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    Clinical Trial Results:
    A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)

    Summary
    EudraCT number
    		 2020-000501-93
    Trial protocol
    		 GB   AT   DK   IT  
    Global end of trial date
    04 Oct 2023

    Results information
    Results version number
    		 v2(current)
    This version publication date
    29 Mar 2025
    First version publication date
    19 Oct 2024
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    Post NIH comments, we need to align the EudraCT draft with CT.gov.

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    BCX9930-201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04702568
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    BioCryst Pharmaceuticals Inc.
    Sponsor organisation address
    4505 Emperor Blvd., Suite 200, Durham, United States, NC 27703
    Public contact
    BioCryst Pharmaceuticals Inc, Study Director, +001 919859 1302, clinicaltrials@biocryst.com
    Scientific contact
    BioCryst Pharmaceuticals Inc, Study Director, +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
    04 Oct 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Oct 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess long-term safety and tolerability data in eligible participants with paroxysmal nocturnal hemoglobinuria (PNH) who previously received BCX9930 in a BioCryst-sponsored study and derived benefit from BCX9930 treatment.
    Protection of trial subjects
    This trial was conducted in compliance with International Conference on Harmonization (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
    18 Dec 2020
    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 Kingdom: 5
    Country: Number of subjects enrolled
    Austria: 2
    Country: Number of subjects enrolled
    South Africa: 12
    Worldwide total number of subjects
    19
    EEA total number of subjects
    2
    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)
    18
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants who had participated in previous BCX9930 trials (BCX9930-101 [NCT04330534], BCX9930-202 [NCT05116774], or BCX9930-203 [NCT05116787]) for Paroxysmal Nocturnal Hemoglobinuria (PNH) and showed a benefit of treatment as determined by the investigator were enrolled in this long-term safety trial.

    Pre-assignment
    Screening details
    		 A total of 19 participants were treated.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group
    Arm description
    		 This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 milligrams (mg) orally twice daily (BID). Treatment duration was up to 144 weeks.
    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
    		 C5 INH Inadequate Response Group
    Arm description
    		 This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.
    Arm type
    		 Experimental

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

    Number of subjects in period 1
    		Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Started
    12
    7
    Completed
    0
    0
    Not completed
    12
    7
         Pregnancy
    1
    -
         Transitioned to the BCX9930-205 roll-over study
    10
    4
         Withdrawal by Subject
    -
    2
         Withdrawn due to Investigator decision
    -
    1
         Bone marrow transplant
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group
    Reporting group description
    		 This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 milligrams (mg) orally twice daily (BID). Treatment duration was up to 144 weeks.

    Reporting group title
    C5 INH Inadequate Response Group
    Reporting group description
    		 This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.

    Reporting group values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group Total
    Number of subjects
    		 12 7 19
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 12 6 18
        From 65-84 years
    		 0 1 1
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 29.3 ( 7.82 ) 44.6 ( 18.59 ) -
    Gender categorical
    Units: Subjects
        Female
    		 3 5 8
        Male
    		 9 2 11
    Ethinicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 12 7 19
        Unknown or Not Reported
    		 0 0 0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 1 1 2
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 7 2 9
        White
    		 3 4 7
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 1 0 1

    End points

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    End points reporting groups
    Reporting group title
    Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group
    Reporting group description
    		 This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 milligrams (mg) orally twice daily (BID). Treatment duration was up to 144 weeks.

    Reporting group title
    C5 INH Inadequate Response Group
    Reporting group description
    		 This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.

    Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Participants With Treatment Emergent Adverse Events (TEAEs) [1]
    End point description
    An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment emergent if its start date and time was on or after the date and time of first on-study dose of study drug. The safety population included all participants who received at least 1 capsule or tablet of study drug.
    End point type
    Primary
    End point timeframe
    From first dose of study drug up to 3 weeks after last dose (Week 147)
    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.
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
        number (not applicable)
    12
    7
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical PNH Symptom Based on Severity: Fatigue

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    End point title
    		 Number of Participants with Clinical PNH Symptom Based on Severity: Fatigue
    End point description
    Data was reported for number of participants with clinical PNH symptom of fatigue. The severity of clinical PNH symptom of fatigue was graded as none, mild, moderate and severe based solely on investigator’s discretion. mITT population included all participants who received at least 1 capsule or tablet of study drug and had post baseline assessment of PNH symptoms and/or laboratory data. Participants with available data at each visit were included.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=12,7)
    8
    6
        Baseline-mild (n=12,7)
    3
    2
        Baseline-moderate (n=12,7)
    0
    1
        Baseline-severe (n=12,7)
    1
    0
        Week 24-none (n=12,7)
    10
    3
        Week 24-mild (n=12,7)
    2
    3
        Week 24-moderate (n=12,7)
    0
    1
        Week 24-severe (n=12,7)
    0
    0
        Week 48-none (n=8,3)
    6
    2
        Week 48-mild (n=8,3)
    2
    0
        Week 48-moderate (n=8,3)
    0
    1
        Week 48- severe (n=8,3)
    0
    0
        Week 72-none (n=8,3)
    7
    1
        Week 72-mild (n=8,3)
    1
    2
        Week 72-moderate (n=8,3)
    0
    0
        Week 72-severe (n=8,3)
    0
    0
        Week 96-none (n=8,3)
    6
    2
        Week 96-mild (n=8,3)
    2
    0
        Week 96-moderate (n=8,3)
    0
    0
        Week 96-severe (n=8,3)
    0
    1
        Week 120-none (n=8,3)
    8
    2
        Week 120-mild (n=8,3)
    0
    0
        Week 120-moderate (n=8,3)
    0
    1
        Week 120-severe (n=8,3)
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical PNH Symptom Based on Severity: Dyspnea

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    End point title
    		 Number of Participants with Clinical PNH Symptom Based on Severity: Dyspnea
    End point description
    Data was reported for number of participants with clinical PNH symptom of dyspnea. The severity of clinical PNH symptom of dyspnea was graded as none, mild, moderate and severe based solely on investigator’s discretion. Participants in the mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=12,7)
    11
    7
        Baseline-mild (n=12,7)
    1
    0
        Baseline-moderate (n=12,7)
    0
    0
        Baseline-severe (n=12,7)
    0
    0
        Week 24-none (n=12,7)
    11
    7
        Week 24-mild (n=12,7)
    1
    0
        Week 24-moderate (n=12,7)
    0
    0
        Week 24-severe (n=12,7)
    0
    0
        Week 48-none (n=8,3)
    6
    2
        Week 48-mild (n=8,3)
    2
    1
        Week 48-moderate (n=8,3)
    0
    0
        Week 48- severe (n=8,3)
    0
    0
        Week 72-none (n=8,3)
    7
    2
        Week 72-mild (n=8,3)
    1
    1
        Week 72-moderate (n=8,3)
    0
    0
        Week 72-severe (n=8,3)
    0
    0
        Week 96-none (n=8,3)
    7
    2
        Week 96-mild (n=8,3)
    0
    0
        Week 96-moderate (n=8,3)
    1
    1
        Week 96-severe (n=8,3)
    0
    0
        Week 120-none (n=8,3)
    8
    2
        Week 120-mild (n=8,3)
    0
    1
        Week 120-moderate (n=8,3)
    0
    0
        Week 120-severe (n=8,3)
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort

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    End point title
    		 Number of Participants with Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort
    End point description
    Data was reported for number of participants with clinical PNH symptom of chest pain/discomfort. The severity of clinical PNH symptom of chest pain/discomfort was graded as none, mild, moderate and severe based solely on investigator’s discretion. Participants in the mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 12
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=12,7)
    12
    7
        Baseline-mild (n=12,7)
    0
    0
        Baseline-moderate (n=12,7)
    0
    0
        Baseline-severe (n=12,7)
    0
    0
        Week 24-none (n=12,7)
    12
    7
        Week 24-mild (n=12,7)
    0
    0
        Week 24-moderate (n=12,7)
    0
    0
        Week 24-severe (n=12,7)
    0
    0
        Week 48-none (n=8,3)
    7
    2
        Week 48-mild (n=8,3)
    1
    1
        Week 48-moderate (n=8,3)
    0
    0
        Week 48- severe (n=8,3)
    0
    0
        Week 72-none (n=8,3)
    7
    2
        Week 72-mild (n=8,3)
    1
    0
        Week 72-moderate (n=8,3)
    0
    1
        Week 72-severe (n=8,3)
    0
    0
        Week 96-none (n=8,3)
    7
    3
        Week 96-mild (n=8,3)
    1
    0
        Week 96-moderate (n=8,3)
    0
    0
        Week 96-severe (n=8,3)
    0
    0
        Week 120-none (n=8,3)
    8
    3
        Week 120-mild (n=8,3)
    0
    0
        Week 120-moderate (n=8,3)
    0
    0
        Week 120-severe (n=8,3)
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical PNH Symptom Based on Severity: Difficulty Swallowing

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    End point title
    		 Number of Participants with Clinical PNH Symptom Based on Severity: Difficulty Swallowing
    End point description
    Data was reported for number of participants with clinical PNH symptom of difficulty swallowing. The severity of clinical PNH symptom of difficulty swallowing was graded as none, mild, moderate and severe based solely on investigator’s discretion. Participants in the mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=12,7)
    11
    6
        Baseline-mild (n=12,7)
    1
    1
        Baseline-moderate (n=12,7)
    0
    0
        Baseline-severe (n=12,7)
    0
    0
        Week 24-none (n=12,7)
    12
    7
        Week 24-mild (n=12,7)
    0
    0
        Week 24-moderate (n=12,7)
    0
    0
        Week 24-severe (n=12,7)
    0
    0
        Week 48-none (n=8,3)
    6
    2
        Week 48-mild (n=8,3)
    2
    1
        Week 48-moderate (n=8,3)
    0
    0
        Week 48- severe (n=8,3)
    0
    0
        Week 72-none (n=8,3)
    7
    2
        Week 72-mild (n=8,3)
    1
    1
        Week 72-moderate (n=8,3)
    0
    0
        Week 72-severe (n=8,3)
    0
    0
        Week 96-none (n=8,3)
    7
    2
        Week 96-mild (n=8,3)
    1
    1
        Week 96-moderate (n=8,3)
    0
    0
        Week 96-severe (n=8,3)
    0
    0
        Week 120-none (n=8,3)
    8
    3
        Week 120-mild (n=8,3)
    0
    0
        Week 120-moderate (n=8,3)
    0
    0
        Week 120-severe (n=8,3)
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical PNH Symptom Based on Severity: Abdominal Pain

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    End point title
    		 Number of Participants with Clinical PNH Symptom Based on Severity: Abdominal Pain
    End point description
    Data was reported for number of participants with clinical PNH symptom of abdominal pain. The severity of clinical PNH symptom of abdominal pain was graded as none, mild, moderate and severe based solely on investigator’s discretion. Participants in the mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=12,7)
    10
    7
        Baseline-mild (n=12,7)
    1
    0
        Baseline-moderate (n=12,7)
    1
    0
        Baseline-severe (n=12,7)
    0
    0
        Week 24-none (n=12,7)
    9
    6
        Week 24-mild (n=12,7)
    2
    0
        Week 24-moderate (n=12,7)
    1
    1
        Week 24-severe (n=12,7)
    0
    0
        Week 48-none (n=8,3)
    6
    2
        Week 48-mild (n=8,3)
    1
    1
        Week 48-moderate (n=8,3)
    0
    0
        Week 48- severe (n=8,3)
    1
    0
        Week 72-none (n=8,3)
    7
    2
        Week 72-mild (n=8,3)
    1
    1
        Week 72-moderate (n=8,3)
    0
    0
        Week 72-severe (n=8,3)
    0
    0
        Week 96-none (n=8,3)
    7
    2
        Week 96-mild (n=8,3)
    1
    1
        Week 96-moderate (n=8,3)
    0
    0
        Week 96-severe (n=8,3)
    0
    0
        Week 120-none (n=8,3)
    8
    3
        Week 120-mild (n=8,3)
    0
    0
        Week 120-moderate (n=8,3)
    0
    0
        Week 120-severe (n=8,3)
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Clinical PNH Symptom Based on Severity: Headache

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    End point title
    		 Number of Participants With Clinical PNH Symptom Based on Severity: Headache
    End point description
    Data was reported for number of participants with clinical PNH symptom of headache. The severity of clinical PNH symptom of headache was graded as none, mild, moderate and severe based solely on investigator’s discretion. Participants in the mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=12,7)
    10
    7
        Baseline-mild (n=12,7)
    2
    0
        Baseline-moderate (n=12,7)
    0
    0
        Baseline-severe (n=12,7)
    0
    0
        Week 24-none (n=12,7)
    11
    6
        Week 24-mild (n=12,7)
    1
    1
        Week 24-moderate (n=12,7)
    0
    0
        Week 24-severe (n=12,7)
    0
    0
        Week 48-none (n=8,3)
    7
    2
        Week 48-mild (n=8,3)
    0
    0
        Week 48-moderate (n=8,3)
    1
    1
        Week 48- severe (n=8,3)
    0
    0
        Week 72-none (n=8,3)
    8
    3
        Week 72-mild (n=8,3)
    0
    0
        Week 72-moderate (n=8,3)
    0
    0
        Week 72-severe (n=8,3)
    0
    0
        Week 96-none (n=8,3)
    7
    3
        Week 96-mild (n=8,3)
    1
    0
        Week 96-moderate (n=8,3)
    0
    0
        Week 96-severe (n=8,3)
    0
    0
        Week 120-none (n=8,3)
    8
    2
        Week 120-mild (n=8,3)
    0
    1
        Week 120-moderate (n=8,3)
    0
    0
        Week 120-severe (n=8,3)
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical PNH Symptom Based on Severity: Erectile Dysfunction

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    End point title
    		 Number of Participants with Clinical PNH Symptom Based on Severity: Erectile Dysfunction
    End point description
    Data was reported for number of participants with clinical PNH symptom of erectile dysfunction. The severity of clinical PNH symptom of erectile dysfunction was graded as none, mild, moderate and severe based solely on investigator’s discretion. Participants in the mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=9,2)
    9
    2
        Baseline-mild (n=9,2)
    0
    0
        Baseline-moderate (n=9,2)
    0
    0
        Baseline-severe (n=9,2)
    0
    0
        Week 24-none (n=9,2)
    9
    2
        Week 24-mild (n=9,2)
    0
    0
        Week 24-moderate (n=9,2)
    0
    0
        Week 24-severe (n=9,2)
    0
    0
        Week 48-none (n=8,1)
    7
    1
        Week 48-mild (n=8,1)
    1
    0
        Week 48-moderate (n=8,1)
    0
    0
        Week 48- severe (n=8,1)
    0
    0
        Week 72-none (n=8,1)
    7
    1
        Week 72-mild (n=8,1)
    1
    0
        Week 72-moderate (n=8,1)
    0
    0
        Week 72-severe (n=8,1)
    0
    0
        Week 96-none (n=8,2)
    7
    2
        Week 96-mild (n=8,2)
    1
    0
        Week 96-moderate (n=8,2)
    0
    0
        Week 96-severe (n=8,2)
    0
    0
        Week 120-none (n=8,1)
    7
    1
        Week 120-mild (n=8,1)
    1
    0
        Week 120-moderate (n=8,1)
    0
    0
        Week 120-severe (n=8,1)
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical PNH Symptom Based on Severity: Hemoglobinuria

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    End point title
    		 Number of Participants with Clinical PNH Symptom Based on Severity: Hemoglobinuria
    End point description
    Data was reported for number of participants with clinical PNH symptom of hemoglobinuria. The severity of clinical PNH symptom of hemoglobinuria was graded as none, mild, moderate and severe based solely on investigator’s discretion. Participants in the mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=12,7)
    10
    7
        Baseline-mild (n=12,7)
    0
    0
        Baseline-moderate (n=12,7)
    0
    0
        Baseline-severe (n=12,7)
    0
    0
        Baseline-missing severity (n=12,7)
    2
    0
        Week 24-none (n=12,7)
    11
    5
        Week 24-mild (n=12,7)
    0
    0
        Week 24-moderate (n=12,7)
    0
    0
        Week 24-severe (n=12,7)
    0
    0
        Week 24-missing severity (n=12,7)
    1
    2
        Week 48-none (n=8,3)
    6
    3
        Week 48-mild (n=8,3)
    0
    0
        Week 48-moderate (n=8,3)
    1
    0
        Week 48- severe (n=8,3)
    0
    0
        Week 48-missing severity (n=8,3)
    1
    0
        Week 72-none (n=8,3)
    7
    2
        Week 72-mild (n=8,3)
    0
    1
        Week 72-moderate (n=8,3)
    0
    0
        Week 72-severe (n=8,3)
    0
    0
        Week 72-missing severity (n=8,3)
    1
    0
        Week 96-none (n=8,3)
    6
    3
        Week 96-mild (n=8,3)
    0
    0
        Week 96-moderate (n=8,3)
    0
    0
        Week 96-severe (n=8,3)
    0
    0
        Week 96-missing severity (n=8,3)
    2
    0
        Week 120-none (n=8,3)
    7
    3
        Week 120-mild (n=8,3)
    0
    0
        Week 120-moderate (n=8,3)
    0
    0
        Week 120-severe (n=8,3)
    0
    0
        Week 120-missing severity (n=8,3)
    1
    0
    No statistical analyses for this end point

    Secondary: Number of Participants with Clinical PNH Symptom Based on Severity: Jaundice

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    End point title
    		 Number of Participants with Clinical PNH Symptom Based on Severity: Jaundice
    End point description
    Data was reported for number of participants with clinical PNH symptom of jaundice. The severity of clinical PNH symptom of jaundice was graded as none, mild, moderate and severe based solely on investigator’s discretion. Participants in the population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Baseline-none (n=12,7)
    11
    7
        Baseline-mild (n=12,7)
    0
    0
        Baseline-moderate (n=12,7)
    0
    0
        Baseline-severe (n=12,7)
    0
    0
        Baseline-missing severity (n=12,7)
    1
    0
        Week 24-none (n=12,7)
    12
    7
        Week 24-mild (n=12,7)
    0
    0
        Week 24-moderate (n=12,7)
    0
    0
        Week 24-severe (n=12,7)
    0
    0
        Week 48-none (n=8,3)
    8
    3
        Week 48-mild (n=8,3)
    0
    0
        Week 48-moderate (n=8,3)
    0
    0
        Week 48- severe (n=8,3)
    0
    0
        Week 72-none (n=8,3)
    8
    3
        Week 72-mild (n=8,3)
    0
    0
        Week 72-moderate (n=8,3)
    0
    0
        Week 72-severe (n=8,3)
    0
    0
        Week 96-none (n=8,3)
    8
    3
        Week 96-mild (n=8,3)
    0
    0
        Week 96-moderate (n=8,3)
    0
    0
        Week 96-severe (n=8,3)
    0
    0
        Week 120-none (n=8,3)
    8
    3
        Week 120-mild (n=8,3)
    0
    0
        Week 120-moderate (n=8,3)
    0
    0
        Week 120-severe (n=8,3)
    0
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Lactate Dehydrogenase (LDH)

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    End point title
    		 Change From Baseline in Lactate Dehydrogenase (LDH)
    End point description
    Participants in the mITT population with available data were analyzed. Here “99999” represents that mean and standard deviation (SD) was not estimable as there were less than 2 participants at the given timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks, 24, 48, 72, 96, 120, and 144
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    6
    Units: Units per liter (U/L)
    arithmetic mean (standard deviation)
        Baseline (n=12,6)
    475.6 ( 210.98 )
    248.3 ( 125.71 )
        Change at Week 24 (n=11,5)
    -46.7 ( 154.55 )
    38.6 ( 45.51 )
        Change at Week 48 (n=8,2)
    -62.5 ( 80.97 )
    38.5 ( 65.76 )
        Change at Week 72 (n=8,2)
    -6.1 ( 238.41 )
    163.5 ( 118.09 )
        Change at Week 96 (n=8,2)
    237.4 ( 347.65 )
    -4.0 ( 134.35 )
        Change at Week 120 (n=8,2)
    195.4 ( 355.03 )
    31.0 ( 125.87 )
        Change at Week 144 (n=1,0)
    -59.0 ( 99999 )
    99999 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Hemoglobin

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    End point title
    		 Change From Baseline in Hemoglobin
    End point description
    Participants in the mITT population with available data were analyzed. Here “99999” represents that mean and SD was not estimable as there were less than 2 participants at the given timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, 120, and 144
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: grams per deciliter (g/dl)
    arithmetic mean (standard deviation)
        Baseline (n=12,7)
    11.88 ( 1.688 )
    11.53 ( 1.803 )
        Change at Week 24 (n=11,6)
    0.75 ( 1.159 )
    -1.32 ( 2.578 )
        Change at Week 48 (n=8,2)
    -0.61 ( 1.974 )
    -0.95 ( 2.333 )
        Change at Week 72 (n=8,3)
    -0.64 ( 2.327 )
    -0.93 ( 1.484 )
        Change at Week 96 (n=8,2)
    -0.33 ( 1.914 )
    -0.70 ( 2.687 )
        Change at Week 120 (n=8,3)
    0.38 ( 1.555 )
    -0.47 ( 2.098 )
        Change at Week 144 (n=1,0)
    -2.70 ( 99999 )
    99999 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Haptoglobin

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    End point title
    		 Change From Baseline in Haptoglobin
    End point description
    Participants in the mITT population with available data were analyzed. Here “99999” represents that mean and SD was not estimable as there were less than 2 participants at the given timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    5
    Units: grams per liter (g/L)
    arithmetic mean (standard deviation)
        Baseline (n=12,5)
    0.325 ( 0.2832 )
    0.216 ( 0.1350 )
        Change at Week 24 (n=11,5)
    0.000 ( 0.0447 )
    -0.082 ( 0.0844 )
        Change at Week 48 (n=8,0)
    0.213 ( 0.1126 )
    99999 ( 99999 )
        Change at Week 72 (n=8,2)
    0.025 ( 0.1165 )
    -0.210 ( 0.1414 )
        Change at Week 96 (n=8,2)
    -0.013 ( 0.0354 )
    0.195 ( 0.4313 )
        Change at Week 120 (n=8,1)
    -0.013 ( 0.0354 )
    -0.100 ( 99999 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Reticulocytes

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    End point title
    		 Change From Baseline in Reticulocytes
    End point description
    Participants in the mITT population with available data were analyzed
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, 96, and 120
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: 10^6 cells per microliter (μL)
    arithmetic mean (standard deviation)
        Baseline (n=12,7)
    0.1066 ( 0.02363 )
    0.0889 ( 0.03939 )
        Change at Week 24 (n=11,6)
    0.0164 ( 0.02339 )
    0.0913 ( 0.09144 )
        Change at Week 48 (n=8,2)
    0.0048 ( 0.03518 )
    0.0507 ( 0.03161 )
        Change at Week 72 (n=8,3)
    0.0044 ( 0.03643 )
    0.0463 ( 0.03008 )
        Change at Week 96 (n=8,2)
    0.0409 ( 0.03805 )
    0.0607 ( 0.03585 )
        Change at Week 120 (n=8,3)
    0.0173 ( 0.03303 )
    0.0653 ( 0.02380 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Blood Transfusions or Thromboses

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    End point title
    		 Number of Participants With Blood Transfusions or Thromboses
    End point description
    Data was reported for number of participants for whom blood transfusion was required or who experienced the thrombosis events. Participants in the mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to 3 weeks after last dose (Week 147)
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    12
    7
    Units: participants
    number (not applicable)
        Blood transfusions
    3
    4
        Thromboses
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Blood Transfusions

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    End point title
    		 Number of Blood Transfusions
    End point description
    Number of blood transfusions were reported. Participants from mITT population who required blood transfusions were evaluated.
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to 3 weeks after last dose (Week 147)
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    3
    4
    Units: Number of blood transfusions
    number (not applicable)
        Number of Blood Transfusion
    13
    25
    No statistical analyses for this end point

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

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    End point title
    		 Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score
    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 mITT population with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 48, 72, and 96
    End point values
    		 Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group C5 INH Inadequate Response Group
    Number of subjects analysed
    11
    7
    Units: Scores on a scale
    arithmetic mean (standard deviation)
        Baseline (n=11,7)
    43.82 ( 9.400 )
    40.14 ( 13.533 )
        Change at Week 24 (n=11,7)
    0.09 ( 4.110 )
    -3.36 ( 8.066 )
        Change at Week 48 (n=8,3)
    -0.63 ( 2.387 )
    2.33 ( 3.215 )
        Change at Week 72 (n=8,3)
    -0.38 ( 2.875 )
    -4.64 ( 12.016 )
        Change at Week 96 (n=8,3)
    -1.13 ( 4.357 )
    -18.00 ( 27.185 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study drug up to 3 weeks after last dose (Week 147)
    Adverse event reporting additional description
    The safety analysis population included all participants who received at least 1 capsule or tablet of study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24
    Reporting groups
    Reporting group title
    C5-INH Inadequate Response Group
    Reporting group description
    		 This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants were to receive BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.

    Reporting group title
    C5-INH Naïve Group
    Reporting group description
    		 This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.

    Serious adverse events
    		 C5-INH Inadequate Response Group C5-INH Naïve Group
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 7 (42.86%)
    6 / 12 (50.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Epstein-Barr virus associated lymphoma
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Breakthrough haemolysis
         subjects affected / exposed
    0 / 7 (0.00%)
    3 / 12 (25.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Febrile neutropenia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Haemolysis
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Crohn's disease
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastritis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Boutonneuse fever
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyelonephritis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Soft tissue infection
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 C5-INH Inadequate Response Group C5-INH Naïve Group
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 7 (100.00%)
    12 / 12 (100.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Vasculitis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Axillary pain
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Chest discomfort
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Chest pain
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Condition aggravated
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Fatigue
         subjects affected / exposed
    3 / 7 (42.86%)
    0 / 12 (0.00%)
         occurrences all number
    8
    0
    Feeling abnormal
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Influenza like illness
         subjects affected / exposed
    2 / 7 (28.57%)
    1 / 12 (8.33%)
         occurrences all number
    3
    1
    Oedema
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Pain
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Pyrexia
         subjects affected / exposed
    2 / 7 (28.57%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Erectile dysfunction
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Heavy menstrual bleeding
         subjects affected / exposed
    2 / 7 (28.57%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Pelvic pain
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Dyspnoea
         subjects affected / exposed
    3 / 7 (42.86%)
    1 / 12 (8.33%)
         occurrences all number
    8
    1
    Epistaxis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    3 / 7 (42.86%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    Painful respiration
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Productive cough
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Sinus disorder
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Sinus pain
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Depression
         subjects affected / exposed
    1 / 7 (14.29%)
    2 / 12 (16.67%)
         occurrences all number
    1
    3
    Insomnia
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Stress
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 7 (0.00%)
    4 / 12 (33.33%)
         occurrences all number
    0
    4
    Blood alkaline phosphatase increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Blood bilirubin increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Blood creatinine increased
         subjects affected / exposed
    0 / 7 (0.00%)
    5 / 12 (41.67%)
         occurrences all number
    0
    7
    Blood iron decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Blood uric acid increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    C-reactive protein increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Creatinine urine increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Fibrin D dimer increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Glomerular filtration rate decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Haemoglobin decreased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Neutrophil count increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    SARS-CoV-2 test positive
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Urine albumin/creatinine ratio increased
         subjects affected / exposed
    0 / 7 (0.00%)
    3 / 12 (25.00%)
         occurrences all number
    0
    6
    Weight increased
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Contusion
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Joint injury
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Ligament sprain
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Maternal exposure via partner during pregnancy
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Nervous system disorders
    Ageusia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Headache
         subjects affected / exposed
    4 / 7 (57.14%)
    4 / 12 (33.33%)
         occurrences all number
    12
    6
    Neuralgia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Restless legs syndrome
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Breakthrough haemolysis
         subjects affected / exposed
    4 / 7 (57.14%)
    4 / 12 (33.33%)
         occurrences all number
    4
    5
    Haemolysis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Lymphadenopathy
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Lymphopenia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    10
    Neutropenia
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    5
    Splenomegaly
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Thrombocytopenia
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    4
    Ear and labyrinth disorders
    Ear haemorrhage
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Tympanic membrane disorder
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Vertigo
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 12 (8.33%)
         occurrences all number
    4
    1
    Abdominal pain
         subjects affected / exposed
    2 / 7 (28.57%)
    2 / 12 (16.67%)
         occurrences all number
    9
    4
    Abdominal pain lower
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    Ascites
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Colitis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Constipation
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Diarrhoea
         subjects affected / exposed
    1 / 7 (14.29%)
    3 / 12 (25.00%)
         occurrences all number
    2
    4
    Dyspepsia
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Dysphagia
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    Gastritis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Nausea
         subjects affected / exposed
    1 / 7 (14.29%)
    4 / 12 (33.33%)
         occurrences all number
    3
    7
    Toothache
         subjects affected / exposed
    1 / 7 (14.29%)
    2 / 12 (16.67%)
         occurrences all number
    1
    2
    Vomiting
         subjects affected / exposed
    1 / 7 (14.29%)
    2 / 12 (16.67%)
         occurrences all number
    1
    3
    Hepatobiliary disorders
    Jaundice
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Skin and subcutaneous tissue disorders
    Dermatitis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Dermatitis allergic
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    5
    Erythema
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Photosensitivity reaction
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Pityriasis rosea
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Rash
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    8
    0
    Skin reaction
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Chromaturia
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Dysuria
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Haemoglobinuria
         subjects affected / exposed
    4 / 7 (57.14%)
    1 / 12 (8.33%)
         occurrences all number
    5
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Back pain
         subjects affected / exposed
    3 / 7 (42.86%)
    3 / 12 (25.00%)
         occurrences all number
    4
    4
    Joint swelling
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Muscle spasms
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Myalgia
         subjects affected / exposed
    2 / 7 (28.57%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Infections and infestations
    Bacterial vaginosis
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Bronchitis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    COVID-19
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Folliculitis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Fungal infection
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Gastroenteritis viral
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Genital herpes
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Helicobacter gastritis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Hordeolum
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Infection
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Influenza
         subjects affected / exposed
    2 / 7 (28.57%)
    1 / 12 (8.33%)
         occurrences all number
    3
    1
    Nasopharyngitis
         subjects affected / exposed
    3 / 7 (42.86%)
    2 / 12 (16.67%)
         occurrences all number
    6
    2
    Pharyngitis
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Post procedural infection
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Rhinovirus infection
         subjects affected / exposed
    2 / 7 (28.57%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Soft tissue infection
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    2
    Tonsillitis
         subjects affected / exposed
    0 / 7 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Tooth abscess
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 7 (14.29%)
    3 / 12 (25.00%)
         occurrences all number
    1
    4
    Urinary tract infection
         subjects affected / exposed
    1 / 7 (14.29%)
    3 / 12 (25.00%)
         occurrences all number
    1
    3
    Viral infection
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Vulvovaginal candidiasis
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 7 (14.29%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Dehydration
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Folate deficiency
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Hypomagnesaemia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Hyponatraemia
         subjects affected / exposed
    0 / 7 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Vitamin B12 deficiency
         subjects affected / exposed
    1 / 7 (14.29%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 Aug 2020
    Added an exclusion criterion for participants with elevated serum bilirubin. - Revised text for exclusion criteria for liver enzymes aspartate aminotransferase (AST) and alanine transaminase (ALT) for consistency with the new serum bilirubin exclusion criterion. - Added the requirement for participant discontinuation in the event of meningococcal infection or any serious infection that occurred after treatment was initiated. - Clarified that if the trial was to be halted due to safety concerns or based on a data monitoring committee (DMC) decision, restarting the trial would only occur following the appropriate authorization via a substantial amendment.
    12 Nov 2020
    Updated the visit schedule to every 4 week visits throughout the study for regular safety laboratory tests. Following findings of possible clinical chemistry changes in nonclinical toxicology studies, 4 week visits were continued after Week 24 as a precaution, - Updated text to include new nonclinical data. - Updated text to include new clinical data. - Clarification for tapering off or discontinuation of eculizumab or ravulizumab in former BCX9930 101 study participants who had added BCX9930 to their existing therapy with eculizumab or ravulizumab. - Updated information on prohibited medications. - Introduced additional text in case a new tablet formulation in development replaced the original hard gelatin capsule formulation. The introduction of the new tablet formulation was pending the results from a relative bioavailability study of the tablet and capsule formulations.
    24 Jun 2021
    Transitioned all participants from hard gelatin capsules to tablets. - Following assessment of the BCX9930-101 and BCX9930-201 study data, combined with pharmacokinetic (PK) modelling activities, it was concluded that 500 mg BID administered using the new tablet formulation was the most appropriate dose. This was the dose that was taken into the registration studies, BCX9930-202 and BCX9930-203. Therefore, all participants in this study were to take 500 mg BID with no dose modifications permitted. - Increased study treatment period from 48 weeks to 96 weeks to allow continued access following the assessment of chronic toxicology studies. - Updated the risk-benefit in accordance with the current available clinical and nonclinical data. - Updated the participant withdrawal criteria to provide adequate participant protection against treatment related injury following a review of the nonclinical data, and current available clinical data. - Updated dosing compliance language following availability of PK modelling data and transition to tablets. - Added laboratory parameters to strengthen ability to detect treatment emergent adverse changes.
    01 Jul 2022
    Participants enrolled into this study were to reach Week 96 in October and November 2022. Therefore, this amendment was submitted to extend the duration of treatment for an additional 48 weeks; i.e., up to Week 144. Additional measures for safety monitoring were included to mirror safety monitoring assessments in registration studies, BCX9930-202 and BCX9930-203.

    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 business reasons. Per change in planned analysis, data were analyzed and reported for safety and selected efficacy parameters.
    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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