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    Clinical Trial Results:
    A Phase 3 Multi-center, Open-label Study to Evaluate the Efficacy and Safety of Lanadelumab (SHP643) in Japanese Subjects with Hereditary Angioedema

    Summary
    EudraCT number
    		 2022-002621-98
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    26 Aug 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Sep 2022
    First version publication date
    07 Sep 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    SHP643-302
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04180163
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Shire
    Sponsor organisation address
    300 Shire Way, Lexington, United States, MA 02421
    Public contact
    Study Director, Shire, ClinicalTransparency@shire.com
    Scientific contact
    Study Director, Shire, ClinicalTransparency@shire.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    26 Aug 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Aug 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this study was to evaluate the safety and efficacy of lanadelumab in Japanese participants with HAE Type I or II.
    Protection of trial subjects
    All study participants were required to read and sign an Informed Consent Form.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    12 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
    Japan: 12
    Worldwide total number of subjects
    12
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    11
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 12 participants took part in the study at 10 investigative sites in Japan from 12 December 2019 to 26 August 2021. Participants who rolled over to TAK-743-5007 (NCT04687137) had a 2-week follow-up while others had 4 weeks of follow-up.

    Pre-assignment
    Screening details
    		 Subjects were observed in 4-week Baseline Run-in that could be extended upto 8 weeks.Subjects experiencing ≥1.0 angioedema attacks per 4 weeks during Run-in Period,who remained eligible per inclusion criteria entered 52-week lanadelumab Treatment Period(TP)[TP:A + TP:B],followed by upto 4-week Safety Follow-up.Lanadelumab was given only during TPs.

    Period 1
    Period 1 title
    Run-in Period (4 or up to 8 Weeks)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Lanadelumab 300 mg q2w or q4w
    Arm description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lanadelumab
    Investigational medicinal product code
    Other name
    DX-2930, TAK-743, SHP643
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Lanadelumab solution, SC

    Number of subjects in period 1
    		Lanadelumab 300 mg q2w or q4w
    Started
    12
    Completed
    12
    Period 2
    Period 2 title
    Treatment Period A (Weeks 1 to 26)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Lanadelumab 300 mg q2w or q4w
    Arm description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lanadelumab
    Investigational medicinal product code
    Other name
    DX-2930, TAK-743, SHP643
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Lanadelumab solution, SC

    Number of subjects in period 2
    		Lanadelumab 300 mg q2w or q4w
    Started
    12
    Completed
    12
    Period 3
    Period 3 title
    Treatment Period B (Weeks 27 to 52)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Lanadelumab 300 mg q2w or q4w
    Arm description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lanadelumab
    Investigational medicinal product code
    Other name
    DX-2930, TAK-743, SHP643
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Lanadelumab solution, SC

    Number of subjects in period 3
    		Lanadelumab 300 mg q2w or q4w
    Started
    12
    Completed
    12
    Period 4
    Period 4 title
    Safety Follow-up Period (Weeks 53 to 56)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Lanadelumab 300 mg q2w or q4w
    Arm description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Lanadelumab
    Investigational medicinal product code
    Other name
    DX-2930, TAK-743, SHP643
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Lanadelumab solution, SC

    Number of subjects in period 4
    		Lanadelumab 300 mg q2w or q4w
    Started
    12
    Completed
    12

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Lanadelumab 300 mg q2w or q4w
    Reporting group description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.

    Reporting group values
    		 Lanadelumab 300 mg q2w or q4w Total
    Number of subjects
    		 12
    Age Categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 41.9 ( 12.36 ) -
    Gender categorical
    Units: Subjects
        Male
    		 3 3
        Female
    		 9 9
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 12 12
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 0 0
        White
    		 0 0
        More than one race
    		 0 0
        Unknown or Not Reported
    		 0 0
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 12 12
        Unknown or Not Reported
    		 0 0
    Weight
    Units: kg
        arithmetic mean (standard deviation)
    		 61.24 ( 10.346 ) -
    Height
    Units: cm
        arithmetic mean (standard deviation)
    		 161.08 ( 9.379 ) -
    Body Mass Index (BMI)
    BMI= weight(kg) / height(meter)^2
    Units: kg/m^2
        arithmetic mean (standard deviation)
    		 23.80 ( 5.065 ) -

    End points

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    End points reporting groups
    Reporting group title
    Lanadelumab 300 mg q2w or q4w
    Reporting group description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.
    Reporting group title
    Lanadelumab 300 mg q2w or q4w
    Reporting group description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.
    Reporting group title
    Lanadelumab 300 mg q2w or q4w
    Reporting group description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.
    Reporting group title
    Lanadelumab 300 mg q2w or q4w
    Reporting group description
    		 Lanadelumab 300 mg solution, subcutaneously (SC), once every 2 weeks (q2w) for 26 weeks in Treatment Period A. This was followed by Treatment Period B (additional 26 weeks, total of 52 weeks including Treatment Period A) during which participants remained on Treatment Period A regimen or received 300 mg lanadelumab solution once every 4 weeks (q4w) for 26 weeks if well-controlled (attack-free) for 26 consecutive weeks with lanadelumab treatment. The dose frequency change was based on the Investigator’s discretion and approval by the Sponsor’s Medical Monitor.

    Subject analysis set title
    Treatment Period A: Non-HAE
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Lanadelumab 300 mg solution, SC, q2w for 26 weeks in Treatment Period A. Non-HAE attack (subset identified in case report form [CRF] as not reported HAE attack) subset participants were included in this arm.

    Subject analysis set title
    Treatment Period A: HAE
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Lanadelumab 300 mg solution, SC, q2w for 26 weeks in Treatment Period A. HAE attack (subset of AEs identified in CRF as reported HAE attack) subset participants were included in this arm.

    Subject analysis set title
    Treatment Period B: Non-HAE
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Lanadelumab 300 mg solution, SC, q2w for 26 weeks or lanadelumab 300 mg solution, SC, q4w for 26 weeks in Treatment Period B if well tolerated (attack-free) for 26 consecutive weeks with lanadelumab treatment during Treatment Period A. Non-HAE attack (subset identified in CRF as not reported HAE attack) subset participants were included in this arm.

    Subject analysis set title
    Treatment Period B: HAE
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Lanadelumab 300 mg solution, SC, q2w for 26 weeks or lanadelumab 300 mg solution, SC, q4w for 26 weeks in Treatment Period B if well tolerated (attack-free) for 26 consecutive weeks with lanadelumab treatment during Treatment Period A. HAE attack (subset of AEs identified in CRF as reported HAE attack) subset participants were included in this arm.

    Subject analysis set title
    Safety Follow-up Period: Non-HAE
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants who completed the lanadelumab 300 mg regimen in Treatment Period B returned on Day 378 (for participants who chose to roll over into study TAK-743-5007 [NCT04687137]) or Day 392 as follow-up visit for final assessment in the Safety Follow-up Period. Non-HAE attack (subset identified in CRF as not reported HAE attack) subset participants were included in this arm.

    Subject analysis set title
    Safety Follow-up Period: HAE
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants who completed the lanadelumab 300 mg regimen in Treatment Period B returned on Day 378 (for participants who chose to roll over into study TAK-743-5007) or Day 392 as follow-up visit for final assessment in the Safety Follow-up Period. HAE attack (subset of AEs identified in CRF as reported HAE attack) subset participants were included in this arm.

    Primary: Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Period of Day 0 Through Day 182

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    End point title
    		 Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Period of Day 0 Through Day 182 [1]
    End point description
    A participant was considered as attack free during an efficacy evaluation period if the participant had no investigator-confirmed hereditary angioedema (HAE) attacks during that efficacy evaluation period. A HAE attack was defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Number of participants achieving attack-free status for the efficacy evaluation period of Day 0 through Day 182 were assessed. Full Analysis Set (FAS) included all participants who received at least 1 dose of investigational medicinal product (IMP).
    End point type
    Primary
    End point timeframe
    Day 0 through Day 182
    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: Only descriptive statistics was planned to be reported for this endpoint.
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: participants
    5
    No statistical analyses for this end point

    Secondary: Number of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods

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    End point title
    		 Number of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods
    End point description
    Efficacy evaluation period consisted of four periods: Day 0 (after study drug administration) through Day 182 (the end of Treatment Period A), Day 0 (after study drug administration) through Day 364 (the end of Treatment Period B), presumed steady-state period from Day 70 through Day 182, presumed steady-state period from Day 70 through Day 364. Number of investigator-confirmed HAE attacks during each of the efficacy evaluation periods were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182, Day 0 through Day 364, Day 70 through Day 182, Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: HAE attacks
        Day 0 Through Day 182
    92
        Day 0 Through Day 364
    189
        Day 70 Through Day 182
    55
        Day 70 Through Day 364
    152
    No statistical analyses for this end point

    Secondary: Number of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks Requiring Acute Treatment During Each of the Efficacy Evaluation Periods

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    End point title
    		 Number of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks Requiring Acute Treatment During Each of the Efficacy Evaluation Periods
    End point description
    Efficacy evaluation period consisted of four periods: Day 0 (after study drug administration) through Day 182 (the end of Treatment Period A), Day 0 (after study drug administration) through Day 364 (the end of Treatment Period B), presumed steady-state period from Day 70 through Day 182, presumed steady-state period from Day 70 through Day 364. Number of investigator-confirmed HAE attacks requiring acute treatment during each of the efficacy evaluation periods were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182, Day 0 through Day 364, Day 70 through Day 182, Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: HAE attacks
        Day 0 Through Day 182
    79
        Day 0 Through Day 364
    168
        Day 70 Through Day 182
    46
        Day 70 Through Day 364
    135
    No statistical analyses for this end point

    Secondary: Number of Moderate or Severe Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods

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    End point title
    		 Number of Moderate or Severe Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods
    End point description
    Severe attack was defined as Grade 3 (some assistance usually required, medical intervention/therapy required, hospitalizations possible), moderate attack was defined as Grade 2 (some assistance may be needed, no or minimal medical intervention/therapy required). Number of investigator-confirmed moderate or severe HAE attacks during the each of efficacy evaluation periods was assessed. Efficacy evaluation period consisted of four periods: Day 0 (after study drug administration) through Day 182 (the end of Treatment Period A), Day 0 (after study drug administration) through Day 364 (the end of Treatment Period B), presumed steady-state period from Day 70 through Day 182, presumed steady-state period from Day 70 through Day 364. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182, Day 0 through Day 364, Day 70 through Day 182, Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: HAE attacks
        Day 0 Through Day 182
    72
        Day 0 Through Day 364
    153
        Day 70 Through Day 182
    41
        Day 70 Through Day 364
    122
    No statistical analyses for this end point

    Secondary: Number of Participants with Maximum Hereditary Angioedema (HAE) Attack Severity During Each of the Efficacy Evaluation Periods

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    End point title
    		 Number of Participants with Maximum Hereditary Angioedema (HAE) Attack Severity During Each of the Efficacy Evaluation Periods
    End point description
    Efficacy evaluation period consisted of four periods: Day 0 (after study drug administration) through Day 182 (the end of Treatment Period A), Day 0 (after study drug administration) through Day 364 (the end of Treatment Period B), presumed steady-state period from Day 70 through Day 182, presumed steady-state period from Day 70 through Day 364. Number of participants with maximum HAE attack severity during each of the efficacy evaluation periods was assessed. HAE attack severity was calculated per participant based on the severity categories as follows: No attack, Mild, Moderate, and Severe. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182, Day 0 through Day 364, Day 70 through Day 182, Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: participants
        Day 0 Through Day 182: No Attack
    5
        Day 0 Through Day 182: Mild
    2
        Day 0 Through Day 182: Moderate
    4
        Day 0 Through Day 182: Severe
    1
        Day 0 Through Day 364: No Attack
    2
        Day 0 Through Day 364: Mild
    1
        Day 0 Through Day 364: Moderate
    8
        Day 0 Through Day 364: Severe
    1
        Day 70 Through Day 182: No Attack
    5
        Day 70 Through Day 182: Mild
    4
        Day 70 Through Day 182: Moderate
    2
        Day 70 Through Day 182: Severe
    1
        Day 70 Through Day 364: No Attack
    2
        Day 70 Through Day 364: Mild
    2
        Day 70 Through Day 364: Moderate
    7
        Day 70 Through Day 364: Severe
    1
    No statistical analyses for this end point

    Secondary: Number of High-Morbidity Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods

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    End point title
    		 Number of High-Morbidity Investigator-Confirmed Hereditary Angioedema (HAE) Attacks During Each of the Efficacy Evaluation Periods
    End point description
    A high morbidity HAE attack was defined as any attack that has at least 1 of the following characteristics: severe, results in hospitalization (except hospitalization for observation <24 hours), hemodynamically significant (systolic blood pressure <90, requires intravenous (IV) hydration, or associated with syncope or near syncope) or laryngeal. Number of high-morbidity investigator-confirmed HAE attacks during each of the 4 efficacy evaluation periods (Treatment Period A, Overall Treatment Period, Presumed Steady-state Period for Treatment Period A and Overall Presumed Steady-state Period) were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182, Day 0 through Day 364, Day 70 through Day 182, Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: HAE attacks
        Day 0 Through Day 182
    5
        Day 0 Through Day 364
    11
        Day 70 Through Day 182
    4
        Day 70 Through Day 364
    10
    No statistical analyses for this end point

    Secondary: Time to First Hereditary Angioedema (HAE) Attack After Day 0 for the Efficacy Evaluation Period

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    End point title
    		 Time to First Hereditary Angioedema (HAE) Attack After Day 0 for the Efficacy Evaluation Period
    End point description
    The time to the first HAE attack (days) after Day 0 for the efficacy evaluation period of Day 0 through Day 182 was calculated from the date and time of the first dose of lanadelumab for the efficacy evaluation period (Day 0 through Day 182) to the date and time of the first in HAE attack after the first open-label dose for the efficacy evaluation period of Day 0 through Day 182. Kaplan-Meier Method was used for analysis and the Kaplan Meier estimate expressed as time (in days) to first HAE attack After Day 0 for Treatment Period A (Day 0 through Day 182) is presented. FAS included all participants who received at least 1 dose of IMP. 99999= The upper limit of 95% confidence interval (CI) was not evaluable due to low number of participants with events.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: days
        median (confidence interval 95%)
    97.2 (4.2 to 99999)
    No statistical analyses for this end point

    Secondary: Time to First Hereditary Angioedema (HAE) Attack After Day 0 for the Efficacy Evaluation Period

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    End point title
    		 Time to First Hereditary Angioedema (HAE) Attack After Day 0 for the Efficacy Evaluation Period
    End point description
    The time to the first HAE attack (days) after Day 0 for the efficacy evaluation period of Day 70 through Day 182 was calculated from the date and time of the first dose of lanadelumab for the efficacy evaluation period (Day 70 through Day 182) to the date and time of the first in HAE attack after the first open-label dose for the efficacy evaluation period of Day 70 through Day 182. Kaplan-Meier Method was used for analysis and the Kaplan Meier estimate expressed as time (in days) to first HAE attack After Day 0 for presumed steady-state period for Treatment Period A (Day 70 through Day 182) is presented. FAS included all participants who received at least 1 dose of IMP. 99999= The upper limit of 95% CI was not evaluable due to low number of participants with events.
    End point type
    Secondary
    End point timeframe
    Day 70 through Day 182
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: days
        median (confidence interval 95%)
    91.0 (4.6 to 99999)
    No statistical analyses for this end point

    Secondary: Number of Participants Achieving at Least 50%, 70% and 90% Reduction in the Investigator-Confirmed Normalized Number of Attacks (NNA) per 4 Weeks Relative to the Run-in Period NNA for Each of Efficacy Evaluation Periods

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    End point title
    		 Number of Participants Achieving at Least 50%, 70% and 90% Reduction in the Investigator-Confirmed Normalized Number of Attacks (NNA) per 4 Weeks Relative to the Run-in Period NNA for Each of Efficacy Evaluation Periods
    End point description
    Run- in Period was 4 weeks and may have been extended up to 8 weeks to determine participants’ Baseline attack rate.period attack rate, multiplied by 100.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182, Day 0 through Day 364, Day 70 through Day 182, Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: participants
        Day 0 Through Day 182: ≥50% Reduction
    10
        Day 0 Through Day 182: ≥70% Reduction
    8
        Day 0 Through Day 182: ≥90% Reduction
    6
        Day 0 Through Day 364: ≥50% Reduction
    10
        Day 0 Through Day 364: ≥70% Reduction
    8
        Day 0 Through Day 364: ≥90% Reduction
    6
        Day 70 Through Day 182: ≥50% Reduction
    11
        Day 70 Through Day 182: ≥70% Reduction
    10
        Day 70 Through Day 182: ≥90% Reduction
    6
        Day 70 Through Day 364: ≥50% Reduction
    10
        Day 70 Through Day 364: ≥70% Reduction
    8
        Day 70 Through Day 364: ≥90% Reduction
    7
    No statistical analyses for this end point

    Secondary: Number of Participants Achieving Normalized Number of Attacks (NNA) <1.0 per 4 Weeks, <0.75 per 4 Weeks, <0.50 per 4 Weeks and <0.25 per 4 Weeks for Each of the Efficacy Evaluation Periods

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    End point title
    		 Number of Participants Achieving Normalized Number of Attacks (NNA) <1.0 per 4 Weeks, <0.75 per 4 Weeks, <0.50 per 4 Weeks and <0.25 per 4 Weeks for Each of the Efficacy Evaluation Periods
    End point description
    The NNA (investigator-confirmed) during each efficacy evaluation period was expressed as a monthly (28 days) HAE attack rate. Number of participants achieving NNA <1.0 per 4 weeks, <0.75 per 4 weeks, <0.50 per 4 weeks, and <0.25 per 4 weeks for each of the 4 efficacy evaluation periods (Treatment Period A, Overall Treatment Period, Presumed Steady-state Period for Treatment Period A, and Overall Presumed Steady-state Period) were assessed. The responder categories were not mutually exclusive, participants may appear in more than one category as applicable based on their HAE attack rate. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182, Day 0 through Day 364, Day 70 through Day 182, Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: participants
        Day 0 Through Day 182: <1.0 per Month
    9
        Day 0 Through Day 182: <0.75 per Month
    9
        Day 0 Through Day 182: <0.50 per Month
    6
        Day 0 Through Day 182: <0.25 per Month
    6
        Day 0 Through Day 364: <1.0 per Month
    9
        Day 0 Through Day 364: <0.75 per Month
    8
        Day 0 Through Day 364: <0.50 per Month
    7
        Day 0 Through Day 364: <0.25 per Month
    6
        Day 70 Through Day 182: <1.0 per Month
    9
        Day 70 Through Day 182: <0.75 per Month
    9
        Day 70 Through Day 182: <0.50 per Month
    8
        Day 70 Through Day 182: <0.25 per Month
    6
        Day 70 Through Day 364: <1.0 per Month
    9
        Day 70 Through Day 364: <0.75 per Month
    8
        Day 70 Through Day 364: <0.50 per Month
    7
        Day 70 Through Day 364: <0.25 per Month
    6
    No statistical analyses for this end point

    Secondary: Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Period Day 0 Through Day 364, Day 70 Through Day 182, and Day 70 Through Day 364

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    End point title
    		 Number of Participants Achieving Attack-Free Status for the Efficacy Evaluation Period Day 0 Through Day 364, Day 70 Through Day 182, and Day 70 Through Day 364
    End point description
    A participant was considered as attack free during an efficacy evaluation period if the participant had no investigator-confirmed HAE attacks during that efficacy evaluation period. A HAE attack was defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Number of participants achieving attack-free status for the 4 efficacy evaluation periods (Overall Treatment Period, Presumed Steady-state Period for Treatment Period A, and Overall Presumed Steady-state Period) were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 364, Day 70 through Day 182, and Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    9
    Units: participants
        Day 0 Through Day 364
    2
        Day 70 Through Day 182
    5
        Day 70 Through Day 364
    2
    No statistical analyses for this end point

    Secondary: Number of Participants Achieving Attack-Free Status for Monthly Increments

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    End point title
    		 Number of Participants Achieving Attack-Free Status for Monthly Increments
    End point description
    A participant was considered as attack free during an efficacy evaluation period if the participant had no investigator-confirmed HAE attacks during that efficacy evaluation period. A HAE attack was defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    At Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 and 13
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: participants
        Month 1
    7
        Month 2
    8
        Month 3
    9
        Month 4
    7
        Month 5
    9
        Month 6
    7
        Month 7
    8
        Month 8
    6
        Month 9
    7
        Month 10
    9
        Month 11
    8
        Month 12
    7
        Month 13
    8
    No statistical analyses for this end point

    Secondary: Percentage of Attack Free Days During Each of the Efficacy Evaluation Periods

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    End point title
    		 Percentage of Attack Free Days During Each of the Efficacy Evaluation Periods
    End point description
    An attack-free day was defined as a calendar day with no investigator-confirmed HAE attack. HAE attack free days were calculated by counting the number of days in the efficacy evaluation period without an HAE attack and dividing by the number of days the participant contributed to the efficacy evaluation period. Percentage of investigator-confirmed HAE attack free days during each of the efficacy evaluation periods (Treatment Period A, Overall Treatment Period, Presumed Steady-state Period for Treatment Period A, and Overall Presumed Steady-state Period) were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182, Day 0 through Day 364, Day 70 through Day 182, Day 70 through Day 364
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: percentage of attack-free days
    arithmetic mean (standard deviation)
        Day 0 Through Day 182
    88.53 ( 27.146 )
        Day 0 Through Day 364
    89.04 ( 27.015 )
        Day 70 Through Day 182
    90.34 ( 21.952 )
        Day 70 Through Day 364
    89.85 ( 25.015 )
    No statistical analyses for this end point

    Secondary: Number of Participants Achieving Investigator-Confirmed Hereditary Angioedema (HAE) Attack-Free Intervals

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    End point title
    		 Number of Participants Achieving Investigator-Confirmed Hereditary Angioedema (HAE) Attack-Free Intervals
    End point description
    A participant was considered as attack free during a time period if the participant had no investigator-confirmed HAE attacks during that time period. Participants who discontinued during a time period were considered as non-responders for that time period. Number of participants achieving investigator-confirmed HAE attack free intervals from Day 0 through Day 182 were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Day 0 through Day 182
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: participants
    5
    No statistical analyses for this end point

    Secondary: Number of Participants with Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs)

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    End point title
    		 Number of Participants with Treatment Emergent Adverse Events (TEAEs) Including Adverse Events of Special Interest (AESI) and Serious Adverse Events (SAEs)
    End point description
    TEAE=any event emerging at or after initiation of treatment with investigational product (IP) or any existing event that worsens in intensity/frequency on exposure to IP. Serious TEAE=any untoward clinical manifestation of signs, symptoms, outcomes (related to IP or not) at any dose: results in death, was life-threatening, requires inpatient/prolongation of hospitalization, resulted in persistent/significant disability/incapacity, congenital abnormality/birth defect, important medical event. AESI=investigator-reported hypersensitivity reactions, events of disordered coagulation as bleeding/hypercoagulable AESI. Adverse events (AEs) were classified as HAE attack and non-HAE attack reported AEs and are categorized accordingly. As Pre-specified in the protocol, TEAEs, SAEs and AESIs were collected per Period i.e., Treatment Period A, B and Safety follow-up Period and data is reported accordingly. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    From first dose of the study drug up to end of study (EOS) (up to Day 392)
    End point values
    		 Treatment Period A: Non-HAE Treatment Period A: HAE Treatment Period B: Non-HAE Treatment Period B: HAE Safety Follow-up Period: Non-HAE Safety Follow-up Period: HAE
    Number of subjects analysed
    12
    12
    12
    12
    12
    12
    Units: participants
        Any TEAEs
    10
    8
    9
    8
    0
    3
        AESI
    3
    0
    0
    0
    0
    0
        Any Serious TEAEs
    1
    1
    1
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Plasma Concentrations of Lanadelumab

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    End point title
    		 Plasma Concentrations of Lanadelumab
    End point description
    Pharmacokinetic (PK) Set included all participants in the FAS who had at least 1 evaluable post dose PK concentration value. n= number analysed indicates the number of participants with data available for analysis at a specific time point.
    End point type
    Secondary
    End point timeframe
    Predose on Days 0, 56, 98, 140, 182, 266, 350, 364, and at any time on Day 378 or 392
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: nanograms per milliliter (ng/mL)
    arithmetic mean (standard deviation)
        Day 0 (n=12)
    25.584 ( 50.2034 )
        Day 56 (n=12)
    23630.973 ( 8665.1099 )
        Day 98 (n=12)
    24142.776 ( 9575.7596 )
        Day 140 (n=11)
    24613.885 ( 9319.6672 )
        Day 182 (n=12)
    23679.526 ( 6793.3003 )
        Day 266 (n=12)
    19269.249 ( 8870.0355 )
        Day 350 (n=3)
    13225.533 ( 2538.8239 )
        Day 364 (n=10)
    22329.820 ( 7527.7165 )
        Day 378/392 (n=12)
    19308.033 ( 7708.1882 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire Total Score

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    End point title
    		 Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire Total Score
    End point description
    The AE-QoL questionnaire is a self-administered validated instrument to assess health related (HR)QoL among participants with recurrent angioedema (including HAE). The AE-QoL consists of 17 disease-specific quality-of-life items, to produce a total AE-QoL score and 4 domain scores (functioning, fatigue/mood, fear/shame, and nutrition) and each of the 17 items had a five-point response scale ranging from 1 (Never) to 5 (Very Often). The questionnaire was scored according to the developers' guidelines to produce 4 domain scores (functioning, fatigue/mood, fear/shame, nutrition) yielding a total score. The raw total score (mean of all item scores) was rescaled using linear transformations into final percentage scores ranging 0 to 100, based on the maximum possible score, where higher the score, greater the QoL impairment. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    Days 0, 28, 56, 98, 126, 154, 182, 266, 364, 378 or 392
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: score on a scale
    arithmetic mean (standard deviation)
        Day 0
    46.57 ( 20.885 )
        Day 28
    26.35 ( 31.129 )
        Day 56
    28.43 ( 28.255 )
        Day 98
    26.96 ( 30.926 )
        Day 126
    32.60 ( 33.999 )
        Day 154
    21.69 ( 28.750 )
        Day 182
    26.10 ( 33.978 )
        Day 266
    26.96 ( 29.951 )
        Day 364
    26.59 ( 34.428 )
        Day 378/392
    28.55 ( 31.934 )
    No statistical analyses for this end point

    Secondary: Plasma Kallikrein (pKal) Activity

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    End point title
    		 Plasma Kallikrein (pKal) Activity
    End point description
    pKal activity was measured by biomarker cleaved high molecular weight kininogen (cHMWK) level to assess pharmacodynamics (PD) of lanadelumab. Pharmacodynamic (PD) Set included all participants in the FAS who had at least 1 evaluable post dose PD value. n= number analysed indicates the number of participants with data available for analysis at the given time point.
    End point type
    Secondary
    End point timeframe
    Predose on Days 0, 56, 98, 140, 182, 266, 350, 364, and at any time on Day 378 or 392
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: percentage of cHMWK
    arithmetic mean (standard deviation)
        Day 0 (n=12)
    63.04 ( 22.096 )
        Day 56 (n=12)
    30.88 ( 17.090 )
        Day 98 (n=12)
    32.40 ( 18.829 )
        Day 140 (n=11)
    35.67 ( 13.787 )
        Day 182 (n=12)
    30.31 ( 17.344 )
        Day 266 (n=12)
    21.69 ( 11.221 )
        Day 350 (n=3)
    39.83 ( 12.407 )
        Day 364 (n=10)
    27.51 ( 12.901 )
        Day 378/392 (n=12)
    40.65 ( 15.828 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Positive Anti-drug Antibody (ADA) in Plasma

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    End point title
    		 Number of Participants With Positive Anti-drug Antibody (ADA) in Plasma
    End point description
    Baseline was defined as the last non-missing value prior to first dose of study drug (based on date or date/time). FAS included all participants who received at least 1 dose of IMP. n= number analysed indicates the number of participants with data available for analysis at the given time point.
    End point type
    Secondary
    End point timeframe
    Predose on Days 0 (or Baseline), 56, 98, 140, 182, 266, 350, 364, and at any time on Day 378 or 392
    End point values
    		 Lanadelumab 300 mg q2w or q4w
    Number of subjects analysed
    12
    Units: participants
        Day 0 [or Baseline] (n=12)
    0
        Day 56 (n=12)
    0
        Day 98 (n=12)
    0
        Day 140 (n=11)
    0
        Day 182 (n=12)
    0
        Day 266 (n=12)
    0
        Day 350 (n=3)
    0
        Day 364 (n=10)
    0
        Day 378/392 (n=12)
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With TEAEs Related to Clinical Laboratory Tests

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    End point title
    		 Number of Participants With TEAEs Related to Clinical Laboratory Tests
    End point description
    A TEAE was defined as any event emerging or manifesting at or after the initiation of treatment with an IP or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the IP or medicinal product. As Pre-specified in the protocol, the TEAEs were collected per Period i.e., Treatment Period A, B and Safety follow-up Period. The data is reported as per HAE attack and non-HAE attack per Period. Number of participants with TEAEs related to clinical laboratory tests (hematology, clinical chemistry, coagulation, and urinalysis) were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    From first dose of the study drug up to end of study (EOS) (up to Day 392)
    End point values
    		 Treatment Period A: Non-HAE Treatment Period A: HAE Treatment Period B: Non-HAE Treatment Period B: HAE Safety Follow-up Period: Non-HAE Safety Follow-up Period: HAE
    Number of subjects analysed
    12
    12
    12
    12
    12
    12
    Units: participants
    0
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With TEAEs Related to Vital Signs

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    End point title
    		 Number of Participants With TEAEs Related to Vital Signs
    End point description
    A TEAE was defined as any event emerging or manifesting at or after the initiation of treatment with an IP or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the IP or medicinal product. As Pre-specified in the protocol, the TEAEs were collected per Period i.e., Treatment Period A, B and Safety follow-up Period. The data is reported as per HAE attack and non-HAE attack per Period. Number of participants with TEAEs related to vital signs (blood pressure (BP), heart rate (HR), body temperature, and respiratory rate) were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    From first dose of the study drug up to end of study (EOS) (up to Day 392)
    End point values
    		 Treatment Period A: Non-HAE Treatment Period A: HAE Treatment Period B: Non-HAE Treatment Period B: HAE Safety Follow-up Period: Non-HAE Safety Follow-up Period: HAE
    Number of subjects analysed
    12
    12
    12
    12
    12
    12
    Units: participants
    0
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With TEAEs Related to Electrocardiogram (ECG)

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    End point title
    		 Number of Participants With TEAEs Related to Electrocardiogram (ECG)
    End point description
    A TEAE was defined as any event emerging or manifesting at or after the initiation of treatment with an IP or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the IP or medicinal product. As Pre-specified in the protocol, the TEAEs were collected per Period i.e., Treatment Period A, B and Safety follow-up Period. The data is reported as per HAE attack and non-HAE attack per Period. Number of participants with TEAEs related to 12 lead-ECG were assessed. FAS included all participants who received at least 1 dose of IMP.
    End point type
    Secondary
    End point timeframe
    From first dose of the study drug up to end of study (EOS) (up to Day 392)
    End point values
    		 Treatment Period A: Non-HAE Treatment Period A: HAE Treatment Period B: Non-HAE Treatment Period B: HAE Safety Follow-up Period: Non-HAE Safety Follow-up Period: HAE
    Number of subjects analysed
    12
    12
    12
    12
    12
    12
    Units: participants
    0
    0
    0
    0
    0
    0
    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 the study drug up to end of study (EOS) (up to Day 392)
    Adverse event reporting additional description
    FAS included all participants who received at least 1 dose of IMP. As Pre-specified in the protocol, the TEAEs were collected per Period i.e., Treatment Period A, B and Safety follow-up Period. The data is reported as per HAE attack and non-HAE attack per Period.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Treatment Period A: Non-HAE
    Reporting group description
    		 Lanadelumab 300 mg solution, SC, q2w for 26 weeks in Treatment Period A. Non-HAE attack (subset identified in case report form [CRF] as not reported HAE attack) subset participants were included in this arm.

    Reporting group title
    Treatment Period A: HAE
    Reporting group description
    		 Lanadelumab 300 mg solution, SC, q2w for 26 weeks in Treatment Period A. HAE attack (subset of AEs identified in CRF as reported HAE attack) subset participants were included in this arm.

    Reporting group title
    Treatment Period B: Non-HAE
    Reporting group description
    		 Lanadelumab 300 mg solution, SC, q2w for 26 weeks or lanadelumab 300 mg solution, SC, q4w for 26 weeks in Treatment Period B if well tolerated (attack-free) for 26 consecutive weeks with lanadelumab treatment during Treatment Period A. Non-HAE attack (subset identified in CRF as not reported HAE attack) subset participants were included in this arm.

    Reporting group title
    Treatment Period B: HAE
    Reporting group description
    		 Lanadelumab 300 mg solution, SC, q2w for 26 weeks or lanadelumab 300 mg solution, SC, q4w for 26 weeks in Treatment Period B if well tolerated (attack-free) for 26 consecutive weeks with lanadelumab treatment during Treatment Period A. HAE attack (subset of AEs identified in CRF as reported HAE attack) subset participants were included in this arm.

    Reporting group title
    Safety Follow-up Period: Non-HAE
    Reporting group description
    		 Participants who completed the lanadelumab 300 mg regimen in Treatment Period B returned on Day 378 (for participants who chose to roll over into study TAK-743-5007 [NCT04687137]) or Day 392 as follow-up visit for final assessment in the Safety Follow-up Period. Non-HAE attack (subset identified in CRF as not reported HAE attack) subset participants were included in this arm.

    Reporting group title
    Safety Follow-up Period: HAE
    Reporting group description
    		 Participants who completed the lanadelumab 300 mg regimen in Treatment Period B returned on Day 378 (for participants who chose to roll over into study TAK-743-5007) or Day 392 as follow-up visit for final assessment in the Safety Follow-up Period. HAE attack (subset of AEs identified in CRF as reported HAE attack) subset participants were included in this arm.

    Serious adverse events
    		 Treatment Period A: Non-HAE Treatment Period A: HAE Treatment Period B: Non-HAE Treatment Period B: HAE Safety Follow-up Period: Non-HAE Safety Follow-up Period: HAE
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Congenital, familial and genetic disorders
    Hereditary angioedema
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Anxiety disorder
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Device related infection
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Treatment Period A: Non-HAE Treatment Period A: HAE Treatment Period B: Non-HAE Treatment Period B: HAE Safety Follow-up Period: Non-HAE Safety Follow-up Period: HAE
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 12 (83.33%)
    7 / 12 (58.33%)
    9 / 12 (75.00%)
    8 / 12 (66.67%)
    0 / 12 (0.00%)
    3 / 12 (25.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Uterine leiomyoma
    Additional description: Number of participants at risk in each arm is based on the female population in this study.
         subjects affected / exposed [1]
    0 / 9 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    General disorders and administration site conditions
    Injection site reaction
         subjects affected / exposed
    6 / 12 (50.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    37
    0
    0
    0
    0
    0
    Injection site erythema
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    5
    0
    0
    0
    Injection site pruritus
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Pyrexia
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    1
    0
    0
    0
    Chest pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Infusion site pruritus
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Injection site pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Injection site swelling
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Malaise
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Immune system disorders
    Allergy to chemicals
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Anaphylactic shock
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Contrast media allergy
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Drug hypersensitivity
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Reproductive system and breast disorders
    Dysmenorrhoea
    Additional description: Number of participants at risk in each arm is based on the female population in this study.
         subjects affected / exposed [2]
    0 / 9 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Psychiatric disorders
    Anxiety disorder
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Fall
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Heat illness
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Procedural pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Skin abrasion
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Thermal burn
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    Tooth fracture
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Congenital, familial and genetic disorders
    Hereditary angioedema
         subjects affected / exposed
    0 / 12 (0.00%)
    7 / 12 (58.33%)
    0 / 12 (0.00%)
    8 / 12 (66.67%)
    0 / 12 (0.00%)
    3 / 12 (25.00%)
         occurrences all number
    0
    92
    0
    97
    0
    11
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    12
    0
    9
    0
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    8
    0
    5
    0
    0
    0
    Hypoaesthesia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    Intercostal neuralgia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    1
    0
    0
    0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    2
    0
    0
    0
    Abdominal pain lower
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Abdominal pain upper
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Constipation
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Dental caries
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Diarrhoea
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Gingival bleeding
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Hyperaesthesia teeth
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Large intestine polyp
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Nausea
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    10
    0
    10
    0
    0
    0
    Toothache
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Alopecia areata
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Angioedema
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Dermatitis contact
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Eczema
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Eczema asteatotic
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Pruritus
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Urticaria
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Back pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Neck pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Infections and infestations
    Cystitis
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    0
    0
    0
    0
    Furuncle
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Gingivitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Laryngopharyngitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    2
    0
    0
    0
    Oral herpes
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Vulvitis
    Additional description: Number of participants at risk in each arm is based on the female population in this study.
         subjects affected / exposed [3]
    0 / 9 (0.00%)
    0 / 9 (0.00%)
    1 / 9 (11.11%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Dehydration
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Diabetes mellitus
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: Number of subjects exposed to this adverse event is based on the female population in this study.
    [2] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: Number of subjects exposed to this adverse event is based on the female population in this study.
    [3] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: Number of subjects exposed to this adverse event is based on the female population in this study.

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    29 Jul 2019
    The changes implemented based on Amendment 1 were: -Added the requirement to exclude participants with a known hypersensitivity to the IMP or its components as a new exclusion criterion. -Corrected text regarding the number of days for reporting non-serious AEs that are reported as HAE attacks for consistency.
    05 Oct 2020
    The changes implemented based on Amendment 2 were: -Revised Sponsor approval -Added that participants may elect to roll over into an expanded access study (Study TAK-743-5007). -Revised interim analysis to include 2 interim analyses: first analysis was to be performed when the first 6 participants enrolled in the study had reached Day 182 or discontinued in Treatment Period A (26 weeks of treatment) and second analysis was to be performed when the first 4 participants enrolled in the study had reached Day 364 or discontinued. -Corrected error that screening visit was also Visit 1. -Corrected error on collection of prior treatment to reflect that prior treatments should be collected prior to screening visit. -Corrected investigator-confirmed HAE attacks to state: 3 or more than 3 investigator-confirmed HAE attacks. -Removed body weight from physical exams at Visit 26 and Visit 27. -Removed pregnancy tests at Visit 26 and Visit 27. -Removed following sentence: Overall attack rates will be estimated using a Poisson general linear model adjusting for run-in period attack rate and accounting for potential overdispersion. -Added 'if applicable' to analysis of efficacy endpoints at 4 efficacy evaluation periods. -Other efficacy endpoints along with statistical methods for their analysis were added. -Added language that subgroup analyses may be performed. -Corrected carbon dioxide to bicarbonate.

    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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