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    Clinical Trial Results:
    A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Luvadaxistat in Subjects With Cognitive Impairment Associated With Schizophrenia, Followed by Open-Label Treatment

    Summary
    EudraCT number
    		 2021-003834-34
    Trial protocol
    		 CZ   ES  
    Global end of trial date
    10 Oct 2024

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

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    NBI-1065844-CIAS2023
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05182476
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Neurocrine Biosciences, Inc.
    Sponsor organisation address
    6027 Edgewood Bend Ct, San Diego, CA, United States, 92130
    Public contact
    Medical Information Call Center, Neurocrine Biosciences, Inc., +34 932483137, medinfo@neurocrine.com
    Scientific contact
    Medical Information Call Center, Neurocrine Biosciences, Inc., +34 932483137, medinfo@neurocrine.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
    10 Oct 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    10 Oct 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the safety and efficacy of luvadaxistat compared with placebo on improving cognitive performance in participants with schizophrenia.
    Protection of trial subjects
    The Sponsor personnel and the investigators ensured that the study was conducted in full compliance with International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) guidelines, and with the laws and regulations of the country in which the study was conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Dec 2021
    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
    United States: 110
    Country: Number of subjects enrolled
    Bulgaria: 59
    Country: Number of subjects enrolled
    Czechia: 12
    Country: Number of subjects enrolled
    Serbia: 20
    Country: Number of subjects enrolled
    Spain: 2
    Worldwide total number of subjects
    203
    EEA total number of subjects
    73
    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)
    203
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Participants were randomized to receive luvadaxistat lower-dose, higher-dose, or placebo once daily (QD) during the Double-blind Treatment Period. Participants who completed the Double-blind Treatment Period and remained on treatment entered the Open-label Period and received luvadaxistat high-dose QD for 6–12 months.

    Pre-assignment
    Screening details
    		 A total of 203 participants were randomized, of which 202 participants received at least 1 dose of study drug.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Luvadaxistat Lower-dose
    Arm description
    		 Participants received luvadaxistat at a lower-dose as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Luvadaxistat
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Luvadaxistat was administered per schedule specified in the arm description.

    Arm title
    		 Luvadaxistat Higher-dose
    Arm description
    		 Participants received luvadaxistat at a higher-dose as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Luvadaxistat
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Luvadaxistat was administered per schedule specified in the arm description.

    Arm title
    		 Placebo
    Arm description
    		 Participants received placebo matched to luvadaxistat as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo was administered per schedule specified in the arm description.

    Number of subjects in period 1 [1]
    		Luvadaxistat Lower-dose Luvadaxistat Higher-dose Placebo
    Started
    49
    52
    101
    Received at Least 1 Dose of Study Drug
    49
    52
    101
    Completed
    44
    46
    88
    Not completed
    5
    6
    13
         Consent withdrawn by subject
    5
    3
    7
         Physician decision
    -
    1
    -
         Adverse event, non-fatal
    -
    1
    4
         Other than specified
    -
    1
    -
         Protocol-specified withdrawal criteria met
    -
    -
    1
         Lost to follow-up
    -
    -
    1
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: A total of 203 participants were randomized, of which 202 participants received at least 1 dose of study drug.
    Period 2
    Period 2 title
    Open-label Treatment Period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Luvadaxistat Lower to Higher-dose
    Arm description
    		 Participants who received luvadaxistat at a lower-dose during the Double-blind Treatment Period continued to receive luvadaxistat at a higher-dose as oral tablets QD for up to either 6 or 12 months.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Luvadaxistat
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Luvadaxistat was administered per schedule specified in the arm description.

    Arm title
    		 Luvadaxistat High to High-dose
    Arm description
    		 Participants who received luvadaxistat at a higher-dose during the Double-blind Treatment Period continued to receive luvadaxistat at a higher-dose as oral tablets QD for up to either 6 or 12 months.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Luvadaxistat
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Luvadaxistat was administered per schedule specified in the arm description.

    Arm title
    		 Placebo to Luvadaxistat Higher-dose
    Arm description
    		 Participants who received placebo matched to luvadaxistat during the Double-blind Treatment Period continued to receive luvadaxistat at a higher-dose as oral tablets QD for up to either 6 or 12 months.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo was administered per schedule specified in the arm description.

    Number of subjects in period 2
    		Luvadaxistat Lower to Higher-dose Luvadaxistat High to High-dose Placebo to Luvadaxistat Higher-dose
    Started
    44
    46
    88
    Received Luvadaxistat High-dose
    44
    46
    88
    Completed
    28
    26
    48
    Not completed
    16
    20
    40
         Consent withdrawn by subject
    2
    1
    6
         Adverse event, non-fatal
    -
    2
    5
         Study terminated by sponsor
    13
    16
    24
         Other than specified
    1
    -
    -
         Lost to follow-up
    -
    1
    4
         Pregnancy
    -
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Luvadaxistat Lower-dose
    Reporting group description
    		 Participants received luvadaxistat at a lower-dose as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.

    Reporting group title
    Luvadaxistat Higher-dose
    Reporting group description
    		 Participants received luvadaxistat at a higher-dose as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to luvadaxistat as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.

    Reporting group values
    		 Luvadaxistat Lower-dose Luvadaxistat Higher-dose Placebo Total
    Number of subjects
    		 49 52 101 202
    Age categorical
    Units: Subjects
        18 to <35 Years
    		 19 20 37 76
        ≥35 to 50 Years
    		 30 32 64 126
    Gender categorical
    Units: Subjects
        Female
    		 14 20 57 91
        Male
    		 35 32 44 111
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 1 1
        Asian
    		 1 1 2 4
        Black or African American
    		 15 9 24 48
        White
    		 32 39 69 140
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0
        Other
    		 1 2 5 8
        Multiple
    		 0 1 0 1
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 8 12 21 41
        Not Hispanic or Latino
    		 41 40 80 161

    End points

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    End points reporting groups
    Reporting group title
    Luvadaxistat Lower-dose
    Reporting group description
    		 Participants received luvadaxistat at a lower-dose as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.

    Reporting group title
    Luvadaxistat Higher-dose
    Reporting group description
    		 Participants received luvadaxistat at a higher-dose as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matched to luvadaxistat as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.
    Reporting group title
    Luvadaxistat Lower to Higher-dose
    Reporting group description
    		 Participants who received luvadaxistat at a lower-dose during the Double-blind Treatment Period continued to receive luvadaxistat at a higher-dose as oral tablets QD for up to either 6 or 12 months.

    Reporting group title
    Luvadaxistat High to High-dose
    Reporting group description
    		 Participants who received luvadaxistat at a higher-dose during the Double-blind Treatment Period continued to receive luvadaxistat at a higher-dose as oral tablets QD for up to either 6 or 12 months.

    Reporting group title
    Placebo to Luvadaxistat Higher-dose
    Reporting group description
    		 Participants who received placebo matched to luvadaxistat during the Double-blind Treatment Period continued to receive luvadaxistat at a higher-dose as oral tablets QD for up to either 6 or 12 months.

    Primary: Change from Baseline in the Brief Assessment of Cognition in Schizophrenia (BACS) Composite Score at Day 98

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    End point title
    		 Change from Baseline in the Brief Assessment of Cognition in Schizophrenia (BACS) Composite Score at Day 98
    End point description
    The BACS is a performance-based assessment that measures treatment-related changes in cognition and assesses 6 domains including verbal memory and learning, working memory, motor function, verbal fluency, attention and speed of processing, and executive function. For each domain, higher scores represent better cognition, and raw scores were converted to age and gender corrected normalized scores. The composite score was calculated as the mean of the normalized scores from the 6 domains. The BACS composite score was normalized based on z-scores transformed to T-scores (mean=50, SD=10 in normative data). There is no theoretical maximum score. Higher scores represent better outcomes. Efficacy Analysis Set: Included all randomized participants who demonstrated study treatment compliance and demonstrated symptoms stability between Visits 1, 2 and 3. Number of participants analysed = participants evaluable for this outcome measure.
    End point type
    Primary
    End point timeframe
    Baseline, Day 98
    End point values
    		 Luvadaxistat Lower-dose Luvadaxistat Higher-dose Placebo
    Number of subjects analysed
    38
    40
    83
    Units: score on a scale
        least squares mean (confidence interval 95%)
    1.9 (0.1 to 3.7)
    2.1 (0.3 to 3.8)
    2.6 (1.4 to 3.8)
    Statistical analysis title
    		 Luvadaxistat Lower-dose versus Placebo
    Comparison groups
    Placebo v Luvadaxistat Lower-dose
    Number of subjects included in analysis
    121
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5023
    Method
    		 ANCOVA
    Parameter type
    		 Least-Squares Mean Difference
    Point estimate
    -0.7
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.8
         upper limit
    		 1.4
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.1
    Statistical analysis title
    		 Luvadaxistat Higher-dose versus Placebo
    Comparison groups
    Placebo v Luvadaxistat Higher-dose
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.6123
    Method
    		 ANCOVA
    Parameter type
    		 Least-Squares Mean Difference
    Point estimate
    -0.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.7
         upper limit
    		 1.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.1

    Secondary: Change from Baseline in Schizophrenia Cognition Rating Scale (SCoRS) Interviewer Score at Day 98

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    End point title
    		 Change from Baseline in Schizophrenia Cognition Rating Scale (SCoRS) Interviewer Score at Day 98
    End point description
    The SCoRS includes 20 items focusing on cognitive impairment and the degree to which it affects day-to-day functioning, as well as a global functioning scale. Number of participants analysed = participants evaluable for this outcome measure. The scores ranged from 20 to 80, with lower scores indicating a better outcome. Efficacy Analysis Set: Included all randomized participants who demonstrated study treatment compliance and demonstrated symptoms stability between Visits 1, 2 and 3.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 98
    End point values
    		 Luvadaxistat Lower-dose Luvadaxistat Higher-dose Placebo
    Number of subjects analysed
    37
    40
    82
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -2.3 (-4.0 to -0.7)
    -2.7 (-4.3 to -1.2)
    -2.1 (-3.2 to -1.0)
    No statistical analyses for this end point

    Secondary: Change from Baseline in the Virtual Reality Functional Capacity Assessment Tool (VRFCAT) Score at Day 98

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    End point title
    		 Change from Baseline in the Virtual Reality Functional Capacity Assessment Tool (VRFCAT) Score at Day 98
    End point description
    The VRFCAT is an immersive, virtual-reality-based computerized assessment that evaluates functional capacity across the following 4 domains: transportation, finances, household management, and planning. Briefly, the VRFCAT consists of 4 mini scenarios, which include checking the kitchen for the availability of items to complete a recipe and planning a trip to the grocery store, taking a bus and paying the correct fare, shopping for the items in a store, and returning home. VRFCAT T-scores were calculated from standardized task performance (mean=50, SD=10). There was no theoretical maximum score. Higher scores indicated faster completion, fewer errors, and fewer forced progressions, reflecting better functional capacity. Efficacy Analysis Set: Included all randomized participants who demonstrated study treatment compliance and demonstrated symptoms stability between Visits 1, 2 and 3. Number of participants analysed = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 98
    End point values
    		 Luvadaxistat Lower-dose Luvadaxistat Higher-dose Placebo
    Number of subjects analysed
    37
    40
    83
    Units: T-score
    least squares mean (confidence interval 95%)
        VRFCAT-Adjusted Total Time T Score
    1.633 (-2.409 to 5.674)
    3.983 (0.075 to 7.892)
    1.844 (-0.859 to 4.548)
        VRFCAT-Total Error Count T Score
    -1.194 (-6.248 to 3.860)
    3.954 (-0.977 to 8.885)
    0.038 (-3.362 to 3.437)
        VRFCAT-Total Forced Progressions T Score
    -4.533 (-12.136 to 3.070)
    3.939 (-3.453 to 11.330)
    -0.891 (-6.015 to 4.232)
    No statistical analyses for this end point

    Secondary: Change from Baseline on the Clinical Global Impression – Severity (CGI-S) Score at Day 98

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    End point title
    		 Change from Baseline on the Clinical Global Impression – Severity (CGI-S) Score at Day 98
    End point description
    The CGI-S scale was based on a 7-point scale (range: 1=normal to 7=among the most extremely ill patients), was used to rate the overall global severity of schizophrenia. Efficacy Analysis Set: Included all randomized participants who demonstrated study treatment compliance and demonstrated symptoms stability between Visits 1, 2 and 3. n=number of participants analyzed at specified time points. Number of participants analysed = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 98
    End point values
    		 Luvadaxistat Lower-dose Luvadaxistat Higher-dose Placebo
    Number of subjects analysed
    41 [1]
    43 [2]
    88 [3]
    Units: participants
        Baseline: 1=Normal, not at all ill
    0
    0
    1
        Baseline: 2=Borderline ill
    3
    3
    6
        Baseline: 3=Mildly ill
    11
    17
    38
        Baseline: 4=Moderately ill
    24
    20
    39
        Baseline: 5=Markedly ill
    3
    3
    3
        Baseline: 6=Severely ill
    0
    0
    1
        Baseline: 7=Among the most extremely ill patients
    0
    0
    0
        Day 98: 1=Normal, not at all ill
    0
    0
    0
        Day 98: 2=Borderline ill
    2
    5
    15
        Day 98: 3=Mildly ill
    18
    20
    35
        Day 98: 4=Moderately ill
    15
    14
    32
        Day 98: 5=Markedly ill
    2
    0
    2
        Day 98: 6=Severely ill
    0
    0
    0
        Day 98: 7=Among the most extremely ill patients
    0
    0
    0
    Notes
    [1] - Baseline: n=41 Day 98: n=37
    [2] - Baseline: n=43 Day 98: n=39
    [3] - Baseline: n=88 Day 98: n=84
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to Week 68
    Adverse event reporting additional description
    Safety Analysis Set: Included all randomized participants who received at least 1 dose of study treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Double-blind Treatment Period: Luvadaxistat Lower-dose
    Reporting group description
    		 Participants received luvadaxistat at a lower-dose as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.

    Reporting group title
    Double-blind Treatment Period: Luvadaxistat Higher-dose
    Reporting group description
    		 Participants received luvadaxistat at a higher-dose as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.

    Reporting group title
    Double-blind Treatment Period: Placebo
    Reporting group description
    		 Participants received placebo matched to luvadaxistat as oral tablets QD for up to 14 weeks during the Double-blind Treatment Period.

    Reporting group title
    Open-label Treatment Period: Luvadaxistat Higher-dose
    Reporting group description
    		 All participants who entered the Open-label Treatment Period continued to receive luvadaxistat at a higher-dose as oral tablets QD for up to either 6 or 12 months.

    Serious adverse events
    		 Double-blind Treatment Period: Luvadaxistat Lower-dose Double-blind Treatment Period: Luvadaxistat Higher-dose Double-blind Treatment Period: Placebo Open-label Treatment Period: Luvadaxistat Higher-dose
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 49 (2.04%)
    1 / 52 (1.92%)
    3 / 101 (2.97%)
    4 / 178 (2.25%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Intentional overdose
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 52 (0.00%)
    0 / 101 (0.00%)
    1 / 178 (0.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Seizure
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 52 (0.00%)
    1 / 101 (0.99%)
    0 / 178 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 52 (0.00%)
    0 / 101 (0.00%)
    1 / 178 (0.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 52 (1.92%)
    0 / 101 (0.00%)
    0 / 178 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Schizophrenia
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 52 (0.00%)
    0 / 101 (0.00%)
    1 / 178 (0.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Suicide attempt
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 52 (0.00%)
    0 / 101 (0.00%)
    1 / 178 (0.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 52 (0.00%)
    0 / 101 (0.00%)
    0 / 178 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 52 (0.00%)
    0 / 101 (0.00%)
    1 / 178 (0.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    0 / 49 (0.00%)
    0 / 52 (0.00%)
    1 / 101 (0.99%)
    0 / 178 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Double-blind Treatment Period: Luvadaxistat Lower-dose Double-blind Treatment Period: Luvadaxistat Higher-dose Double-blind Treatment Period: Placebo Open-label Treatment Period: Luvadaxistat Higher-dose
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 49 (0.00%)
    3 / 52 (5.77%)
    1 / 101 (0.99%)
    5 / 178 (2.81%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 49 (0.00%)
    3 / 52 (5.77%)
    1 / 101 (0.99%)
    5 / 178 (2.81%)
         occurrences all number
    0
    3
    1
    5

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Sep 2021
    Global Amendment 1.0
    12 Sep 2022
    Global Amendment 2.0
    20 Mar 2023
    Global Amendment 3.0

    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.
    This study was terminated early as the study failed to meet the primary endpoint.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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