who participated in Study ISIS 443139-CS1. Each subject was assigned to the same screening and subject identification numbers as in the prior MAD study (Study ISIS 443139-CS1).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    Randomised, open label with no control

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 RO7234292 Monthly
    Arm description
    		 RO7234292 was administered intrathecally every 28 days for 14 months.
    Arm type
    		 Experimental

    Investigational medicinal product name
    RO7234292 (RG6042)
    Investigational medicinal product code
    Other name
    Tominersen
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    RO7234292 is administered intrathecally every 28 days for 14 months at a dose of 120mg.

    Arm title
    		 RO7234292 Bimonthly
    Arm description
    		 RO7234292 was administered intrathecally every 56 days for 14 months following 2 monthly doses to serve as a loading dose.
    Arm type
    		 Experimental

    Investigational medicinal product name
    RO7234292 (RG6042)
    Investigational medicinal product code
    Other name
    Tominersen
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    RO7234292 is administered intrathecally every 56 days at a dose of 120mg for 14 months following 2 monthly doses to serve as a loading dose.

    Number of subjects in period 1
    		RO7234292 Monthly RO7234292 Bimonthly
    Started
    23
    23
    Completed
    21
    22
    Not completed
    2
    1
         Adverse event, serious fatal
    1
    -
         Consent withdrawn by subject
    -
    1
         Adverse event, non-fatal
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    RO7234292 Monthly
    Reporting group description
    		 RO7234292 was administered intrathecally every 28 days for 14 months.

    Reporting group title
    RO7234292 Bimonthly
    Reporting group description
    		 RO7234292 was administered intrathecally every 56 days for 14 months following 2 monthly doses to serve as a loading dose.

    Reporting group values
    		 RO7234292 Monthly RO7234292 Bimonthly Total
    Number of subjects
    		 23 23 46
    Age Categorical
    Units: Participants
        <=18 years
    		 0 0 0
        Between 18 and 65 years
    		 23 20 43
        >=65 years
    		 0 3 3
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 47.7 ± 9.3 49.5 ± 11.3 -
    Sex: Female, Male
    Units: Participants
        Female
    		 8 10 18
        Male
    		 15 13 28
    Race (NIH/OMB)
    One participant's race is known, was reported but cannot be classified in Roche database.
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 0 0 0
        White
    		 22 23 45
        More than one race
    		 0 0 0
        Other
    		 1 0 1
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 23 23 46
        Unknown or Not Reported
    		 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    RO7234292 Monthly
    Reporting group description
    		 RO7234292 was administered intrathecally every 28 days for 14 months.

    Reporting group title
    RO7234292 Bimonthly
    Reporting group description
    		 RO7234292 was administered intrathecally every 56 days for 14 months following 2 monthly doses to serve as a loading dose.

    Primary: Percentage of Participants with Treatment-Emergent Adverse Events (AEs)

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    End point title
    		 Percentage of Participants with Treatment-Emergent Adverse Events (AEs) [1]
    End point description
    Descriptive summary of percentage of participants with treatment emergent AEs
    End point type
    Primary
    End point timeframe
    From baseline up to 1 year 9 months
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistics was planned to be reported in the endpoint.
    End point values
    		 RO7234292 Monthly RO7234292 Bimonthly
    Number of subjects analysed
    23
    23
    Units: Percentage
    number (not applicable)
        Percentage of Subjects with Treatment-Emergent AEs
    100
    95.7
    No statistical analyses for this end point

    Secondary: RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)

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    End point title
    		 RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)
    End point description
    Here "9999" means that value is not available (NA). Data for Bi-monthly arm was not collected on days 57, 113, 169, 225, 281, 337, 393.
    End point type
    Secondary
    End point timeframe
    From baseline to Day 421
    End point values
    		 RO7234292 Monthly RO7234292 Bimonthly
    Number of subjects analysed
    23
    23
    Units: ng/mL
    geometric mean (full range (min-max))
        Day 29
    2.59 (1.31 to 12.7)
    2.52 (0.817 to 7.85)
        Day 57
    3.47 (1.43 to 19.2)
    9999 (9999 to 9999)
        Day 85
    3.70 (1.38 to 7.34)
    1.39 (0.164 to 5.74)
        Day 113
    3.97 (1.96 to 16.4)
    9999 (9999 to 9999)
        Day 141
    4.57 (1.72 to 13)
    1.35 (0.396 to 3.69)
        Day 169
    4.47 (2.04 to 12.7)
    9999 (9999 to 9999)
        Day 197
    4.58 (1.74 to 8.61)
    1.26 (0.281 to 3.37)
        Day 225
    5.21 (1.63 to 14.3)
    9999 (9999 to 9999)
        Day 253
    4.96 (1.56 to 11.8)
    1.45 (0.417 to 3.76)
        Day 281
    4.96 (1.30 to 12.4)
    9999 (9999 to 9999)
        Day 309
    5.53 (1.74 to 11.6)
    1.35 (0.195 to 2.77)
        Day 337
    5.12 (2.17 to 10.5)
    9999 (9999 to 9999)
        Day 365
    4.50 (1.18 to 13.9)
    1.45 (0.507 to 2.95)
        Day 393
    3.10 (0.220 to 9.83)
    9999 (9999 to 9999)
        Day 421
    3.01 (0.239 to 9.54)
    1.34 (0.325 to 2.45)
    No statistical analyses for this end point

    Secondary: CSF mHTT Protein Concentration Change in Geometric Mean (95%CI) from Baseline

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    End point title
    		 CSF mHTT Protein Concentration Change in Geometric Mean (95%CI) from Baseline
    End point description
    The results of the planned analysis related to mHTT protein levels in CSF are reported. Log (10) fmol/L changes in geometric mean (95%CI) were reported. Here "999" means that the values are not available (NA)
    End point type
    Secondary
    End point timeframe
    From Baseline to Day 421
    End point values
    		 RO7234292 Monthly RO7234292 Bimonthly
    Number of subjects analysed
    23 [2]
    23 [3]
    Units: Log (10) fmol/L
    geometric mean (confidence interval 95%)
        Day 29
    -27.95 (-38.37 to -15.76)
    -21.84 (-33.22 to -8.52)
        Day 57
    -26.90 (-40.95 to -9.50)
    10.11 (-41.68 to 107.88)
        Day 85
    -50.30 (-59.00 to -39.75)
    -32.63 (-44.31 to -18.50)
        Day 113
    -49.13 (-58.77 to -37.18)
    999 (999 to 999)
        Day 141
    -54.02 (-61.05 to -45.72)
    -41.76 (-50.59 to -31.35)
        Day 169
    -42.33 (-53.66 to -28.23)
    999 (999 to 999)
        Day 197
    -40.79 (-52.59 to -26.05)
    -36.48 (-48.99 to -20.89)
        Day 225
    -47.01 (-58.69 to -32.02)
    999 (999 to 999)
        Day 253
    -43.63 (-55.39 to -28.77)
    -41.06 (-53.19 to -25.78)
        Day 281
    -40.29 (-58.63 to -13.80)
    999 (999 to 999)
        Day 309
    -36.67 (-50.11 to -19.60)
    -40.82 (-52.37 to -26.46)
        Day 337
    -42.71 (-58.93 to -20.07)
    999 (999 to 999)
        Day 365
    -49.55 (-62.02 to -32.98)
    -55.07 (-65.46 to -41.57)
        Day 393
    -41.55 (-59.46 to -15.74)
    999 (999 to 999)
        Day 421
    -45.45 (-56.21 to -32.03)
    -41.51 (-52.22 to -28.39)
    Notes
    [2] - Only subjects for whom data were collected are included in the analysis.
    [3] - Only subjects for whom data were collected are included in the analysis.
    No statistical analyses for this end point

    Secondary: Mean Percentage Change in Ventricular Volume Boundary Shift Integral from Baseline to 15 months

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    End point title
    		 Mean Percentage Change in Ventricular Volume Boundary Shift Integral from Baseline to 15 months
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline up to 15 months
    End point values
    		 RO7234292 Monthly RO7234292 Bimonthly
    Number of subjects analysed
    20
    18
    Units: Percentage
        arithmetic mean (standard deviation)
    46.09 ± 32.14
    18.77 ± 10.36
    No statistical analyses for this end point

    Secondary: Mean Percentage Change in Caudate Volume Boundary Shift Integral from Baseline to 15 months

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    End point title
    		 Mean Percentage Change in Caudate Volume Boundary Shift Integral from Baseline to 15 months
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline up to 15 months
    End point values
    		 RO7234292 Monthly RO7234292 Bimonthly
    Number of subjects analysed
    22
    18
    Units: Percentage
        arithmetic mean (standard deviation)
    8.64 ± 6.26
    5.67 ± 2.22
    No statistical analyses for this end point

    Secondary: Mean Percentage Change in Whole Brain Volume Boundary Shift Integral from Baseline to 15 months

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    End point title
    		 Mean Percentage Change in Whole Brain Volume Boundary Shift Integral from Baseline to 15 months
    End point description
    The numbers of analyzed subjects are lower than the actual subjects at that time point because some of the images did not pass quality review and data was lost.
    End point type
    Secondary
    End point timeframe
    Baseline up to 15 months
    End point values
    		 RO7234292 Monthly RO7234292 Bimonthly
    Number of subjects analysed
    13
    18
    Units: Percentage
        arithmetic mean (standard deviation)
    1.63 ± 1.41
    0.89 ± 0.92
    No statistical analyses for this end point

    Secondary: EEG Parameters: Mean Change from Baseline to 15 Months in Absolute Power [8-12Hz]

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    End point title
    		 EEG Parameters: Mean Change from Baseline to 15 Months in Absolute Power [8-12Hz]
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 15 Months
    End point values
    		 RO7234292 Monthly RO7234292 Bimonthly
    Number of subjects analysed
    17
    19
    Units: log10(uV2)
        arithmetic mean (standard deviation)
    0.11 ± 0.18
    0.02 ± 0.21
    No statistical analyses for this end point

    Secondary: Mean Change from Baseline in Huntington's Disease Cognitive Assessment Battery Composite Score

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    End point title
    		 Mean Change from Baseline in Huntington's Disease Cognitive Assessment Battery Composite Score
    End point description
    Huntington's Disease Cognitive Assessment Battery Composite Score measures cognitive function. A positive change from baseline indicates improvement in cognitive function; a negative change indicates worsening in cognitive function.
    End point type
    Secondary
    End point timeframe
    Baseline to 15 Months
    End point values
    		 RO7234292 Monthly RO7234292 Bimonthly
    Number of subjects analysed
    17
    21
    Units: z-score
    arithmetic mean (standard deviation)
        Mean change from baseline
    -0.33 ± 0.27
    -0.15 ± 0.23
    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 1 year 9 months
    Adverse event reporting additional description
    Safety population comprising all subjects that were randomized and received at least one dose of RO7234292
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    RO7234292 Bimonthly
    Reporting group description
    		 RO7234292 was administered intrathecally every 56 days for 14 months following 2 monthly doses to serve as a loading dose.

    Reporting group title
    RO7234292 Monthly
    Reporting group description
    		 RO7234292 was administered intrathecally every 28 days for 14 months.

    Serious adverse events
    		 RO7234292 Bimonthly RO7234292 Monthly
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 23 (13.04%)
    4 / 23 (17.39%)
         number of deaths (all causes)
    0
    1
         number of deaths resulting from adverse events
    0
    0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 23 (4.35%)
    0 / 23 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin laceration
         subjects affected / exposed
    1 / 23 (4.35%)
    0 / 23 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cervical vertebral fracture
         subjects affected / exposed
    1 / 23 (4.35%)
    0 / 23 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Chest injury
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Concussion
         subjects affected / exposed
    1 / 23 (4.35%)
    0 / 23 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Meningitis chemical
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Rib fracture
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Spinal column injury
         subjects affected / exposed
    1 / 23 (4.35%)
    0 / 23 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Thoracic vertebral fracture
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Cerebrovascular accident
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hemiparesis
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hydrocephalus
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hyporeflexia
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Neuritis
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Radiculopathy
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pneumothorax
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Completed suicide
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Suicide attempt
         subjects affected / exposed
    1 / 23 (4.35%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Myelitis
         subjects affected / exposed
    0 / 23 (0.00%)
    1 / 23 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 RO7234292 Bimonthly RO7234292 Monthly
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    22 / 23 (95.65%)
    22 / 23 (95.65%)
    Investigations
    CSF white blood cell count increased
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    CSF protein increased
         subjects affected / exposed
    1 / 23 (4.35%)
    2 / 23 (8.70%)
         occurrences all number
    1
    3
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    12 / 23 (52.17%)
    18 / 23 (78.26%)
         occurrences all number
    17
    87
    Contusion
         subjects affected / exposed
    5 / 23 (21.74%)
    6 / 23 (26.09%)
         occurrences all number
    5
    21
    Head injury
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Ligament sprain
         subjects affected / exposed
    2 / 23 (8.70%)
    1 / 23 (4.35%)
         occurrences all number
    2
    1
    Limb injury
         subjects affected / exposed
    0 / 23 (0.00%)
    3 / 23 (13.04%)
         occurrences all number
    0
    3
    Post lumbar puncture syndrome
         subjects affected / exposed
    5 / 23 (21.74%)
    4 / 23 (17.39%)
         occurrences all number
    6
    10
    Skin abrasion
         subjects affected / exposed
    4 / 23 (17.39%)
    7 / 23 (30.43%)
         occurrences all number
    6
    11
    Procedural pain
         subjects affected / exposed
    12 / 23 (52.17%)
    7 / 23 (30.43%)
         occurrences all number
    15
    19
    Procedural headache
         subjects affected / exposed
    2 / 23 (8.70%)
    0 / 23 (0.00%)
         occurrences all number
    4
    0
    Skin laceration
         subjects affected / exposed
    3 / 23 (13.04%)
    2 / 23 (8.70%)
         occurrences all number
    3
    2
    Vascular disorders
    Haematoma
         subjects affected / exposed
    2 / 23 (8.70%)
    4 / 23 (17.39%)
         occurrences all number
    2
    7
    Nervous system disorders
    Cerebral ventricle dilatation
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Balance disorder
         subjects affected / exposed
    1 / 23 (4.35%)
    3 / 23 (13.04%)
         occurrences all number
    1
    3
    Dizziness
         subjects affected / exposed
    2 / 23 (8.70%)
    2 / 23 (8.70%)
         occurrences all number
    2
    2
    Dysarthria
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    3
    Dyskinesia
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Headache
         subjects affected / exposed
    4 / 23 (17.39%)
    6 / 23 (26.09%)
         occurrences all number
    10
    13
    Hyperkinesia
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Lumbar radiculopathy
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Motor dysfunction
         subjects affected / exposed
    0 / 23 (0.00%)
    3 / 23 (13.04%)
         occurrences all number
    0
    3
    Parkinsonism
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Paraesthesia
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Presyncope
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Syncope
         subjects affected / exposed
    2 / 23 (8.70%)
    0 / 23 (0.00%)
         occurrences all number
    2
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 23 (4.35%)
    3 / 23 (13.04%)
         occurrences all number
    1
    3
    Gait disturbance
         subjects affected / exposed
    0 / 23 (0.00%)
    6 / 23 (26.09%)
         occurrences all number
    0
    14
    Puncture site pain
         subjects affected / exposed
    2 / 23 (8.70%)
    2 / 23 (8.70%)
         occurrences all number
    4
    3
    Injection site pain
         subjects affected / exposed
    4 / 23 (17.39%)
    2 / 23 (8.70%)
         occurrences all number
    4
    3
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    5 / 23 (21.74%)
    0 / 23 (0.00%)
         occurrences all number
    6
    0
    Nausea
         subjects affected / exposed
    3 / 23 (13.04%)
    3 / 23 (13.04%)
         occurrences all number
    5
    3
    Vomiting
         subjects affected / exposed
    2 / 23 (8.70%)
    2 / 23 (8.70%)
         occurrences all number
    2
    2
    Respiratory, thoracic and mediastinal disorders
    Rhinorrhoea
         subjects affected / exposed
    2 / 23 (8.70%)
    1 / 23 (4.35%)
         occurrences all number
    3
    1
    Cough
         subjects affected / exposed
    2 / 23 (8.70%)
    0 / 23 (0.00%)
         occurrences all number
    2
    0
    Skin and subcutaneous tissue disorders
    Hyperhidrosis
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    3
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Irritability
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    3
    Insomnia
         subjects affected / exposed
    2 / 23 (8.70%)
    1 / 23 (4.35%)
         occurrences all number
    2
    1
    Depression
         subjects affected / exposed
    2 / 23 (8.70%)
    4 / 23 (17.39%)
         occurrences all number
    2
    4
    Depressed mood
         subjects affected / exposed
    2 / 23 (8.70%)
    2 / 23 (8.70%)
         occurrences all number
    2
    2
    Tension
         subjects affected / exposed
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 23 (8.70%)
    3 / 23 (13.04%)
         occurrences all number
    2
    4
    Pain in extremity
         subjects affected / exposed
    2 / 23 (8.70%)
    3 / 23 (13.04%)
         occurrences all number
    2
    6
    Back pain
         subjects affected / exposed
    4 / 23 (17.39%)
    2 / 23 (8.70%)
         occurrences all number
    6
    2
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 23 (4.35%)
    2 / 23 (8.70%)
         occurrences all number
    1
    2
    Ear infection
         subjects affected / exposed
    0 / 23 (0.00%)
    3 / 23 (13.04%)
         occurrences all number
    0
    3
    Nasopharyngitis
         subjects affected / exposed
    10 / 23 (43.48%)
    9 / 23 (39.13%)
         occurrences all number
    15
    14
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 23 (4.35%)
    3 / 23 (13.04%)
         occurrences all number
    2
    4
    Urinary tract infection
         subjects affected / exposed
    2 / 23 (8.70%)
    2 / 23 (8.70%)
         occurrences all number
    4
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Sep 2017
    Amendment 1 (Protocol version 2) made minor changes for consistency and to promote clarity prior to patient enrolment.
    30 Jan 2018
    Amendment 2 (Protocol version 3) changed the study design for the less frequent (quarterly, Q12W) dosing regimen to ensure that all study subjects would be exposed to a dose schedule that was predicted to be sufficient to provide clinical benefit if the drug acted as intended.
    29 Jun 2018
    Amendment 3 (Protocol version 4) included the option for the Sponsor to conduct one or more interim analyses to support internal decision-making on the overall RO7234292 clinical development program.
    30 Aug 2018
    Amendment 4 (Protocol version 5) reflected a change in sponsorship for the study and overall RO7234292 development from Ionis Pharmaceuticals, Inc. to F. Hoffmann-La Roche Ltd. and related updates to study drug name and manufacturer as well as to the assigned medical monitor. Provision was made for post-trial access to treatment through an OLE study BN40955 and for collection of subjects’ HDID numbers to enable data from this study to be linked with data from other studies and registries.

    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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