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    Clinical Trial Results:
    A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

    Summary
    EudraCT number
    2020-004403-14
    Trial protocol
    ES   LT   CZ   IT  
    Global end of trial date
    18 Sep 2023

    Results information
    Results version number
    v1(current)
    This version publication date
    29 Sep 2024
    First version publication date
    29 Sep 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    BCX9930-203
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT05116787
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    BioCryst Pharmaceuticals Inc
    Sponsor organisation address
    4505 Emperor Boulevard Nottingham Hall, Suite 200, Durham, North Carolina , United States, 27703
    Public contact
    Study Director, BioCryst Pharmaceuticals Inc, +001 919859 1302, clinicaltrials@biocryst.com
    Scientific contact
    Study Director, BioCryst Pharmaceuticals Inc, +001 919859 1302, clinicaltrials@biocryst.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
    18 Sep 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Sep 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of oral BCX9930 monotherapy administered for 12 weeks, as compared to placebo, in participants with paroxysmal nocturnal hemoglobinuria (PNH).
    Protection of trial subjects
    This trial was conducted in compliance with International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) guidelines for conducting, recording, and reporting trials, and in accordance with the Declaration of Helsinki.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    26 Oct 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
    Malaysia: 3
    Country: Number of subjects enrolled
    South Africa: 8
    Country: Number of subjects enrolled
    Korea, Republic of: 1
    Worldwide total number of subjects
    12
    EEA total number of subjects
    0
    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)
    12
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study was conducted in Malaysia, South Africa, and South Korea.

    Pre-assignment
    Screening details
    A total 12 participants were randomized and treated.

    Period 1
    Period 1 title
    Overall (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator

    Arms
    Are arms mutually exclusive
    No

    Arm title
    BCX9930
    Arm description
    Participants received BCX9930 monotherapy in double blind manner for 12 weeks, then in an open label manner for the remainder of the study. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open label BCX9930 prior to Week 12.
    Arm type
    Experimental

    Investigational medicinal product name
    BCX9930
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Administered orally twice daily.

    Arm title
    Placebo
    Arm description
    Participants received BCX9930 matching placebo in double blind manner for 12 weeks. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy prior to Week 12, if earlier. The maximum treatment duration on placebo was 84 days.
    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
    Administered orally twice daily.

    Arm title
    BCX9930 After Placebo
    Arm description
    Participants who were initially randomized to placebo group received BCX9930 monotherapy in open label manner, if they had completed Week 12 on placebo, or earlier after the sponsor decided to halt enrolment in the study permanently and terminate the study.
    Arm type
    Experimental

    Investigational medicinal product name
    BCX9930
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Administered orally twice daily.

    Number of subjects in period 1
    BCX9930 Placebo BCX9930 After Placebo
    Started
    10
    2
    1
    Completed
    4
    1
    1
    Not completed
    6
    1
    0
         Consent withdrawn by subject
    -
    1
    -
         Adverse event, non-fatal
    1
    -
    -
         Pregnancy
    1
    -
    -
         Miscellaneous
    4
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    BCX9930
    Reporting group description
    Participants received BCX9930 monotherapy in double blind manner for 12 weeks, then in an open label manner for the remainder of the study. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open label BCX9930 prior to Week 12.

    Reporting group title
    Placebo
    Reporting group description
    Participants received BCX9930 matching placebo in double blind manner for 12 weeks. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy prior to Week 12, if earlier. The maximum treatment duration on placebo was 84 days.

    Reporting group title
    BCX9930 After Placebo
    Reporting group description
    Participants who were initially randomized to placebo group received BCX9930 monotherapy in open label manner, if they had completed Week 12 on placebo, or earlier after the sponsor decided to halt enrolment in the study permanently and terminate the study.

    Reporting group values
    BCX9930 Placebo BCX9930 After Placebo Total
    Number of subjects
    10 2 1 12
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    10 2 1 12
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    37.8 ( 12.70 ) 37.0 ( 9.90 ) 30.0 ( 0 ) -
    Gender categorical
    Units: Subjects
        Female
    6 2 1 8
        Male
    4 0 0 4
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    0 0 0 0
        Not Hispanic or Latino
    10 2 1 12
    Race
    Units: Subjects
        Asian
    3 2 1 5
        Black or African American
    7 0 0 7

    End points

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    End points reporting groups
    Reporting group title
    BCX9930
    Reporting group description
    Participants received BCX9930 monotherapy in double blind manner for 12 weeks, then in an open label manner for the remainder of the study. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open label BCX9930 prior to Week 12.

    Reporting group title
    Placebo
    Reporting group description
    Participants received BCX9930 matching placebo in double blind manner for 12 weeks. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy prior to Week 12, if earlier. The maximum treatment duration on placebo was 84 days.

    Reporting group title
    BCX9930 After Placebo
    Reporting group description
    Participants who were initially randomized to placebo group received BCX9930 monotherapy in open label manner, if they had completed Week 12 on placebo, or earlier after the sponsor decided to halt enrolment in the study permanently and terminate the study.

    Primary: Change From Baseline in Hemoglobin at Week 12

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    End point title
    Change From Baseline in Hemoglobin at Week 12 [1] [2]
    End point description
    Participants in the All Subjects as Treated (ASaT) population (all participants who received at least 1 dose of study drug and had a post baseline laboratory assessment) were analyzed.
    End point type
    Primary
    End point timeframe
    Baseline, Week 12
    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: There was no formal hypothesis testing and only descriptive analyses was performed.
    [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: No participant who switched from placebo to BCX9930 had data available at Week 12. Therefore, BCX9930 after placebo group was not applicable for this endpoint.
    End point values
    BCX9930 Placebo
    Number of subjects analysed
    10
    2
    Units: grams per deciliter (g/dL)
    arithmetic mean (standard deviation)
        Baseline
    8.49 ( 1.556 )
    9.48 ( 1.721 )
        Change at Week 12
    2.23 ( 2.146 )
    -1.23 ( 0.330 )
    No statistical analyses for this end point

    Secondary: Number of Participants Who Were Transfusion-free

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    End point title
    Number of Participants Who Were Transfusion-free
    End point description
    The number of participants who did not receive any transfusions (packed red blood cells [pRBCs] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 12, or (2) did not receive a transfusion during the period of interest despite recording a haemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free. Participants in the ASaT population were analyzed.
    End point type
    Secondary
    End point timeframe
    From Week 4 to Week 12
    End point values
    BCX9930 Placebo BCX9930 After Placebo
    Number of subjects analysed
    10
    2
    1
    Units: participants
    8
    1
    1
    No statistical analyses for this end point

    Secondary: Number of Units of pRBCs Transfused

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    End point title
    Number of Units of pRBCs Transfused
    End point description
    Participants in the ASaT population were analyzed.
    End point type
    Secondary
    End point timeframe
    From Week 4 to Week 12
    End point values
    BCX9930 Placebo BCX9930 After Placebo
    Number of subjects analysed
    10
    2
    1
    Units: units of pRBCs
    1
    0
    0
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Lactate Dehydrogenase

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    End point title
    Percent Change From Baseline in Lactate Dehydrogenase [3]
    End point description
    Participants in the ASaT population were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12
    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: No participant who switched from placebo to BCX9930 had data available at Week 12. Therefore, BCX9930 after placebo group was not applicable for this endpoint.
    End point values
    BCX9930 Placebo
    Number of subjects analysed
    10
    2
    Units: percent change
    arithmetic mean (standard deviation)
        Baseline
    1705.4 ( 715.96 )
    1613.5 ( 103.94 )
        Change at Week 12
    -69.8 ( 26.00 )
    9.1 ( 45.47 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

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    End point title
    Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score [4]
    End point description
    The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 ("not at all") to 4 ("very much"), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life. Participants in the ASaT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12
    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: No participant who switched from placebo to BCX9930 had data available at Week 12. Therefore, BCX9930 after placebo group was not applicable for this endpoint.
    End point values
    BCX9930 Placebo
    Number of subjects analysed
    9
    2
    Units: scores on a scale
    arithmetic mean (standard deviation)
        Baseline (n=9, n=2)
    34.3 ( 13.47 )
    13.0 ( 8.49 )
        Change at Week 12 (n=8, n=2)
    -2.3 ( 13.48 )
    -2.0 ( 0.00 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Day 1 up to 408 days
    Adverse event reporting additional description
    The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    24
    Reporting groups
    Reporting group title
    BCX9930
    Reporting group description
    Participants received BCX9930 monotherapy in double blind manner for 12 weeks, then in an open label manner for the remainder of the study. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open label BCX9930 prior to Week 12.

    Reporting group title
    Placebo
    Reporting group description
    Participants received BCX9930 matching placebo in double blind manner for, 12 weeks. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy prior to Week 12, if earlier. The maximum treatment duration on placebo was 84 days.

    Reporting group title
    BCX9930 After Placebo
    Reporting group description
    Participants who were initially randomized to placebo group received BCX9930 monotherapy in open label manner, if they had completed Week 12 on placebo, or earlier after the sponsor decided to halt enrolment in the study permanently and terminate the study.

    Serious adverse events
    BCX9930 Placebo BCX9930 After Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 10 (50.00%)
    1 / 2 (50.00%)
    1 / 1 (100.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Blood and lymphatic system disorders
    Breakthrough haemolysis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Budd-Chiari syndrome
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Renal failure
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Intervertebral disc protrusion
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Enterovirus infection
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia fungal
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Tuberculosis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    BCX9930 Placebo BCX9930 After Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 10 (100.00%)
    2 / 2 (100.00%)
    1 / 1 (100.00%)
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Fatigue
         subjects affected / exposed
    3 / 10 (30.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
         occurrences all number
    4
    1
    0
    Influenza like illness
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Malaise
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Peripheral swelling
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
         occurrences all number
    0
    1
    0
    Swelling face
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Vaccination site pruritus
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Reproductive system and breast disorders
    Breast pain
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Dysmenorrhoea
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Heavy menstrual bleeding
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Menometrorrhagia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Epistaxis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Rhinitis allergic
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Depression
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Insomnia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    1
    0
    1
    Investigations
    Blood uric acid increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Serum ferritin decreased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Weight increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Injury, poisoning and procedural complications
    Subcutaneous haematoma
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    1
    0
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Headache
         subjects affected / exposed
    4 / 10 (40.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    6
    0
    1
    Blood and lymphatic system disorders
    Breakthrough haemolysis
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Haemolysis
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Neutropenia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    2
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    3 / 10 (30.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    4
    0
    0
    Abdominal tenderness
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Constipation
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    1
    0
    Diarrhoea
         subjects affected / exposed
    3 / 10 (30.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    3
    0
    2
    Dyspepsia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Dysphagia
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Gastric varices
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Nausea
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Varices oesophageal
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 2 (50.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    1
    0
    Vomiting
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Hepatobiliary disorders
    Jaundice
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Hyperhidrosis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Pruritus
         subjects affected / exposed
    6 / 10 (60.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    8
    0
    0
    Rash
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Rash maculo-papular
         subjects affected / exposed
    3 / 10 (30.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    5
    0
    0
    Rash papular
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Skin ulcer
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Haematuria
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Haemoglobinuria
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Nephropathy
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 10 (30.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    4
    0
    1
    Arthritis reactive
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Flank pain
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Joint stiffness
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal stiffness
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Periarthritis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    0
    Conjunctivitis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    0
    1
    Influenza
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Pneumonia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Pulmonary tuberculosis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Tonsillitis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 10 (40.00%)
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    5
    0
    1
    Urinary tract infection
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 May 2021
    - The primary efficacy endpoint for Part 1 was updated from percent change in lactate dehydrogenase at Week 12 to change from baseline in Hb at Week 12. - The secondary efficacy endpoints for Part 1 were revised to emphasize key secondary endpoints that will demonstrate clinical benefit in support of the new primary endpoint. - The sample size calculation and power statement were updated to reflect the change in the primary endpoint. - The remaining endpoints were categorized as other secondary efficacy endpoints, other health-related quality of life endpoints, or exploratory endpoints. - The secondary endpoints for Part 2 were revised to reflect the changes made to the Part 1 endpoints. - New secondary endpoints were described for both parts and others were removed. - The statistical methods described were revised extensively to reflect the changes made to the endpoints. - The randomization stratum based on receipt of any blood transfusion in the 6 months prior to baseline was redefined to refer to receipt of pRBC transfusion. - The benefit-risk was updated in accordance with the currently available clinical and nonclinical data. - Section (Prohibited and Restricted Medications) was extensively revised. Prohibited medications were redefined.
    29 Jun 2022
    - The recommended dose of BCX9930 was reduced from 500 mg BID to 400 mg BID for all participants. - For newly randomized participants, treatment with BCX9930 was to begin at 200 mg BID for the first 14 days before escalation to 400 mg BID. - Additional safety assessments were added for all participants through the first 12 weeks of BCX9930 dosing. - Guidance was provided for the management of participants with treatment-emergent increases in serum creatinine. - Inclusion Criterion 5(f) was modified. - Recommendations were provided for dose tapering following discontinuation of BCX9930.
    01 Aug 2022
    - A study stopping rule was added.

    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 sponsor decided to prematurely terminate the study due to changes in the competitive landscape. As the study was terminated early and data was limited, per planned analysis only key efficacy endpoints and safety were analyzed and reported.
    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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