Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44356   clinical trials with a EudraCT protocol, of which   7382   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    Long-Term, Open-Label Extension Study to Evaluate the Safety and Tolerability of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep

    Summary
    EudraCT number
    		 2021-006788-11
    Trial protocol
    		 DK   ES   DE  
    Global end of trial date
    27 Jan 2025

    Results information
    Results version number
    		 v1(current)
    This version publication date
    14 Aug 2025
    First version publication date
    14 Aug 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    NBI-827104-CSWS2025
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05301894
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Neurocrine Biosciences, Inc.
    Sponsor organisation address
    6027 Edgewood Bend Court , San Diego, United States, 92130
    Public contact
    Neurocrine Medical Information Call Center, Neurocrine Biosciences, Inc., 1 8776413461, medinfo@neurocrine.com
    Scientific contact
    Neurocrine Medical Information Call Center, Neurocrine Biosciences, Inc. , 1 8776413461, 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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    27 Jan 2025
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    27 Jan 2025
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Jan 2025
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective for this study is to evaluate the long-term safety and tolerability of NBI-827104 in pediatric participants with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS).
    Protection of trial subjects
    The study was conducted in accordance with Good Clinical Practice (GCP), International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) GCP guidelines, and the laws and regulations of the countries in which the study was conducted
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Jun 2022
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 11
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    Denmark: 3
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    Switzerland: 1
    Worldwide total number of subjects
    19
    EEA total number of subjects
    6
    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)
    18
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study was terminated early by the Sponsor; therefore, no participants completed the study.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 NBI-827104
    Arm description
    		 NBI-827104 administered orally
    Arm type
    		 Experimental

    Investigational medicinal product name
    NBI-827104
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    NBI-827104 was administered per dose and schedule specified in the arm description.

    Number of subjects in period 1
    		NBI-827104
    Started
    19
    Received at Least 1 Dose of Study Drug
    19
    Completed
    0
    Not completed
    19
         Consent withdrawn by subject
    2
         Study Terminated by Sponsor
    15
         Lack of efficacy
    2

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    NBI-827104
    Reporting group description
    		 NBI-827104 administered orally

    Reporting group values
    		 NBI-827104 Total
    Number of subjects
    		 19 19
    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)
    		 18 18
        Adolescents (12-17 years)
    		 1 1
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 9.04 ( 1.78 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 9 9
        Male
    		 10 10
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 2 2
        Not Hispanic or Latino
    		 17 17
        Unknown or Not Reported
    		 0 0
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 18 18
        Other
    		 1 1

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    NBI-827104
    Reporting group description
    		 NBI-827104 administered orally

    Primary: The Number of Participants with Serious Treatment-emergent Adverse Events (TEAEs)

    		 Close Top of page
    End point title
    		 The Number of Participants with Serious Treatment-emergent Adverse Events (TEAEs) [1]
    End point description
    A TEAE is an adverse event (AE) that was not present prior to the initiation of study treatment or was an already present condition that worsened either in intensity or frequency following the initiation of study treatment. Safety Analysis Set: All enrolled participants who took at least 1 dose of NBI-827104.
    End point type
    Primary
    End point timeframe
    Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
    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: As per protocol, the endpoint is descriptive in nature.
    End point values
    		 NBI-827104
    Number of subjects analysed
    19
    Units: participants
    2
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Up to 884 days of treatment and 4-week safety follow-up (mean duration of exposure was 649.7 days)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    NBI-827104
    Reporting group description
    		 NBI-827104 administered orally

    Serious adverse events
    		 NBI-827104
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 19 (10.53%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Nervous system disorders
    Seizure cluster
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Seizure
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Osteomyelitis acute
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 NBI-827104
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    17 / 19 (89.47%)
    General disorders and administration site conditions
    Thirst
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Pyrexia
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    5
    Influenza like illness
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Gait disturbance
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Fatigue
         subjects affected / exposed
    4 / 19 (21.05%)
         occurrences all number
    6
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Oropharyngeal pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Throat irritation
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Psychiatric disorders
    Aggression
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Anxiety
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    3
    Change in sustained attention
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Emotional disorder
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Insomnia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Irritability
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Mood altered
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Nervousness
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Perseveration
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Regressive behaviour
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Tic
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Product issues
    Device dislocation
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Investigations
    Neutrophil count decreased
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    3
    Foot fracture
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Head injury
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Limb injury
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Skin abrasion
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Skin laceration
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Wrist fracture
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Dyspraxia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Fine motor skill dysfunction
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Headache
         subjects affected / exposed
    4 / 19 (21.05%)
         occurrences all number
    7
    Lethargy
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Memory impairment
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Seizure
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Somnolence
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    3
    Tremor
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    3
    Eye disorders
    Ocular hyperaemia
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Abdominal pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    6 / 19 (31.58%)
         occurrences all number
    8
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    4 / 19 (21.05%)
         occurrences all number
    5
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Dermatitis contact
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Erythema
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Neck pain
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Pain in extremity
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Infections and infestations
    Conjunctivitis bacterial
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 19 (21.05%)
         occurrences all number
    11
    Ear infection
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Eye infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Helicobacter gastritis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Lymphangitis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    6
    Parainfluenzae virus infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Pharyngitis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    2
    Respiratory syncytial virus infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Streptococcal infection
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Tonsillitis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Conjunctivitis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Coronavirus infection
         subjects affected / exposed
    2 / 19 (10.53%)
         occurrences all number
    2
    Viral myositis
         subjects affected / exposed
    1 / 19 (5.26%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    3 / 19 (15.79%)
         occurrences all number
    3

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    29 Nov 2021
    - Study assessments revised - Procedures related to COVID-19 were added
    30 Mar 2023
    - Increased doses of NBI-827104 - The maximum duration of the study was revised. - An interim analysis was added. - PK blood sample collection was revised. - Primary endpoint was revised. - Secondary objectives were recategorized. - Enrollment criteria were revised.

    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.
    The study was terminated early by the Sponsor; therefore, no participants completed the study.
    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.

    European Medicines Agency © 1995-Tue Aug 26 17:58:10 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA