Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS)

    Summary
    EudraCT number
    		 2019-004619-30
    Trial protocol
    		 IE   SE   GB   DE   DK   ES   NL   BE   FR   IT  
    Global end of trial date
    17 Oct 2021

    Results information
    Results version number
    		 v2(current)
    This version publication date
    14 May 2023
    First version publication date
    11 Oct 2022
    Other versions
    		 v1
    Version creation reason

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    ALXN1210-ALS-308
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04248465
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals Inc.
    Sponsor organisation address
    100 College Street, New Haven, CT, United States, 06510
    Public contact
    Alexion Europe SAS European Clinical Trial Information, Alexion Pharmaceuticals Inc., +33 787148158, clinicaltrials.eu@alexion.com
    Scientific contact
    Alexion Europe SAS European Clinical Trial Information, Alexion Pharmaceuticals Inc., +33 787148158, 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
    18 Feb 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    17 Oct 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Oct 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the effect of ravulizumab compared with placebo on amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R) score in adult participants with amyotrophic lateral sclerosis (ALS)
    Protection of trial subjects
    This study was conducted in accordance with International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Mar 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 7
    Country: Number of subjects enrolled
    Canada: 33
    Country: Number of subjects enrolled
    Belgium: 3
    Country: Number of subjects enrolled
    Denmark: 22
    Country: Number of subjects enrolled
    France: 36
    Country: Number of subjects enrolled
    Germany: 14
    Country: Number of subjects enrolled
    Ireland: 1
    Country: Number of subjects enrolled
    Italy: 51
    Country: Number of subjects enrolled
    Netherlands: 12
    Country: Number of subjects enrolled
    Poland: 17
    Country: Number of subjects enrolled
    Spain: 48
    Country: Number of subjects enrolled
    Sweden: 17
    Country: Number of subjects enrolled
    Israel: 11
    Country: Number of subjects enrolled
    Japan: 26
    Country: Number of subjects enrolled
    Switzerland: 10
    Country: Number of subjects enrolled
    United Kingdom: 4
    Country: Number of subjects enrolled
    United States: 70
    Worldwide total number of subjects
    382
    EEA total number of subjects
    221
    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)
    267
    From 65 to 84 years
    114
    85 years and over
    1

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study was conducted from 30 Mar 2020 to 17 Oct 2021.

    Period 1
    Period 1 title
    Randomized-Controlled Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Ravulizumab/Ravulizumab
    Arm description
    		 Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then once every 8 weeks (q8w) up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 milligrams (mg) dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    ALXN1210
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received ALXN1210 at prespecified dose and timepoints.

    Arm title
    		 Placebo/Ravulizumab
    Arm description
    		 Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received placebo matched to ravulizumab at prespecified dose and timepoints.

    Number of subjects in period 1
    		Ravulizumab/Ravulizumab Placebo/Ravulizumab
    Started
    255
    127
    Received at least 1 dose of study drug
    255
    127
    Completed
    15
    5
    Not completed
    240
    122
         Adverse event, serious fatal
    12
    5
         Consent withdrawn by subject
    30
    17
         Physician decision
    1
    1
         Adverse event, non-fatal
    2
    -
         Study Terminated by Sponsor
    194
    99
         Lost to follow-up
    1
    -
    Period 2
    Period 2 title
    Open-label Extension Period
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Ravulizumab/Ravulizumab
    Arm description
    		 Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then once every 8 weeks (q8w) up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 milligrams (mg) dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    ALXN1210
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received ALXN1210 at prespecified dose and timepoints.

    Arm title
    		 Placebo/Ravulizumab
    Arm description
    		 Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    ALXN1210
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received ALXN1210 at prespecified dose and timepoints.

    Number of subjects in period 2 [1]
    		Ravulizumab/Ravulizumab Placebo/Ravulizumab
    Started
    14
    5
    Received at Least 1 Dose of Study Drug
    14
    5
    Completed
    0
    0
    Not completed
    14
    5
         Consent withdrawn by subject
    -
    1
         Study Terminated by Sponsor
    14
    4
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: 1 Subject discontinued the study from Ravulizumab arm prior to entering the Open Label Extension Period.

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Ravulizumab/Ravulizumab
    Reporting group description
    		 Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then once every 8 weeks (q8w) up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 milligrams (mg) dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Reporting group title
    Placebo/Ravulizumab
    Reporting group description
    		 Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Reporting group values
    		 Ravulizumab/Ravulizumab Placebo/Ravulizumab Total
    Number of subjects
    		 255 127 382
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 58.6 ( 10.57 ) 58.0 ( 11.03 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 94 58 152
        Male
    		 161 69 230
    Subject analysis sets

    Subject analysis set title
    Ravulizumab
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Subject analysis sets values
    		 Ravulizumab Placebo
    Number of subjects
    255
    127
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    58.6 ( 10.57 )
    58.0 ( 11.03 )
    Sex: Female, Male
    Units: participants
        Female
    94
    58
        Male
    161
    69

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Ravulizumab/Ravulizumab
    Reporting group description
    		 Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then once every 8 weeks (q8w) up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 milligrams (mg) dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Reporting group title
    Placebo/Ravulizumab
    Reporting group description
    		 Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.
    Reporting group title
    Ravulizumab/Ravulizumab
    Reporting group description
    		 Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then once every 8 weeks (q8w) up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 milligrams (mg) dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Reporting group title
    Placebo/Ravulizumab
    Reporting group description
    		 Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Subject analysis set title
    Ravulizumab
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then, during the Open Label Extension Period, participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive) during the Randomized Controlled Period. Then during the Open Label Extension Period, participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Primary: Change From Baseline In Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score

    		 Close Top of page
    End point title
    		 Change From Baseline In Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score [1]
    End point description
    The ALSFRS-Revised is a validated instrument for evaluating the levels of the functional status of participants with amyotrophic lateral sclerosis (ALS) in 4 areas, including bulbar, gross motor activity, fine motor activity, and respiratory functions. The scale included 12 functional items and each item is rated on a 0 to 4 scale, with a maximum total score of 48. A higher score indicated greater retention of function. Baseline was defined as last non-missing value on or before first study drug administration. Full analysis set (FAS) included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.
    End point type
    Primary
    End point timeframe
    Baseline, Week 50
    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 analysis was planned to be reported for this endpoint.
    End point values
    		 Ravulizumab Placebo
    Number of subjects analysed
    32
    14
    Units: units on a scale
        arithmetic mean (standard deviation)
    -11.9 ( 7.30 )
    -10.6 ( 6.05 )
    No statistical analyses for this end point

    Secondary: Time To Ventilator Assistance-free Survival

    		 Close Top of page
    End point title
    		 Time To Ventilator Assistance-free Survival
    End point description
    Ventilation Assistance-Free Survival (VAFS) is a composite endpoint of survival and severe and irreversible respiratory decline. The use of VAFS allowed for the collection of survival data that was not impacted by survival prolongation from noninvasive or permanent ventilatory interventions which could prolong life without impacting underlying disease progression. FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group.
    End point type
    Secondary
    End point timeframe
    Up to Week 50
    End point values
    		 Ravulizumab Placebo
    Number of subjects analysed
    255
    127
    Units: months
        median (full range (min-max))
    6.05 (0.79 to 11.10)
    7.69 (4.83 to 9.53)
    No statistical analyses for this end point

    Secondary: Change From Baseline In Percent Predicted Slow Vital Capacity

    		 Close Top of page
    End point title
    		 Change From Baseline In Percent Predicted Slow Vital Capacity
    End point description
    Slow vital capacity measures slow and gradual expulsion of air from the lungs using a spirometer. FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 50
    End point values
    		 Ravulizumab Placebo
    Number of subjects analysed
    19
    9
    Units: percentage of predicted volume
        arithmetic mean (standard deviation)
    -20.9 ( 19.74 )
    -21.3 ( 13.90 )
    No statistical analyses for this end point

    Secondary: Number of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events, and TEAEs Leading To Study Drug Discontinuation

    		 Close Top of page
    End point title
    		 Number of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events, and TEAEs Leading To Study Drug Discontinuation
    End point description
    An adverse event (AE) was defined as any unfavorable and unintended sign (for example, including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product or procedure, whether or not considered related to the medicinal product or procedure, which occurred during the course of the clinical study. TEAEs were defined as AEs that occurred on or after the date and time of study drug administration, or those that first occurred before dosing but worsened in frequency or severity after study drug administration. A summary of all Serious Adverse Events 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 study drug grouped by treatment actually received (for reporting exposure and safety data).
    End point type
    Secondary
    End point timeframe
    Baseline up to Week 156
    End point values
    		 Ravulizumab Placebo
    Number of subjects analysed
    255
    127
    Units: participants
        TEAEs
    204
    108
        Treatment Emergent Serious AEs
    41
    24
        TEAE Leading to Study Drug Discontinuation
    2
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline In Muscle Strength As Assessed By Handheld Dynamometry

    		 Close Top of page
    End point title
    		 Change From Baseline In Muscle Strength As Assessed By Handheld Dynamometry
    End point description
    Handheld dynamometry (HHD) is a procedure for quantitative strength testing. Muscle strength testing was performed on prespecified muscles in the upper and lower extremities bilaterally and the force measurements were recorded. Force of measurement is reported in megascores (lower, upper, total). The total megascore is defined as the average of the non-missing ratios over baseline for all the muscles involved. The megascore at baseline is always 100. The range of a potential megascore can not be determined in advance. A megascore >100 indicates more strength compared to baseline. FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 50
    End point values
    		 Ravulizumab Placebo
    Number of subjects analysed
    26
    13
    Units: % (as the unit of megascore)
        arithmetic mean (standard deviation)
    -46.5 ( 27.57 )
    -53.4 ( 20.28 )
    No statistical analyses for this end point

    Secondary: Change From Baseline In Serum Neurofilament Light Chain

    		 Close Top of page
    End point title
    		 Change From Baseline In Serum Neurofilament Light Chain
    End point description
    FAS included all randomized participants who received at least 1 dose of study drug grouped by randomized treatment group. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 50
    End point values
    		 Ravulizumab Placebo
    Number of subjects analysed
    14
    7
    Units: units on a scale
        arithmetic mean (standard deviation)
    91.5 ( 40.40 )
    73.1 ( 27.82 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Serum Ravulizumab Concentration Over the Study Duration

    		 Close Top of page
    End point title
    		 Change From Baseline in Serum Ravulizumab Concentration Over the Study Duration
    End point description
    Pharmacokinetic Analysis Set (PKAS) included all participants who received at least 1 dose of the study drug and had at least 1 postdose pharmacokinetic (PK) sample. This endpoint was planned to be reported for Ravulizumab arm only.
    End point type
    Secondary
    End point timeframe
    Baseline, Predose at Week 50
    End point values
    		 Ravulizumab
    Number of subjects analysed
    14
    Units: micrograms per milliliter
        geometric mean (geometric coefficient of variation)
    634 ( 29.1 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Serum Free Complement Component 5 (C5) Concentration Over the Study Duration

    		 Close Top of page
    End point title
    		 Change From Baseline in Serum Free Complement Component 5 (C5) Concentration Over the Study Duration
    End point description
    Pharmacodynamic analysis set (PDAS) included all participants who received at least 1 dose of the study drug and had at least 1 postdose pharmacodynamics (PD) sample. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure. There were no participants with evaluable C5 data in the Placebo arm at Week 50.
    End point type
    Secondary
    End point timeframe
    Baseline, Predose at Week 50
    End point values
    		 Ravulizumab Placebo
    Number of subjects analysed
    13
    0 [2]
    Units: micrograms/milliliter
        arithmetic mean (standard deviation)
    -155.2 ( 24.42 )
    ( )
    Notes
    [2] - As per the planned analysis, number of participants in this group were 0 for this endpoint.
    No statistical analyses for this end point

    Secondary: Number of Participants With Positive Antidrug Antibodies (ADAs) to ALXN1210

    		 Close Top of page
    End point title
    		 Number of Participants With Positive Antidrug Antibodies (ADAs) to ALXN1210
    End point description
    Blood samples were collected to evaluate antibody response through development of ADAs. PDAS included all participants who received at least 1 dose of the study drug and had at least 1 postdose PD sample. Here, Number of Participants analyzed signifies those participants who were evaluable at Week 50. There were no participants with evaluable C5 data in the Placebo arm at Week 50.
    End point type
    Secondary
    End point timeframe
    Week 50
    End point values
    		 Ravulizumab Placebo
    Number of subjects analysed
    23
    0 [3]
    Units: participants
    0
    Notes
    [3] - As per the planned analysis, number of participants in this group were 0 for this endpoint.
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Week 156
    Adverse event reporting additional description
    Safety Set included all participants who received at least 1 dose of study drug grouped by treatment actually received (for reporting exposure and safety data). "All-Cause Mortality" reports all deaths that occurred during the study, including the deaths that led to Study Discontinuation.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    Randomized Controlled Period: Ravulizumab
    Reporting group description
    		 Participants received a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive).

    Reporting group title
    Open Label Extension Period: Placebo
    Reporting group description
    		 Participants received ravulizumab, with a blinded loading dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Reporting group title
    Open Label Extension Period: Ravulizumab
    Reporting group description
    		 Participants received ravulizumab, with a blinded 900 mg dose at Week 50, followed by an open-label ravulizumab maintenance dose at Week 52, then q8w for up to 106 weeks of treatment.

    Reporting group title
    Randomized Controlled Period: Placebo
    Reporting group description
    		 Participants received a weight-based loading dose of placebo matched to ravulizumab on Day 1, followed by a weight-based maintenance dose on Day 15, then q8w up to Week 42 (inclusive).

    Serious adverse events
    		 Randomized Controlled Period: Ravulizumab Open Label Extension Period: Placebo Open Label Extension Period: Ravulizumab Randomized Controlled Period: Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    41 / 255 (16.08%)
    0 / 5 (0.00%)
    2 / 14 (14.29%)
    24 / 127 (18.90%)
         number of deaths (all causes)
    15
    0
    0
    6
         number of deaths resulting from adverse events
    Surgical and medical procedures
    Euthanasia
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    General disorders and administration site conditions
    Death
         subjects affected / exposed
    2 / 255 (0.78%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
    Complication associated with device
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Infusion related hypersensitivity reaction
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Social circumstances
    Feeding tube user
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Aspiration
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory failure
         subjects affected / exposed
    8 / 255 (3.14%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    4 / 127 (3.15%)
         occurrences causally related to treatment / all
    0 / 8
    0 / 0
    0 / 0
    0 / 4
         deaths causally related to treatment / all
    0 / 5
    0 / 0
    0 / 0
    0 / 2
    Respiratory distress
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Atelectasis
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonitis aspiration
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    4 / 127 (3.15%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    1 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    Respiratory disorder
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Dyspnoea
         subjects affected / exposed
    3 / 255 (1.18%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    2 / 127 (1.57%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Mental status changes
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Assisted suicide
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Completed suicide
         subjects affected / exposed
    2 / 255 (0.78%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
    Investigations
    SARS-CoV-2 test positive
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Myocardial necrosis marker increased
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Weight decreased
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Femur fracture
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Femoral neck fracture
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrostomy failure
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infusion related reaction
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin laceration
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Rib fracture
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hip fracture
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrostomy tube site complication
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Cardio-respiratory arrest
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Syncope
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Haemorrhage intracranial
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Transient ischaemic attack
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Subarachnoid haemorrhage
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Headache
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Amyotrophic lateral sclerosis
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Abdominal pain
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Colitis ulcerative
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Constipation
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pancreatitis relapsing
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Dysphagia
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    2 / 127 (1.57%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    1 / 14 (7.14%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cholelithiasis
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    3 / 255 (1.18%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    5 / 127 (3.94%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
    0 / 0
    2 / 6
         deaths causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 1
    COVID-19 pneumonia
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    COVID-19
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    1 / 14 (7.14%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Appendicitis
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    1 / 127 (0.79%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pyelonephritis
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia bacterial
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Bronchitis
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia aspiration
         subjects affected / exposed
    2 / 255 (0.78%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    2 / 127 (1.57%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Malnutrition
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Euglycaemic diabetic ketoacidosis
         subjects affected / exposed
    1 / 255 (0.39%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Randomized Controlled Period: Ravulizumab Open Label Extension Period: Placebo Open Label Extension Period: Ravulizumab Randomized Controlled Period: Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    196 / 255 (76.86%)
    1 / 5 (20.00%)
    3 / 14 (21.43%)
    106 / 127 (83.46%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    54 / 255 (21.18%)
    1 / 5 (20.00%)
    1 / 14 (7.14%)
    36 / 127 (28.35%)
         occurrences all number
    81
    1
    1
    65
    Ankle fracture
         subjects affected / exposed
    0 / 255 (0.00%)
    1 / 5 (20.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Contusion
         subjects affected / exposed
    11 / 255 (4.31%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    8 / 127 (6.30%)
         occurrences all number
    13
    0
    0
    13
    Nervous system disorders
    Headache
         subjects affected / exposed
    42 / 255 (16.47%)
    0 / 5 (0.00%)
    1 / 14 (7.14%)
    22 / 127 (17.32%)
         occurrences all number
    62
    0
    1
    29
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    22 / 255 (8.63%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    11 / 127 (8.66%)
         occurrences all number
    40
    0
    0
    11
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    26 / 255 (10.20%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    5 / 127 (3.94%)
         occurrences all number
    29
    0
    0
    5
    Nausea
         subjects affected / exposed
    23 / 255 (9.02%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    10 / 127 (7.87%)
         occurrences all number
    28
    0
    0
    10
    Diarrhoea
         subjects affected / exposed
    4 / 255 (1.57%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    9 / 127 (7.09%)
         occurrences all number
    6
    0
    0
    10
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    1 / 14 (7.14%)
    0 / 127 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    0 / 255 (0.00%)
    0 / 5 (0.00%)
    1 / 14 (7.14%)
    0 / 127 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    13 / 255 (5.10%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    8 / 127 (6.30%)
         occurrences all number
    14
    0
    0
    8
    Musculoskeletal and connective tissue disorders
    Back Pain
         subjects affected / exposed
    21 / 255 (8.24%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    5 / 127 (3.94%)
         occurrences all number
    31
    0
    0
    7
    Arthralgia
         subjects affected / exposed
    18 / 255 (7.06%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    4 / 127 (3.15%)
         occurrences all number
    23
    0
    0
    5
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    12 / 255 (4.71%)
    0 / 5 (0.00%)
    0 / 14 (0.00%)
    11 / 127 (8.66%)
         occurrences all number
    12
    0
    0
    12
    Hordeolum
         subjects affected / exposed
    0 / 255 (0.00%)
    1 / 5 (20.00%)
    0 / 14 (0.00%)
    0 / 127 (0.00%)
         occurrences all number
    0
    1
    0
    0

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Oct 2020
    The main purpose of this amendment is to update study procedures: ventilator utilization at all visits, timing of PK and ADA sampling, allowance of home or alternative healthcare facility visits and home SVC assessment, and deletion of selected Short-Form Health Survey (SF-36) assessments, deoxyribonucleic acid/ribonucleic acid sample collections, and HHD assessments. Updates also include addition of a second methodology for the primary analysis.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    The IDMC recommended the study be discontinued due to lack of efficacy with ravulizumab.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri May 02 07:55:31 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA