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    Clinical Trial Results:
    A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy

    Summary
    EudraCT number
    		 2020-004438-39
    Trial protocol
    		 FR   HU   ES   SK   NL   IT  
    Global end of trial date
    14 Sep 2023

    Results information
    Results version number
    		 v2(current)
    This version publication date
    24 May 2025
    First version publication date
    29 Sep 2024
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    Need to align with CT.gov draft post NIH comments.

    Trial information

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    Trial identification
    Sponsor protocol code
    BCX9930-202
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05116774
    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
    14 Sep 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Sep 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To determine the efficacy of oral BCX9930 monotherapy administered for 24 weeks, compared to continued complement component 5 (C5) inhibitor therapy, in participants with paroxysmal nocturnal hemoglobinuria (PNH) with an inadequate response to C5 inhibitor therapy.
    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
    06 Dec 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
    Spain: 2
    Country: Number of subjects enrolled
    France: 4
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    United Kingdom: 5
    Worldwide total number of subjects
    12
    EEA total number of subjects
    7
    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)
    7
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted in France, Hungary, Italy, Spain, and the United Kingdom (UK).

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

    Period 1
    Period 1 title
    Part 1 (Up to 24 weeks)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    No

    Arm title
    		 BCX9930/BCX9930
    Arm description
    		 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) orally and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.
    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-Inhibitor (C5-INH)/BCX9930
    Arm description
    		 Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Soliris
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered by intravenous infusion per current dose regimen.

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    Other name
    Ultomiris, ALXN1210, ravulizumab-cwvz
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous drip use
    Dosage and administration details
    Administered by intravenous infusion per current dose regimen

    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/BCX9930 C5-Inhibitor (C5-INH)/BCX9930
    Started
    8
    4
    Completed
    7
    3
    Not completed
    1
    1
         Consent withdrawn by subject
    -
    1
         Adverse event, non-fatal
    1
    -
    Period 2
    Period 2 title
    Part 2 (Up to 52 weeks)
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 BCX9930/BCX9930
    Arm description
    		 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) orally and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.
    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-Inhibitor (C5-INH)/BCX9930
    Arm description
    		 Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Soliris
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered by intravenous infusion per current dose regimen.

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    Other name
    Ultomiris, ALXN1210, ravulizumab-cwvz
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous drip use
    Dosage and administration details
    Administered by intravenous infusion per current dose regimen

    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 2
    		BCX9930/BCX9930 C5-Inhibitor (C5-INH)/BCX9930
    Started
    7
    3
    Completed
    3
    2
    Not completed
    4
    1
         Adverse event, non-fatal
    1
    -
         Miscellaneous
    3
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    BCX9930/BCX9930
    Reporting group description
    		 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) orally and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.

    Reporting group title
    C5-Inhibitor (C5-INH)/BCX9930
    Reporting group description
    		 Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.

    Reporting group values
    		 BCX9930/BCX9930 C5-Inhibitor (C5-INH)/BCX9930 Total
    Number of subjects
    		 8 4 12
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 58.8 ( 17.66 ) 48.5 ( 14.25 ) -
    Gender categorical
    Units: Subjects
        Female
    		 4 3 7
        Male
    		 4 1 5
    Ethnicity
    Units: Subjects
        Not Hispanic or Latino
    		 5 2 7
        Unknown or Not Reported
    		 3 2 5
    Race
    Units: Subjects
        Asian
    		 0 1 1
        Black or African American
    		 2 0 2
        White
    		 5 2 7
        Unknown or Not Reported
    		 1 1 2

    End points

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    End points reporting groups
    Reporting group title
    BCX9930/BCX9930
    Reporting group description
    		 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) orally and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.

    Reporting group title
    C5-Inhibitor (C5-INH)/BCX9930
    Reporting group description
    		 Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.
    Reporting group title
    BCX9930/BCX9930
    Reporting group description
    		 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) orally and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.

    Reporting group title
    C5-Inhibitor (C5-INH)/BCX9930
    Reporting group description
    		 Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.

    Subject analysis set title
    BCX9930
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.

    Subject analysis set title
    C5-INH
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

    Primary: Part 1: Change From Baseline in Hemoglobin at Week 24

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    End point title
    		 Part 1: Change From Baseline in Hemoglobin at Week 24 [1]
    End point description
    Participants in the All Subjects as Treated (ASaT) population in Part 1 with available data were analyzed.
    End point type
    Primary
    End point timeframe
    Baseline, Week 24
    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
    		 BCX9930 C5-INH
    Number of subjects analysed
    8
    4
    Units: grams per deciliter (g/dL)
    arithmetic mean (standard deviation)
        Baseline (n=8, n=4)
    9.12 ( 1.096 )
    9.13 ( 0.847 )
        Change at Week 24 (n=6, n=2)
    3.48 ( 0.674 )
    0.72 ( 0.884 )
    No statistical analyses for this end point

    Secondary: Part 1: Number of Participants Who Were Transfusion-free

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    End point title
    		 Part 1: 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 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (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 in Part 1 were analyzed.
    End point type
    Secondary
    End point timeframe
    From Week 4 to Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    8
    4
    Units: participants
    7
    4
    No statistical analyses for this end point

    Secondary: Part 1: Number of Units of pRBCs Transfused

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    End point title
    		 Part 1: Number of Units of pRBCs Transfused
    End point description
    Participants in the ASaT population in Part 1 were analyzed.
    End point type
    Secondary
    End point timeframe
    From Week 4 to Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    8
    4
    Units: units of pRBCs
    0
    0
    No statistical analyses for this end point

    Secondary: Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

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    End point title
    		 Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale 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 ASaT population in Part 1 with available data were analyzed. '99999' signifies standard deviation could not be calculated due to single participant.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    6
    2
    Units: Scores on a scale
    arithmetic mean (standard deviation)
        Baseline (n=6, n=2)
    36.3 ( 9.69 )
    40.0 ( 8.49 )
        Change at Week 24 (n=3, n=1)
    -0.3 ( 12.86 )
    1.0 ( 99999 )
    No statistical analyses for this end point

    Secondary: Part 1: Percent Change From Baseline in Lactate Dehydrogenase

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    End point title
    		 Part 1: Percent Change From Baseline in Lactate Dehydrogenase
    End point description
    Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    7
    2
    Units: percent change
        arithmetic mean (standard deviation)
    24.3 ( 53.07 )
    -21.0 ( 4.03 )
    No statistical analyses for this end point

    Secondary: Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused

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    End point title
    		 Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused
    End point description
    The rate of pRBC units transfused from Week 4 to Week 24 was calculated and compared to the rate of pRBC units transfused prestudy during the 12 months prior to screening. The percent reduction in rate of pRBC units transfused was the percent difference in rate relative to the prestudy rate, calculated as: (current rate - prestudy rate)/prestudy rate * 100%. Total rate among all participants was evaluated here. Rate of pRBC units transfusion was defined as the percentage of participants who received pRBC transfusions. Participants in the ASaT population in Part 1 were analyzed. 99999= Not evaluable. No participants received pRBC transfusion at pre-study in the "C5-INH" arm, and therefore the percentage reduction, based on the formula provided, was not calculable.
    End point type
    Secondary
    End point timeframe
    Prestudy (12 months prior to screening) and from Week 4 to Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    8
    4
    Units: percent reduction
    100
    99999
    No statistical analyses for this end point

    Secondary: Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL)

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    End point title
    		 Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL)
    End point description
    Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    6
    4
    Units: participants
    6
    0
    No statistical analyses for this end point

    Secondary: Part 1: Number of Participants Who Achieved Hemoglobin Stabilization

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    End point title
    		 Part 1: Number of Participants Who Achieved Hemoglobin Stabilization
    End point description
    Hemoglobin stabilization was defined as the participants who avoided 2 g/dL or greater decrease in hemoglobin in the absence of transfusion from Week 4 to Week 24. Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    From Week 4 to Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    5
    4
    Units: participants
    5
    4
    No statistical analyses for this end point

    Secondary: Part 1: Change From Baseline in Complement Component 3 (C3)-Opsonized Red Blood Cells (RBCs)

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    End point title
    		 Part 1: Change From Baseline in Complement Component 3 (C3)-Opsonized Red Blood Cells (RBCs)
    End point description
    Red blood cells opsonized by C3 were to be assessed by flow cytometry. No data was collected for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: percentage of opsonized RBCs
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [2] - No data was collected for this endpoint.
    [3] - No data was collected for this endpoint.
    No statistical analyses for this end point

    Secondary: Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size

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    End point title
    		 Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size
    End point description
    The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBC cells within the total RBC population. Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    6
    3
    Units: % of PNH-RBC within total RBC population
    arithmetic mean (standard deviation)
        Baseline (n=6,3)
    80.51 ( 16.94 )
    83.40 ( 14.34 )
        Change at Week 24 (n=3,2)
    23.18 ( 11.81 )
    -6.34 ( 8.986 )
    No statistical analyses for this end point

    Secondary: Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size

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    End point title
    		 Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size
    End point description
    The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBCs within the total RBC population. The PNH WBC clone size refers to the percentage of PNH-affected WBCs within the total WBC population. The ratio of total PNH RBC clone size to PNH WBC clone size = ratio of percent total PNH RBCs / percent PNH WBCs. Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    4
    2
    Units: ratio
    arithmetic mean (standard deviation)
        Baseline (n=4,2)
    0.8128 ( 0.16528 )
    0.9059 ( 0.12113 )
        Change at Week 24 (n=3,2)
    0.2336 ( 0.11595 )
    -0.0668 ( 0.09462 )
    No statistical analyses for this end point

    Secondary: Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)

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    End point title
    		 Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)
    End point description
    Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    7
    4
    Units: 10^6 cells/microliter (μL)
    arithmetic mean (standard deviation)
        Baseline (n=7,4)
    0.2933 ( 0.10453 )
    0.3129 ( 0.16201 )
        Change at Week 24 (n=6,4)
    -0.2142 ( 0.05384 )
    -0.0462 ( 0.01823 )
    No statistical analyses for this end point

    Secondary: Part 1: Number of Participants With ARC in the Normal Range

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    End point title
    		 Part 1: Number of Participants With ARC in the Normal Range
    End point description
    Number of participants with ARC in the normal range (0.03 - 0.12 10^6 cells/uL) were reported. Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    6
    4
    Units: participants
    4
    1
    No statistical analyses for this end point

    Secondary: Part 1: Change From Baseline in Haptoglobin

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    End point title
    		 Part 1: Change From Baseline in Haptoglobin
    End point description
    Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    7
    4
    Units: grams per liter (g/L)
    arithmetic mean (standard deviation)
        Baseline
    0.126 ( 0.0732 )
    0.105 ( 0.0268 )
        Change at Week 24
    -0.024 ( 0.0409 )
    -0.015 ( 0.0268 )
    No statistical analyses for this end point

    Secondary: Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range

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    End point title
    		 Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range
    End point description
    Number of Participants With Haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported. Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    7
    4
    Units: participants
    0
    0
    No statistical analyses for this end point

    Secondary: Part 1: Change From Baseline in Total Bilirubin

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    End point title
    		 Part 1: Change From Baseline in Total Bilirubin
    End point description
    Participants in the ASaT population in Part 1 with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 BCX9930 C5-INH
    Number of subjects analysed
    7
    4
    Units: milligram per deciliter (mg/dL)
    arithmetic mean (standard deviation)
        Baseline
    3.44 ( 3.679 )
    1.10 ( 0.424 )
        Change at Week 24
    -2.66 ( 3.672 )
    -0.02 ( 0.250 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Day 407
    Adverse event reporting additional description
    The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24
    Reporting groups
    Reporting group title
    BCX9930/BCX9930
    Reporting group description
    		 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.

    Reporting group title
    BCX9930 After C5-INH
    Reporting group description
    		 Participants who were initially randomized to continue C5-INH therapy received BCX9930 monotherapy after they had completed 24 weeks on C5-INH, or earlier after the date when sponsor decided to halt enrolment in the study permanently and terminate the study. Initially participants were to receive BCX9930 500 mg BID orally. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 was 197 days.

    Reporting group title
    C5-INH
    Reporting group description
    		 Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH was 24 weeks.

    Serious adverse events
    		 BCX9930/BCX9930 BCX9930 After C5-INH C5-INH
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (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
    Skin and subcutaneous tissue disorders
    Rash erythematous
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (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
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 BCX9930/BCX9930 BCX9930 After C5-INH C5-INH
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 8 (87.50%)
    3 / 3 (100.00%)
    3 / 4 (75.00%)
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    3 / 4 (75.00%)
         occurrences all number
    1
    0
    4
    Chest pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Face oedema
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Fatigue
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    5
    0
    0
    Pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Reproductive system and breast disorders
    Erectile dysfunction
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Scrotal oedema
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    0
    Dyspnoea exertional
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    0
    0
    2
    Rhinorrhoea
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Investigations
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Prothrombin time prolonged
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Tachycardia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 8 (50.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    0
    Lethargy
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Loss of consciousness
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorder
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Sciatica
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Haemolysis
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Eye disorders
    Vision blurred
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Abdominal pain upper
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Diarrhoea
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    0
    Dysphagia
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    Inguinal hernia
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Nausea
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Hepatobiliary disorders
    Hepatic cytolysis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Jaundice
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    3 / 8 (37.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    0
    Rash erythematous
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Rash macular
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    0
    Rash maculo-papular
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Chromaturia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    Haemoglobinuria
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Urinary incontinence
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    Axillary mass
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Back pain
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 3 (0.00%)
    2 / 4 (50.00%)
         occurrences all number
    3
    0
    2
    Bone pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Myalgia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    COVID-19
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    0
    Influenza
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    0
    1
    Localised infection
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    0
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    0
    Respiratory tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Subcutaneous abscess
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 3 (33.33%)
    0 / 4 (0.00%)
         occurrences all number
    1
    1
    0
    Viral infection
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    0
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    0 / 4 (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 secondary efficacy endpoints for Part 1 were revised to emphasize key secondary endpoints that would demonstrate clinical benefit in support of the primary endpoint of change from baseline in hemoglobin (Hb). - 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 sample size calculation was reworked using an assumed treatment difference of 2 g/dL CFB in Hb (previously 1.5 g/dL) for participants randomly assigned to BCX9930 compared to participants randomly assigned to continue C5 INH therapy. This change allowed for the overall sample size to be reduced from 135 to 81 participants (with 54 participants in the BCX9930 group and 27 participants in the continued C5 INH therapy group). - The benefit-risk text was updated in accordance with the currently available clinical and non-clinical data. - Section (Prohibited and Restricted Medications) was extensively revised to take into account the preliminary results from a recently completed drug-drug interaction study, BCX9930 102. - The requirement for the screening Hb value to be from a blood sample collected prior to pRBC transfusion or at least 14 days after transfusion was removed.
    29 Jun 2022
    - Recommended dose of BCX9930 was reduced from 500 mg BID to 400 mg BID for all participants. - For newly randomly assigned 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. - Revised guidance was provided for the management of participants with treatment-emergent increases in serum creatinine, including the discontinuation of any participant with a confirmed increase in serum creatinine. - An independent Nephrology Risk Mitigation Working Group was established. - 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 terminate the development program including this study.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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