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    Clinical Trial Results:
    A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy

    Summary
    EudraCT number
    		 2019-003830-17
    Trial protocol
    		 GB   IT   ES  
    Global end of trial date
    20 Mar 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    21 Nov 2024
    First version publication date
    21 Nov 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ACH228-110
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04170023
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals, Inc.
    Sponsor organisation address
    121 Seaport Boulevard, Boston, MA, United States, 02210
    Public contact
    European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +33 147100606, clinicaltrials.eu@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +33 147100606, clinicaltrials.eu@alexion.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
    20 Mar 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Mar 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main objective of the trial was to evaluate the efficacy of ALXN2050 based on improvement in hemoglobin (Hgb).
    Protection of trial subjects
    This study was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines including the Declaration of Helsinki, Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines, applicable International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) Guidelines, and other applicable laws and regulations.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 Dec 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    New Zealand: 9
    Country: Number of subjects enrolled
    Korea, Republic of: 6
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Canada: 3
    Country: Number of subjects enrolled
    Türkiye: 3
    Country: Number of subjects enrolled
    Italy: 4
    Country: Number of subjects enrolled
    Spain: 1
    Worldwide total number of subjects
    29
    EEA total number of subjects
    5
    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)
    27
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study included a 12-week Treatment Period and a 148-week or 200-week (for sites in New Zealand) Long-term Extension (LTE) Period.

    Period 1
    Period 1 title
    Treatment Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Group 1: Treatment Naive
    Arm description
    		 Participants who were treatment-naive received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN2050
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet, Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN2050 was administered per schedule specified in the arm description.

    Arm title
    		 Group 2: Eculizumab Switch
    Arm description
    		 Participants who had received component 5 (C5) inhibition with eculizumab for at least 6 months, who continued to experience anemia (Hgb <10 grams/deciliter [dL]) and reticulocytes above the upper limit of normal (ULN), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN2050
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN2050 was administered per schedule specified in the arm description.

    Arm title
    		 Group 3: Danicopan Rollover
    Arm description
    		 Participants who had received danicopan monotherapy during Study ACH471-103 (NCT03181633), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN2050
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN2050 was administered per schedule specified in the arm description.

    Number of subjects in period 1
    		Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Started
    12
    11
    6
    Received at least 1 dose of study drug
    12
    11
    6
    Completed
    12
    11
    6
    Period 2
    Period 2 title
    LTE Period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Group 1: Treatment Naive
    Arm description
    		 Participants who were treatment-naive received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN2050
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN2050 was administered per schedule specified in the arm description.

    Arm title
    		 Group 2: Eculizumab Switch
    Arm description
    		 Participants who had received component 5 (C5) inhibition with eculizumab for at least 6 months, who continued to experience anemia (hemoglobin [Hgb] <10 grams/deciliter [dL]) and reticulocytes above the upper limit of normal (ULN), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN2050
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN2050 was administered per schedule specified in the arm description.

    Arm title
    		 Group 3: Danicopan Rollover
    Arm description
    		 Participants who had received danicopan monotherapy during Study ACH471-103 (NCT03181633), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN2050
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    ALXN2050 was administered per schedule specified in the arm description.

    Number of subjects in period 2
    		Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Started
    12
    11
    6
    Received at least 1 dose of study drug
    12
    11
    6
    Completed
    0
    0
    1
    Not completed
    12
    11
    5
         Consent withdrawn by subject
    1
    -
    -
         Study Termination
    11
    11
    5

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Group 1: Treatment Naive
    Reporting group description
    		 Participants who were treatment-naive received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group title
    Group 2: Eculizumab Switch
    Reporting group description
    		 Participants who had received component 5 (C5) inhibition with eculizumab for at least 6 months, who continued to experience anemia (Hgb <10 grams/deciliter [dL]) and reticulocytes above the upper limit of normal (ULN), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group title
    Group 3: Danicopan Rollover
    Reporting group description
    		 Participants who had received danicopan monotherapy during Study ACH471-103 (NCT03181633), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover Total
    Number of subjects
    		 12 11 6 29
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 43.75 ( 17.099 ) 42.45 ( 13.419 ) 40.50 ( 16.718 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 4 7 3 14
        Male
    		 8 4 3 15
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0
        Asian
    		 8 1 0 9
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0
        Black or African American
    		 0 0 0 0
        White
    		 3 10 6 19
        More than one race
    		 0 0 0 0
        Unknown or Not Reported
    		 1 0 0 1
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0 0 0
        Not Hispanic or Latino
    		 12 10 6 28
        Unknown or Not Reported
    		 0 1 0 1
    HgB
    Units: grams/liter
        arithmetic mean (standard deviation)
    		 81.17 ( 11.731 ) 91.00 ( 9.602 ) 130.67 ( 19.180 ) -

    End points

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    End points reporting groups
    Reporting group title
    Group 1: Treatment Naive
    Reporting group description
    		 Participants who were treatment-naive received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group title
    Group 2: Eculizumab Switch
    Reporting group description
    		 Participants who had received component 5 (C5) inhibition with eculizumab for at least 6 months, who continued to experience anemia (Hgb <10 grams/deciliter [dL]) and reticulocytes above the upper limit of normal (ULN), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group title
    Group 3: Danicopan Rollover
    Reporting group description
    		 Participants who had received danicopan monotherapy during Study ACH471-103 (NCT03181633), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.
    Reporting group title
    Group 1: Treatment Naive
    Reporting group description
    		 Participants who were treatment-naive received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group title
    Group 2: Eculizumab Switch
    Reporting group description
    		 Participants who had received component 5 (C5) inhibition with eculizumab for at least 6 months, who continued to experience anemia (hemoglobin [Hgb] <10 grams/deciliter [dL]) and reticulocytes above the upper limit of normal (ULN), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group title
    Group 3: Danicopan Rollover
    Reporting group description
    		 Participants who had received danicopan monotherapy during Study ACH471-103 (NCT03181633), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Primary: Change From Baseline in Hgb at Week 12

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    End point title
    		 Change From Baseline in Hgb at Week 12 [1]
    End point description
    Hgb baseline was defined as the lowest Hgb value observed between and including screening and first dose date. To address the impact of transfusion, Hgb values collected within 4 weeks after transfusion were not included in the primary efficacy analysis. Change from Baseline = Hgb at Week 12 - Baseline Hgb. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Primary
    End point timeframe
    Baseline, Week 12
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Statistical analysis was not planned for this endpoint.
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    11
    10
    6
    Units: grams/liter
        arithmetic mean (standard deviation)
    35.6 ( 14.70 )
    32.5 ( 20.03 )
    -3.7 ( 15.77 )
    No statistical analyses for this end point

    Secondary: Number of Participants who had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050

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    End point title
    		 Number of Participants who had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050
    End point description
    Transfusion avoidance: participants remained transfusion-free and did not require a transfusion during the period of interest. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study.
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    12
    11
    6
    Units: participants
    9
    10
    5
    No statistical analyses for this end point

    Secondary: Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment

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    End point title
    		 Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment
    End point description
    Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study.
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    12
    11
    6
    Units: RBC units
        arithmetic mean (standard deviation)
    0.6 ( 1.24 )
    0.1 ( 0.30 )
    0.8 ( 2.04 )
    No statistical analyses for this end point

    Secondary: Number of Transfusion Instances During 12 Weeks of Treatment

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    End point title
    		 Number of Transfusion Instances During 12 Weeks of Treatment
    End point description
    Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study.
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    12
    11
    6
    Units: transfusion instances
        arithmetic mean (standard deviation)
    0.4 ( 0.90 )
    0.1 ( 0.30 )
    0.5 ( 1.22 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12

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    End point title
    		 Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12
    End point description
    Change from Baseline = Serum LDH levels at Week 12 - Baseline Serum LDH levels. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    11
    11
    6
    Units: units/liter
        arithmetic mean (standard deviation)
    -1310.8 ( 424.89 )
    160.2 ( 279.57 )
    88.7 ( 265.00 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Absolute Reticulocyte Count at Week 12

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    End point title
    		 Change From Baseline in Absolute Reticulocyte Count at Week 12
    End point description
    Change from Baseline = absolute reticulocyte count at Week 12 - Baseline reticulocyte count. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    12
    10
    5
    Units: 10^3 cells/microliter (μL)
        arithmetic mean (standard deviation)
    -100.6 ( 72.18 )
    -179.0 ( 117.30 )
    -24.0 ( 22.10 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Direct and Total Bilirubin at Week 12

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    End point title
    		 Change From Baseline in Direct and Total Bilirubin at Week 12
    End point description
    Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for the specified category.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    11
    11
    6
    Units: micromoles (μmol)/liter
    arithmetic mean (standard deviation)
        Direct Bilirubin
    -2.1 ( 2.10 )
    -6.3 ( 5.16 )
    0.4 ( 0.89 )
        Total Bilirubin
    -12.9 ( 10.07 )
    -23.3 ( 24.89 )
    1.5 ( 2.81 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12

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    End point title
    		 Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12
    End point description
    The PNH RBC clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 12 - Baseline PNH clone size. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    9
    3
    2
    Units: percentage of the total cell population
        arithmetic mean (standard deviation)
    41.9 ( 13.68 )
    27.5 ( 21.60 )
    -12.0 ( 26.94 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12

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    End point title
    		 Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12
    End point description
    C3 fragment deposition on PNH RBC was used as a marker of intra and extravascular hemolysis. Data are presented for the change from baseline to Week 12 in percentage of PNH RBCs with C3 fragment deposition. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    10
    3
    2
    Units: percentage of PNH RBC
        arithmetic mean (standard deviation)
    0.0 ( 0.00 )
    -388.6 ( 590.08 )
    0.3 ( 0.35 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication

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    End point title
    		 Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication
    End point description
    An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an important medical event or reaction. A TEAE was defined as an AE that emerged during treatment, had been absent prior to treatment, or worsened relative to the pretreatment state. A summary of all SAEs and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. Safety set included all participants who received at least 1 dose of ALXN2050 in this study.
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to Week 217
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    12
    11
    6
    Units: participants
        Any AEs
    12
    10
    6
        SAEs
    7
    5
    2
        AEs Leading to Discontinuation of Study Medication
    1
    1
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Hgb at the End of Treatment (EOT) During the LTE Period

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    End point title
    		 Change From Baseline in Hgb at the End of Treatment (EOT) During the LTE Period
    End point description
    Hgb baseline was defined as the lowest Hgb value observed between and including screening and first dose date. Change from Baseline = Hgb at the EOT visit - Baseline Hgb. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT visit (Maximum exposure: 213.4 weeks)
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    10
    8
    5
    Units: grams/liter
        arithmetic mean (standard deviation)
    44.9 ( 21.07 )
    32.5 ( 7.17 )
    2.4 ( 19.44 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in LDH at the EOT During the LTE Period

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    End point title
    		 Change From Baseline in LDH at the EOT During the LTE Period
    End point description
    Change from Baseline = Serum LDH levels at the EOT visit - Baseline Serum LDH levels. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT visit (Maximum exposure: 213.4 weeks)
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    11
    10
    5
    Units: units/liter
        arithmetic mean (standard deviation)
    -1084.1 ( 738.26 )
    95.7 ( 223.88 )
    -61.4 ( 62.85 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale (Version 4) Total Score at Week 12

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    End point title
    		 Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale (Version 4) Total Score at Week 12
    End point description
    The FACIT-Fatigue scale is a collection of quality-of-life questionnaires pertaining to the management of fatigue symptoms due to chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores ranged from 0 to 52, with higher score indicating better quality of life. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 12
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    11
    7
    6
    Units: units on a scale
        arithmetic mean (standard deviation)
    11.2 ( 13.20 )
    5.1 ( 8.47 )
    0.2 ( 3.13 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in FACIT-Fatigue Scale (Version 4) Total Score at the EOT During the LTE Period

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    End point title
    		 Change From Baseline in FACIT-Fatigue Scale (Version 4) Total Score at the EOT During the LTE Period
    End point description
    The FACIT-Fatigue scale is a collection of quality-of-life questionnaires pertaining to the management of fatigue symptoms due to chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores ranged from 0 to 52, with higher score indicating better quality of life. Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT visit (Maximum exposure: 213.4 weeks)
    End point values
    		 Group 1: Treatment Naive Group 2: Eculizumab Switch Group 3: Danicopan Rollover
    Number of subjects analysed
    11
    9
    5
    Units: units on a scale
        arithmetic mean (standard deviation)
    12.6 ( 12.51 )
    4.4 ( 7.26 )
    -3.4 ( 10.26 )
    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 Week 217
    Adverse event reporting additional description
    Safety set included all participants who received at least 1 dose of ALXN2050 in this study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    Group 1: Treatment Naive
    Reporting group description
    		 Participants who were treatment-naive received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group title
    Group 3: Danicopan Rollover
    Reporting group description
    		 Participants who had received danicopan monotherapy during Study ACH471-103 (NCT03181633), switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Reporting group title
    Group 2: Eculizumab Switch
    Reporting group description
    		 Participants who had received C5 inhibition with eculizumab for at least 6 months, who continued to experience anemia (Hgb <10 grams/dL) and reticulocytes above the ULN, switched to ALXN2050 in this study and received ALXN2050 orally for 12 weeks during the treatment period. At the end of Week 12, participants entered the 148-week LTE Period and continued to receive ALXN2050 orally.

    Serious adverse events
    		 Group 1: Treatment Naive Group 3: Danicopan Rollover Group 2: Eculizumab Switch
    Total subjects affected by serious adverse events
         subjects affected / exposed
    7 / 12 (58.33%)
    2 / 6 (33.33%)
    5 / 11 (45.45%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (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
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Breakthrough haemolysis
         subjects affected / exposed
    4 / 12 (33.33%)
    1 / 6 (16.67%)
    2 / 11 (18.18%)
         occurrences causally related to treatment / all
    0 / 5
    1 / 1
    1 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Intravascular haemolysis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Haemolysis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (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
    Immune system disorders
    Anaphylactic reaction
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Haemoglobinuria
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (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
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Campylobacter gastroenteritis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (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
    COVID-19
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Staphylococcal bacteraemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (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
    Meningitis aseptic
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection bacterial
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Group 1: Treatment Naive Group 3: Danicopan Rollover Group 2: Eculizumab Switch
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 12 (100.00%)
    6 / 6 (100.00%)
    10 / 11 (90.91%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Lipoma
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Flushing
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Chills
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    1
    0
    3
    Fatigue
         subjects affected / exposed
    6 / 12 (50.00%)
    3 / 6 (50.00%)
    1 / 11 (9.09%)
         occurrences all number
    6
    3
    1
    Influenza like illness
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 6 (33.33%)
    2 / 11 (18.18%)
         occurrences all number
    1
    2
    8
    Vaccination site pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Catheter site rash
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Asthenia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Chest discomfort
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    General physical health deterioration
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Hyperthermia
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    0
    Malaise
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    0
    3
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Oedema
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Oedema peripheral
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    2
    Pyrexia
         subjects affected / exposed
    2 / 12 (16.67%)
    2 / 6 (33.33%)
    2 / 11 (18.18%)
         occurrences all number
    2
    2
    2
    Rebound effect
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Nipple pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Nasal congestion
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 6 (33.33%)
    1 / 11 (9.09%)
         occurrences all number
    0
    2
    1
    Atelectasis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Dysphonia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Haemoptysis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Hypoxia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    1
    Pleural effusion
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Rhinorrhoea
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Upper respiratory tract irritation
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Psychiatric disorders
    Sleep disorder
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    Anxiety
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    2 / 11 (18.18%)
         occurrences all number
    0
    2
    2
    Attention deficit hyperactivity disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Depression
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    1
    1
    0
    Insomnia
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    0
    Investigations
    Liver function test increased
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    International normalised ratio increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    3 / 11 (27.27%)
         occurrences all number
    0
    1
    4
    Blood creatine increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    2
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    2
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    3 / 11 (27.27%)
         occurrences all number
    0
    0
    4
    Cardiac murmur
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Blood bicarbonate decreased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Blood bilirubin increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Blood creatinine increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Epstein-Barr virus antibody positive
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Glomerular filtration rate decreased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Hepatic enzyme increased
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    2
    0
    1
    Low density lipoprotein increased
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Troponin increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Weight decreased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Weight increased
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Free haemoglobin present
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 6 (16.67%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    1
    Ankle fracture
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Foot fracture
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Immunisation reaction
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Joint dislocation
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Limb injury
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Radius fracture
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Scar
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Skin abrasion
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Tendon injury
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Transfusion reaction
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Upper limb fracture
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    6 / 12 (50.00%)
    1 / 6 (16.67%)
    3 / 11 (27.27%)
         occurrences all number
    13
    2
    6
    Taste disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Paraesthesia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Migraine
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    4
    Memory impairment
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Disturbance in attention
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Dizziness
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 6 (33.33%)
    2 / 11 (18.18%)
         occurrences all number
    1
    2
    2
    Hypoaesthesia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Restless legs syndrome
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    0
    2
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Anaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    1
    0
    11
    Haemolysis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    4 / 11 (36.36%)
         occurrences all number
    0
    0
    10
    Breakthrough haemolysis
         subjects affected / exposed
    3 / 12 (25.00%)
    1 / 6 (16.67%)
    2 / 11 (18.18%)
         occurrences all number
    5
    1
    3
    Aplastic anaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Haemolytic anaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Intravascular haemolysis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    Eye disorders
    Episcleritis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Cataract
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Conjunctival hyperaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Exudative retinopathy
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Retinal degeneration
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Vitreous floaters
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    9
    0
    0
    Abdominal pain
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    4
    0
    4
    Vomiting
         subjects affected / exposed
    4 / 12 (33.33%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    5
    0
    4
    Toothache
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Tongue geographic
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Stomatitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Pancreatitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Enteritis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    Dyspepsia
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    4
    0
    1
    Diarrhoea
         subjects affected / exposed
    2 / 12 (16.67%)
    1 / 6 (16.67%)
    2 / 11 (18.18%)
         occurrences all number
    3
    1
    2
    Nausea
         subjects affected / exposed
    4 / 12 (33.33%)
    1 / 6 (16.67%)
    2 / 11 (18.18%)
         occurrences all number
    4
    1
    4
    Large intestine polyp
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Abdominal distension
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Chronic gastritis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Constipation
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Gastritis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    4
    0
    0
    Gingival hypertrophy
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Haemorrhoidal haemorrhage
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Haemorrhoids
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    1
    1
    0
    Hiatus hernia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Mouth ulceration
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Jaundice
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    0
    3
    Autoimmune hepatitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Cholelithiasis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Hepatic steatosis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Rash pruritic
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Rash
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Night sweats
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    Pruritus
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Ecchymosis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Erythema
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Rash maculo-papular
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    Yellow skin
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Renal and urinary disorders
    Renal impairment
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Haemoglobinuria
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    6
    0
    4
    Haematuria
         subjects affected / exposed
    3 / 12 (25.00%)
    2 / 6 (33.33%)
    4 / 11 (36.36%)
         occurrences all number
    5
    2
    5
    Chromaturia
         subjects affected / exposed
    2 / 12 (16.67%)
    2 / 6 (33.33%)
    1 / 11 (9.09%)
         occurrences all number
    2
    4
    1
    Acute kidney injury
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    Dysuria
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Choluria
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 6 (16.67%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    1
    Muscle twitching
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Pain in extremity
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 6 (33.33%)
    4 / 11 (36.36%)
         occurrences all number
    2
    3
    7
    Fibromyalgia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Arthralgia
         subjects affected / exposed
    5 / 12 (41.67%)
    1 / 6 (16.67%)
    4 / 11 (36.36%)
         occurrences all number
    6
    1
    5
    Arthritis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Flank pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Muscle spasms
         subjects affected / exposed
    3 / 12 (25.00%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    0
    Myalgia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Neck pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Rotator cuff syndrome
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Tendonitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Joint swelling
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    Otitis media
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    0
    2
    Nasopharyngitis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    2
    Bacterial infection
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Pulpitis dental
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Urinary tract infection
         subjects affected / exposed
    2 / 12 (16.67%)
    1 / 6 (16.67%)
    2 / 11 (18.18%)
         occurrences all number
    2
    1
    2
    Tinea cruris
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    3
    Rhinitis
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    1
    1
    0
    Abscess limb
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Bronchitis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Tinea pedis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Cellulitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Conjunctivitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Cystitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Ear infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Furuncle
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    Gastroenteritis
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    2
    0
    1
    Influenza
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Onychomycosis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Oral herpes
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Pneumocystis jirovecii pneumonia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Pyelonephritis acute
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory syncytial virus infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Skin infection
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    COVID-19
         subjects affected / exposed
    4 / 12 (33.33%)
    3 / 6 (50.00%)
    2 / 11 (18.18%)
         occurrences all number
    5
    3
    2
    Tinea versicolour
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 12 (50.00%)
    2 / 6 (33.33%)
    1 / 11 (9.09%)
         occurrences all number
    10
    3
    1
    Metabolism and nutrition disorders
    Vitamin B12 deficiency
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Iron deficiency
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 6 (16.67%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Hyperglycaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    Hyperlipidaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Hypervolaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Hypokalaemia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Hypophagia
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 6 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Apr 2020
    This global amendment was initiated to allow females of childbearing potential to participate in the study and to align the protocol with Alexion standards in all applicable sections. The changes include updates to the eligibility criteria, SAE reporting, list of protocol-specific laboratory assessments, vaccination requirements, and the statistical sections. The Schedule of Activities was updated to reflect these changes.
    07 Jan 2021
    This global amendment was initiated to align the participant population of this Phase 2 study with the expected Phase 3 populations and to explore the efficacy and safety of ALXN2050 as a monotherapy option for participants with PNH. The changes include updates to the study design, eligibility criteria, and clarification of the endpoints. Changes were also made to further align the protocol with Alexion standards in all applicable sections, including SAE reporting, list of protocol-specific laboratory assessments, vaccination requirements, and other safety monitoring and the statistical sections. The Schedule of Activities was updated to reflect these changes.
    12 Apr 2021
    This global amendment was initiated to add back an exclusion criterion regarding history or presence of any risk factors for Torsades de Pointes, a screening QTcF interval > 450 milliseconds (msec) for males and > 470 msec for females, or receiving medications known to significantly increase QTc interval, which was inadvertently removed from Protocol Amendment 2.0.
    03 May 2021
    This global amendment was initiated to remove the following language from the dose-escalation instructions: “Dose escalation to 180 mg bid will be allowed once additional safety data from the currently ongoing Study ALXN2050-HV-107 is available. The Sponsor will inform study sites when dose escalation can be implemented.” Preliminary data from Study ALXN2050-HV-107 were now available to support the dose escalation to proceed. This amendment also included additional information regarding prohibited medications (that is, list of inhibitors, inducers, and substrates of cytochrome P3A [CYP3A], and list of medications known to lower seizure threshold).
    07 Oct 2021
    This global amendment was initiated to extend the LTE Period for an additional 52 weeks to collect safety and efficacy data for longer term. This amendment also included: • Clarification of exclusion criteria and additional prohibited medications to align with exclusion criteria. • Changes implemented in response to questions from the regulatory body in Germany (Protocol Amendment 3.2).

    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.
    None reported
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    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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