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    Clinical Trial Results:
    A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington’s Disease

    Summary
    EudraCT number
    		 2020-004556-15
    Trial protocol
    		 DE   PL   FR   DK   ES   IT   NL  
    Global end of trial date
    24 Jun 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 Jun 2025
    First version publication date
    09 Jun 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    WVE-003-001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05032196
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Wave Life Sciences UK Limited
    Sponsor organisation address
    1 Chamberlain Square CS, Birmingham, United Kingdom, B33AX
    Public contact
    Medical Director, MD, Wave Life Sciences UK Limited, info@wavelifesci.com
    Scientific contact
    Medical Director, MD, Wave Life Sciences UK Limited, info@wavelifesci.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
    25 Jun 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    24 May 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    24 Jun 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of WVE-003 in patients with Huntington’s disease (HD).
    Protection of trial subjects
    The study was conducted according to the study protocol and standard operating procedures that meet the guidelines provided by the International Conference on Harmonisation for Good Clinical Practice in clinical studies, and any other applicable local regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    13 Aug 2021
    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
    Netherlands: 3
    Country: Number of subjects enrolled
    Poland: 6
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    Denmark: 2
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    Australia: 3
    Country: Number of subjects enrolled
    Canada: 10
    Worldwide total number of subjects
    47
    EEA total number of subjects
    29
    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)
    47
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Pts underwent prescreening to confirm they were heterozygous for SNP3 with the A variant on the same allele as the CAG triplet expansion. Prescreening was allowed to happen any time before Screening. The prescreening testing process was expected to take up to 6 weeks. If Pts met these criteria, they continued to the Screening visits.

    Period 1
    Period 1 title
    Period 1: Single Ascending Dose
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 SAD: Pooled Placebo
    Arm description
    		 SAD: Single dose of placebo.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    20mL of placebo for intrathecal injection.

    Arm title
    		 SAD: 30mg WVE-003
    Arm description
    		 SAD: Single ascending dose of 30mg WVE-003. 1 Pt was randomized to WVE-003 60mg SAD group but received 30mg instead. This Pt is counted in the 30mg arm .
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-003
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    20mL of 30mg WVE-003 for intrathecal injection

    Arm title
    		 SAD: 60mg WVE-003
    Arm description
    		 SAD: Single ascending dose of 60mg WVE-003.
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-003
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    20mL of 60mg WVE-003 for intrathecal injection.

    Arm title
    		 SAD: 90mg WVE-003
    Arm description
    		 SAD: Single ascending dose of 90mg WVE-003.
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-003
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    20mL of 90mg WVE-003 for intrathecal injection.

    Number of subjects in period 1
    		SAD: Pooled Placebo SAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003
    Started
    16
    13
    10
    8
    Completed
    16
    12
    10
    7
    Not completed
    0
    1
    0
    1
         Physician decision
    -
    1
    -
    -
         Adverse event, non-fatal
    -
    -
    -
    1
    Period 2
    Period 2 title
    Period 2: Multiple Dose
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 MD: Placebo
    Arm description
    		 MD: 3 doses of placebo once every 8 weeks (Q8W). 7 Pts from Period 1 rolled over into Period 2.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    20mL placebo for 3 intrathecal injections Q8W

    Arm title
    		 MD: 30mg WVE-003
    Arm description
    		 MD: 3 doses of 30mg WVE-003 Q8W. 16 Pts rolled over from Period 1 into Period 2.
    Arm type
    		 Experimental

    Investigational medicinal product name
    WVE-003
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    20mL of 30mg WVE-003 for 3 intrathecal injection Q8W

    Number of subjects in period 2 [1]
    		MD: Placebo MD: 30mg WVE-003
    Started
    7
    16
    Completed
    6
    16
    Not completed
    1
    0
         Physician decision
    1
    -
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: In this study, the number of subjects starting Period 2 is lower than the number of subjects completing Period 1, as per protocol.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    SAD: Pooled Placebo
    Reporting group description
    		 SAD: Single dose of placebo.

    Reporting group title
    SAD: 30mg WVE-003
    Reporting group description
    		 SAD: Single ascending dose of 30mg WVE-003. 1 Pt was randomized to WVE-003 60mg SAD group but received 30mg instead. This Pt is counted in the 30mg arm .

    Reporting group title
    SAD: 60mg WVE-003
    Reporting group description
    		 SAD: Single ascending dose of 60mg WVE-003.

    Reporting group title
    SAD: 90mg WVE-003
    Reporting group description
    		 SAD: Single ascending dose of 90mg WVE-003.

    Reporting group values
    		 SAD: Pooled Placebo SAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003 Total
    Number of subjects
    		 16 13 10 8 47
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0 0
        Adults (18-64 years)
    		 16 13 10 8 47
        From 65-84 years
    		 0 0 0 0 0
        85 years and over
    		 0 0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 45.5 ( 7.4 ) 47.8 ( 9.2 ) 43.9 ( 8.6 ) 49.6 ( 5.3 ) -
    Gender categorical
    Units: Subjects
        Female
    		 6 6 3 3 18
        Male
    		 10 7 7 5 29
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 1 0 0 0 1
        Not Hispanic or Latino
    		 15 13 10 8 46
        Unknown or not reported
    		 0 0 0 0 0
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0 0
        Asian
    		 0 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0 0
        Black or African American
    		 0 0 0 0 0
        White
    		 16 13 10 8 47
        More than one race
    		 0 0 0 0 0
        Unknown or Not Reported
    		 0 0 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    SAD: Pooled Placebo
    Reporting group description
    		 SAD: Single dose of placebo.

    Reporting group title
    SAD: 30mg WVE-003
    Reporting group description
    		 SAD: Single ascending dose of 30mg WVE-003. 1 Pt was randomized to WVE-003 60mg SAD group but received 30mg instead. This Pt is counted in the 30mg arm .

    Reporting group title
    SAD: 60mg WVE-003
    Reporting group description
    		 SAD: Single ascending dose of 60mg WVE-003.

    Reporting group title
    SAD: 90mg WVE-003
    Reporting group description
    		 SAD: Single ascending dose of 90mg WVE-003.
    Reporting group title
    MD: Placebo
    Reporting group description
    		 MD: 3 doses of placebo once every 8 weeks (Q8W). 7 Pts from Period 1 rolled over into Period 2.

    Reporting group title
    MD: 30mg WVE-003
    Reporting group description
    		 MD: 3 doses of 30mg WVE-003 Q8W. 16 Pts rolled over from Period 1 into Period 2.

    Primary: Primary: Safety: Proportion of Patients With Treatment-Emergent Adverse Events (TEAEs) related to study drug

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    End point title
    		 Primary: Safety: Proportion of Patients With Treatment-Emergent Adverse Events (TEAEs) related to study drug [1]
    End point description
    Proportion of Patients with Treatment Emergent Adverse Events (TEAEs) related to study drug.
    End point type
    Primary
    End point timeframe
    Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2)
    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: The primary endpoint of this study was safety, and as per protocol, the safety analysis was performed descriptively.
    End point values
    		 SAD: Pooled Placebo SAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003 MD: Placebo MD: 30mg WVE-003
    Number of subjects analysed
    16
    13
    10
    8
    7
    16
    Units: N Participants
    2
    1
    3
    3
    0
    8
    No statistical analyses for this end point

    Secondary: Pharmacokinetics of WVE-003 in Plasma - AUC0-6

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    End point title
    		 Pharmacokinetics of WVE-003 in Plasma - AUC0-6 [2]
    End point description
    AUC0-6 = area under the concentration-time curve from time 0 to 6 hrs
    End point type
    Secondary
    End point timeframe
    Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Since these endpoints consist of pharmacokinetic analysis measuring levels of WVE-003 product, the analyses performed on placebo were done only to confirm no WVE-003 was present in these samples.
    End point values
    		 SAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003 MD: 30mg WVE-003
    Number of subjects analysed
    13
    10
    8
    16
    Units: hr*ng/mL
    arithmetic mean (standard deviation)
        Day 1
    1000 ( 702 )
    2310 ( 3090 )
    3890 ( 2900 )
    822 ( 409 )
        Day 113
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
    698 ( 432 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetics of WVE-003 in Plasma - Cmax

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    End point title
    		 Pharmacokinetics of WVE-003 in Plasma - Cmax [3]
    End point description
    Cmax = maximum observed concentration.
    End point type
    Secondary
    End point timeframe
    Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Since these endpoints consist of pharmacokinetic analysis measuring levels of WVE-003 product, the analyses performed on placebo were done only to confirm no WVE-003 was present in these samples.
    End point values
    		 SAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003 MD: 30mg WVE-003
    Number of subjects analysed
    13
    10
    8
    16
    Units: ng/mL
    arithmetic mean (standard deviation)
        Day 1
    240 ( 168 )
    601 ( 774 )
    1061 ( 942 )
    186 ( 106 )
        Day 113
    0 ( 0 )
    0 ( 0 )
    0 ( 0 )
    157 ( 97.3 )
    No statistical analyses for this end point

    Secondary: Concentration of WVE-003 in cerebrospinal fluid (CSF)

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    End point title
    		 Concentration of WVE-003 in cerebrospinal fluid (CSF) [4]
    End point description
    WVE-003 concentration in cerebrospinal fluid (CSF) is reported in ng/mL.
    End point type
    Secondary
    End point timeframe
    28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141)
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Since these endpoints consist of pharmacokinetic analysis measuring levels of WVE-003 product, the analyses performed on placebo were done only to confirm no WVE-003 was present in these samples.
    End point values
    		 SAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003 MD: 30mg WVE-003
    Number of subjects analysed
    13
    10
    8
    16
    Units: ng/mL
        arithmetic mean (standard deviation)
    3.6405 ( 1.6133 )
    5.5346 ( 2.8402 )
    7.0983 ( 3.1335 )
    4.2903 ( 1.9722 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 52 (multi dose Period 2)
    Adverse event reporting additional description
    5 months after a patient completed their final safety visit, an SAE was reported that sponsor assessed to be not-related to WVE-003.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    SAD: Pooled Placebo
    Reporting group description
    		 SAD: Single dose of placebo.

    Reporting group title
    SAD: 30mg WVE-003
    Reporting group description
    		 SAD: 30mg WVE-003: Single ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

    Reporting group title
    SAD: 60mg WVE-003
    Reporting group description
    		 SAD: 60mg WVE-003: Single ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

    Reporting group title
    SAD: 90mg WVE-003
    Reporting group description
    		 SAD: 90mg WVE-003: Single ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

    Reporting group title
    MD: Placebo
    Reporting group description
    		 MD: Three doses of placebo Q8W

    Reporting group title
    MD: 30mg WVE-003
    Reporting group description
    		 MD: 30mg WVE-003: Three doses of 30mg WVE-003 Q8W, an allele-selective stereopure antisense oligonucleotide (ASO)

    Serious adverse events
    		 SAD: Pooled Placebo SAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003 MD: Placebo MD: 30mg WVE-003
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Post lumbar puncture syndrome
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Ataxia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 SAD: Pooled Placebo SAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003 MD: Placebo MD: 30mg WVE-003
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    13 / 16 (81.25%)
    9 / 13 (69.23%)
    8 / 10 (80.00%)
    8 / 8 (100.00%)
    7 / 7 (100.00%)
    13 / 16 (81.25%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Haematoma
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 16 (12.50%)
    2 / 13 (15.38%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    1 / 7 (14.29%)
    2 / 16 (12.50%)
         occurrences all number
    3
    2
    0
    1
    4
    7
    Influenza like illness
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    2 / 16 (12.50%)
         occurrences all number
    0
    0
    0
    1
    0
    5
    Puncture site pain
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    5
    0
    0
    0
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Vessel puncture site haematoma
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Reproductive system and breast disorders
    Amenorrhoea
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Intermenstrual bleeding
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Nasal congestion
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Attention deficit hyperactivity disorder
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Compulsions
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Depressed mood
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Depression
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    1 / 7 (14.29%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Disorientation
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Insomnia
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Irritability
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Sleep disorder
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    C-reactive protein increased
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    CSF protein increased
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 13 (15.38%)
    1 / 10 (10.00%)
    2 / 8 (25.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    2
    1
    2
    0
    1
    CSF red blood cell count positive
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Concussion
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Eye injury
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Fall
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    3 / 8 (37.50%)
    2 / 7 (28.57%)
    2 / 16 (12.50%)
         occurrences all number
    1
    0
    1
    4
    4
    2
    Head injury
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Injury
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Ligament sprain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Limb injury
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Lip injury
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Post lumbar puncture syndrome
         subjects affected / exposed
    3 / 16 (18.75%)
    1 / 13 (7.69%)
    1 / 10 (10.00%)
    2 / 8 (25.00%)
    1 / 7 (14.29%)
    0 / 16 (0.00%)
         occurrences all number
    2
    4
    1
    6
    1
    0
    Procedural complication
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Procedural pain
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    2 / 8 (25.00%)
    1 / 7 (14.29%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    2
    1
    0
    Road traffic accident
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Skin abrasion
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Skin laceration
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    2 / 16 (12.50%)
         occurrences all number
    0
    0
    1
    0
    0
    2
    Upper limb fracture
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    CSF white blood cell count increased
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    2 / 8 (25.00%)
    0 / 7 (0.00%)
    2 / 16 (12.50%)
         occurrences all number
    0
    1
    0
    2
    0
    2
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Nervous system disorders
    Balance disorder
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    5
    0
    3
    Coordination abnormal
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    3
    Dizziness
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    0
    0
    0
    1
    Dyskinesia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Headache
         subjects affected / exposed
    6 / 16 (37.50%)
    4 / 13 (30.77%)
    2 / 10 (20.00%)
    4 / 8 (50.00%)
    3 / 7 (42.86%)
    4 / 16 (25.00%)
         occurrences all number
    14
    5
    2
    7
    4
    5
    Hyperaesthesia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Hyporeflexia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Intracranial hypotension
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    Paraesthesia
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    2
    0
    0
    0
    0
    1
    Paresis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Pleocytosis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    Presyncope
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    Sciatica
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Tension headache
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Blood and lymphatic system disorders
    Eosinophilia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Eye disorders
    Eye pain
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Constipation
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    Dental caries
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Diarrhoea
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    2
    0
    0
    0
    0
    1
    Nausea
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 13 (7.69%)
    0 / 10 (0.00%)
    2 / 8 (25.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    2
    0
    0
    Toothache
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Vomiting
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Eczema
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Rash
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Rash erythematous
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Skin irritation
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Back pain
         subjects affected / exposed
    1 / 16 (6.25%)
    3 / 13 (23.08%)
    2 / 10 (20.00%)
    1 / 8 (12.50%)
    2 / 7 (28.57%)
    0 / 16 (0.00%)
         occurrences all number
    1
    4
    3
    1
    2
    0
    Muscle contracture
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Myalgia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    2
    0
    0
    Pain in extremity
         subjects affected / exposed
    0 / 16 (0.00%)
    2 / 13 (15.38%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Infected seroma
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Influenza
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    2 / 16 (12.50%)
         occurrences all number
    0
    0
    0
    0
    1
    2
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 16 (0.00%)
         occurrences all number
    1
    0
    0
    0
    1
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Contusion
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 13 (0.00%)
    1 / 10 (10.00%)
    1 / 8 (12.50%)
    1 / 7 (14.29%)
    1 / 16 (6.25%)
         occurrences all number
    1
    0
    1
    1
    1
    1
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 13 (0.00%)
    0 / 10 (0.00%)
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 May 2021
    Amendment 1, version 2: updated protocol with modified Period 2 stopping criteria, clarified timing of effective contraception, minor exclusion criteria, and language regarding specific guidance to Investigators on disposition and follow-up of participants experiencing severe and/or serious laboratory abnormalities.
    06 Sep 2022
    Amendment 2, version 3: updated to clarify endpoints
    26 Sep 2022
    Amendment 4, version 5: Amendment 3 not implemented but changes therein were contained in Amendment 4 (Updated to further clarify endpoints, clarified that the number of cohorts and participant numbers may be modified as per DEC/ SMC recommendation. Clarified participant completion required in Period 1 to be eligible for Period 2.). Updated study design for Period 2 cohort, based on data and enrollment in Period 1. Clarified that the follow-up of up to 24 weeks post-dose.

    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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