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    Clinical Trial Results:
    A Two Part Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of SBT-020 in Patients with Early Stage Huntington’s Disease.

    Summary
    EudraCT number
    		 2016-003730-25
    Trial protocol
    		 NL  
    Global end of trial date
    21 Dec 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    29 Nov 2021
    First version publication date
    29 Nov 2021
    Other versions
    Summary report(s)
    		 CSR Synopsis

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    SBT20-102
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Stealth BioTherapeutics Inc
    Sponsor organisation address
    140 Kendrick Street, Building C-West, Needham, United States, MA 02494
    Public contact
    Jim Carr, PharmD, Chief Clinical Development Officer, Stealth BioTherapeutics Inc, +1 617 600-6888, jim.carr@stealthbt.com
    Scientific contact
    Jim Carr, PharmD, Chief Clinical Development Officer, Stealth BioTherapeutics Inc, +1 617 600-6888, jim.carr@stealthbt.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
    05 Oct 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    21 Dec 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Dec 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Part 1: Primary Objective -To assess the safety of SBT-020 in early stage HD patients Part 2: Primary Objective -To assess the safety and tolerability of longer term treatment with SBT-020 in early stage HD patients.
    Protection of trial subjects
    This study was performed in compliance with International Council for Harmonisation (ICH) Good Clinical Practices (GCP), including the archiving of essential documents as well as the ethical principles of the Declaration of Helsinki. A Dose Escalation Committee analysed safety data before allowing dose escalations. The Dose Escalation Committee consisted of representatives from the investigational site and sponsor and included both the Principal Investigator and Sponsor Medical Monitor.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    14 Apr 2017
    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
    Netherlands: 24
    Worldwide total number of subjects
    24
    EEA total number of subjects
    24
    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)
    24
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 One study center: Centre for Human Drug Research (CHDR), Zernikedreef 8 Leiden 2333 CL, the Netherlands First subject, first dose: 14 Apr 2017; Last subject, last visit: 21 Dec 2017

    Pre-assignment
    Screening details
    		 Male and female subjects aged at least 18 years with a DNA confirmed diagnosis of of mild to moderate Huntington Disease. In Part 1 a total of 24 subjects were randomized to 3 dosing cohorts. In Part 2, a total of 24 subjects were to be enrolled, however one subject did not continue into the second part of the study (23 subjects were analysed).

    Period 1
    Period 1 title
    Part 1
    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
    		 5 mg/day SBT020
    Arm description
    		 6 subjects were randomised to 5 mg/day SBT020
    Arm type
    		 Experimental

    Investigational medicinal product name
    SBT-020
    Investigational medicinal product code
    SBT-020
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    SBT-020 as supplied as lyophilized powder (44 mg/vial) for reconstitution with sterile saline for injection. SBT-020 was administered as a once daily SC injection for 7 days.

    Arm title
    		 5 mg/day Placebo
    Arm description
    		 2 subjects were randomised to 5 mg/day Placebo
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    The placebo for this trial was composed of sterile saline for injection. The placebo was handled and administered identically to the active drug.

    Arm title
    		 15 mg/day SBT020
    Arm description
    		 6 subjects randomised to 15 mg/day SBT020
    Arm type
    		 Experimental

    Investigational medicinal product name
    SBT-020
    Investigational medicinal product code
    SBT-020
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    SBT-020 as supplied as lyophilized powder (44 mg/vial) for reconstitution with sterile saline for injection. SBT-020 was administered as a once daily SC injection for 7 days.

    Arm title
    		 15 mg/day Placebo
    Arm description
    		 2 subjects randomised to 15 mg/day Placebo
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    The placebo for this trial was composed of sterile saline for injection. The placebo was handled and administered identically to the active drug.

    Arm title
    		 25 mg/day SBT020
    Arm description
    		 6 subjects randomised to 25 mg/day SBT020
    Arm type
    		 Experimental

    Investigational medicinal product name
    SBT-020
    Investigational medicinal product code
    SBT-020
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    SBT-020 as supplied as lyophilized powder (44 mg/vial) for reconstitution with sterile saline for injection. SBT-020 was administered as a once daily SC injection for 7 days.

    Arm title
    		 25 mg/day Placebo
    Arm description
    		 2 subjects randomised to 25 mg/day Placebo
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    The placebo for this trial was composed of sterile saline for injection. The placebo was handled and administered identically to the active drug.

    Number of subjects in period 1
    		5 mg/day SBT020 5 mg/day Placebo 15 mg/day SBT020 15 mg/day Placebo 25 mg/day SBT020 25 mg/day Placebo
    Started
    6
    2
    6
    2
    6
    2
    Completed
    6
    2
    6
    2
    6
    2
    Period 2
    Period 2 title
    Wash-out
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Arm title
    		 Wash-out
    Arm description
    		 A 28-day period when patients from Part 1 did not receive study drug (SBT020 or placebo)
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 2
    		Wash-out
    Started
    24
    Completed
    23
    Not completed
    1
         Adverse event, non-fatal
    1
    Period 3
    Period 3 title
    Part 2
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Monitor, Data analyst, Carer, Subject, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 25 mg/day SBT020
    Arm description
    		 11 subjects were randomised to SBT020 25 mg/day
    Arm type
    		 Experimental

    Investigational medicinal product name
    SBT-020
    Investigational medicinal product code
    SBT-020
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    SBT-020 as supplied as lyophilized powder (44 mg/vial) for reconstitution with sterile saline for injection. SBT-020 was administered as a once daily SC injection for 28 days.

    Arm title
    		 25 mg/day Placebo
    Arm description
    		 12 subjects were randomised to Placebo 25 mg/day
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    The placebo for this trial was composed of sterile saline for injection. The placebo was handled and administered identically to the active drug.

    Number of subjects in period 3
    		25 mg/day SBT020 25 mg/day Placebo
    Started
    11
    12
    Completed
    11
    12

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Part 1
    Reporting group description
    		 Part 1 study population

    Reporting group values
    		 Part 1 Total
    Number of subjects
    		 24 24
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 24 24
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 47.5 ( 9.3 ) -
    Gender categorical
    Units: Subjects
        Female
    		 11 11
        Male
    		 13 13

    End points

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    End points reporting groups
    Reporting group title
    5 mg/day SBT020
    Reporting group description
    		 6 subjects were randomised to 5 mg/day SBT020

    Reporting group title
    5 mg/day Placebo
    Reporting group description
    		 2 subjects were randomised to 5 mg/day Placebo

    Reporting group title
    15 mg/day SBT020
    Reporting group description
    		 6 subjects randomised to 15 mg/day SBT020

    Reporting group title
    15 mg/day Placebo
    Reporting group description
    		 2 subjects randomised to 15 mg/day Placebo

    Reporting group title
    25 mg/day SBT020
    Reporting group description
    		 6 subjects randomised to 25 mg/day SBT020

    Reporting group title
    25 mg/day Placebo
    Reporting group description
    		 2 subjects randomised to 25 mg/day Placebo
    Reporting group title
    Wash-out
    Reporting group description
    		 A 28-day period when patients from Part 1 did not receive study drug (SBT020 or placebo)
    Reporting group title
    25 mg/day SBT020
    Reporting group description
    		 11 subjects were randomised to SBT020 25 mg/day

    Reporting group title
    25 mg/day Placebo
    Reporting group description
    		 12 subjects were randomised to Placebo 25 mg/day

    Primary: General TEAEs

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    End point title
    		 General TEAEs
    End point description
    End point type
    Primary
    End point timeframe
    Part 1 and Part 2
    End point values
    		 5 mg/day SBT020 5 mg/day Placebo 15 mg/day SBT020 15 mg/day Placebo 25 mg/day SBT020 25 mg/day Placebo 25 mg/day SBT020 25 mg/day Placebo
    Number of subjects analysed
    6
    2
    6
    2
    6
    2
    11
    12
    Units: Events
    9
    3
    4
    3
    6
    4
    8
    23
    Statistical analysis title
    		 TEAE Descriptive
    Statistical analysis description
    Descriptive
    Comparison groups
    5 mg/day SBT020 v 5 mg/day Placebo v 15 mg/day SBT020 v 15 mg/day Placebo v 25 mg/day SBT020 v 25 mg/day Placebo v 25 mg/day SBT020 v 25 mg/day Placebo
    Number of subjects included in analysis
    47
    Analysis specification
    Pre-specified
    Analysis type
    		 other [1]
    P-value
    		 = 0.05 [2]
    Method
    		 No p-value analysis
    Parameter type
    		 Sum
    Confidence interval
    Notes
    [1] - Descriptive numerical
    [2] - No formal p-value analysis was performed for safety analysis

    Primary: Injection Site Reactions

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    End point title
    		 Injection Site Reactions
    End point description
    End point type
    Primary
    End point timeframe
    Part 1 and Part 2
    End point values
    		 5 mg/day SBT020 5 mg/day Placebo 15 mg/day SBT020 15 mg/day Placebo 25 mg/day SBT020 25 mg/day Placebo 25 mg/day SBT020 25 mg/day Placebo
    Number of subjects analysed
    6
    2
    6
    2
    6
    2
    11
    12
    Units: Events
    32
    1
    61
    2
    93
    2
    423
    42
    Statistical analysis title
    		 Decsriptive numerical
    Statistical analysis description
    Numerical comparison of events
    Comparison groups
    5 mg/day SBT020 v 5 mg/day Placebo v 15 mg/day SBT020 v 15 mg/day Placebo v 25 mg/day SBT020 v 25 mg/day Placebo v 25 mg/day SBT020 v 25 mg/day Placebo
    Number of subjects included in analysis
    47
    Analysis specification
    Pre-specified
    Analysis type
    		 other [3]
    P-value
    		 = 0.05 [4]
    Method
    		 No p-value analysis
    Parameter type
    		 events
    Confidence interval
    Notes
    [3] - Numerical comparison
    [4] - No p-value analysis was performed

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From time of informed consent until completion of the study follow-up visit. Adverse events occurring at any time following the first administration of study drug were considered treatment emergent (TEAE).
    Adverse event reporting additional description
    We report only the TEAEs
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20
    Reporting groups
    Reporting group title
    Part 1 - 5 mg/day SBT020
    Reporting group description
    		 6 subjects were randomised to 5 mg/day SBT020

    Reporting group title
    Part 1 - 15 mg/day SBT020
    Reporting group description
    		 6 subjects randomised to 15 mg/day SBT020

    Reporting group title
    Part 2 - 25 mg/day SBT020
    Reporting group description
    		 6 subjects randomised to 25 mg/day SBT020

    Reporting group title
    Part 2 - 25 mg/day Placebo
    Reporting group description
    		 12 subjects randomised to 25 mg/day Placebo

    Reporting group title
    Part 1 - 25 mg/day SBT020
    Reporting group description
    		 25 mg SBT-020 per day

    Reporting group title
    Washout period
    Reporting group description
    		 Off-drug period between Part 1 and Part 2

    Reporting group title
    Part 1 - Placebo
    Reporting group description
    		 Placebo patients combined from all 3 Part 1 arms

    Serious adverse events
    		 Part 1 - 5 mg/day SBT020 Part 1 - 15 mg/day SBT020 Part 2 - 25 mg/day SBT020 Part 2 - 25 mg/day Placebo Part 1 - 25 mg/day SBT020 Washout period Part 1 - Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 24 (4.17%)
    0 / 6 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 24 (4.17%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Part 1 - 5 mg/day SBT020 Part 1 - 15 mg/day SBT020 Part 2 - 25 mg/day SBT020 Part 2 - 25 mg/day Placebo Part 1 - 25 mg/day SBT020 Washout period Part 1 - Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    6 / 6 (100.00%)
    11 / 11 (100.00%)
    11 / 12 (91.67%)
    6 / 6 (100.00%)
    0 / 24 (0.00%)
    5 / 6 (83.33%)
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    2
    0
    2
    0
    0
    0
    Headache
         subjects affected / exposed
    2 / 6 (33.33%)
    1 / 6 (16.67%)
    3 / 11 (27.27%)
    5 / 12 (41.67%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    3 / 6 (50.00%)
         occurrences all number
    3
    1
    3
    10
    1
    0
    7
    Paraesthesia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    Somnolence
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    General disorders and administration site conditions
    Energy increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Feeling cold
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Influenza like illness
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    1
    Injection site erythema
         subjects affected / exposed
    6 / 6 (100.00%)
    6 / 6 (100.00%)
    11 / 11 (100.00%)
    7 / 12 (58.33%)
    6 / 6 (100.00%)
    0 / 24 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    23
    38
    258
    39
    37
    0
    2
    Injection site haematoma
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    2 / 11 (18.18%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    2
    0
    0
    0
    2
    Injection site pain
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    3 / 11 (27.27%)
    0 / 12 (0.00%)
    3 / 6 (50.00%)
    0 / 24 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    5
    3
    0
    12
    0
    1
    Injection site paraesthesia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    2
    0
    3
    0
    0
    Injection site pruritus
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 6 (33.33%)
    5 / 11 (45.45%)
    1 / 12 (8.33%)
    5 / 6 (83.33%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    6
    78
    1
    18
    0
    0
    Injection site swelling
         subjects affected / exposed
    3 / 6 (50.00%)
    4 / 6 (66.67%)
    9 / 11 (81.82%)
    2 / 12 (16.67%)
    5 / 6 (83.33%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    9
    11
    78
    2
    22
    0
    0
    Injection site warmth
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    Chest pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    Injection site irritation
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    0
    Malaise
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    0
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    Eye disorders
    Ocular hyperaemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    Toothache
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    0
    Frequent bowel movements
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Abdominal distension
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Psychiatric disorders
    Flat affect
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    Restlessness
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Muscle strain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    0
    Myalgia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    0
    Neck pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    Infections and infestations
    Infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    0
    Gastroenteritis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    0
    Lice infestation
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    3 / 11 (27.27%)
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    3
    2
    0
    0
    0
    Pharyngitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    0
    0
    Root canal infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 24 (0.00%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    23 May 2017
    Non-substantial change: the expiry period of the reconstituted product was prolonged from 48 hours to 7 days Substantial change: Expiry period of screening results (including the medical screening, 31P-MRS scan and UHDRS assessment) prolonged from 28 to 90 days Substantial change: Addition of possibility to perform an X-ray in patients in whom it was not clear if they were safe for MRI scanning Non-substantial change: The day 7 blood sample for plasma PK was to be taken on 1h15m post dose instead of 1h30m post dose Substantial change: The minimal τPCr for inclusion was lowered from 40 to 32.4 seconds
    02 Oct 2017
    • Substantial change: Patients with history of photosensitive epilepsy to be excluded • Non-substantial: Switch of NeuroCart and MRI assessments between day 27 and 28 in Part 2 • Non-substantial change: Addition of physical examination on day 27 • Non-substantial change: Eligibility to be checked via telephone prior to Part 2 • Non-substantial change: Removal of exploratory plasma and urine biomarker from Part 2 • Substantial change: Statement regarding the administered dose in Part 2 and safety statement of Part 1 • Substantial change: Addition of PK analysis of SBT-020-related component, SBT-127, in plasma and SBT-020-related components, SBT-127 and SBT-098, in urine • Non-substantial change: Change in PK sample storage temperature • Non-substantial change: Clarification that PK analysis was not to be performed on patients receiving placebo

    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

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/33197078
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