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   43865   clinical trials with a EudraCT protocol, of which   7286   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 2 Open-label Study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

    Summary
    EudraCT number
    		 2016-003526-16
    Trial protocol
    		 GB   IT  
    Global end of trial date
    05 Jan 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    14 Dec 2023
    First version publication date
    14 Dec 2023
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    ACH471-101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03472885
    WHO universal trial number (UTN)
    		 U1111-1209-4655
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals, Inc.
    Sponsor organisation address
    121 Seaport Blvd, Boston, MA, United States, 02210
    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
    05 Jan 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Jan 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia, as measured by increased blood hemoglobin, when given with eculizumab (a drug commonly used for treatment of PNH) for 24 weeks in participants with PNH.
    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
    08 May 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Efficacy, Safety
    Long term follow-up duration
    		 3 Years
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 6
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    United Kingdom: 4
    Worldwide total number of subjects
    12
    EEA total number of subjects
    2
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    10
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study included a 24-Week Treatment Period and Long-term Extension (LTE) Period.

    Period 1
    Period 1 title
    24-Week Treatment Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Danicopan + Eculizumab
    Arm description
    		 Participants were administered 100, 150, or 200 milligrams (mg) danicopan three times daily (TID) in combination with eculizumab for 24 weeks. Danicopan dose may have been increased within each participant, to a maximum of 200 mg TID based on safety and efficacy. After completing the 24-Week Treatment Period, participants who received clinical benefit (as assessed by the Investigator based on improvement in hemoglobin) continued into the LTE and received the same dose of danicopan plus eculizumab treatment as that received at the end of the 24-Week Treatment Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Soliris
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eculizumab was administered per schedule specified in the arm description.

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    ACH-0144471
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Danicopan was administered per schedule specified in the arm description.

    Number of subjects in period 1
    		Danicopan + Eculizumab
    Started
    12
    Safety Population
    12
    Efficacy Population
    11
    Completed
    11
    Not completed
    1
         Adverse event, non-fatal
    1
    Period 2
    Period 2 title
    LTE Period (Maximum Exposure: 1463 Days)
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Danicopan + Eculizumab
    Arm description
    		 Participants were administered 100, 150, or 200 milligrams (mg) danicopan three times daily (TID) in combination with eculizumab for 24 weeks. Danicopan dose may have been increased within each participant, to a maximum of 200 mg TID based on safety and efficacy. After completing the 24-Week Treatment Period, participants who received clinical benefit (as assessed by the Investigator based on improvement in hemoglobin) continued into the LTE and received the same dose of danicopan plus eculizumab treatment as that received at the end of the 24-Week Treatment Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Soliris
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eculizumab was administered per schedule specified in the arm description.

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    ACH-0144471
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Danicopan was administered per schedule specified in the arm description.

    Number of subjects in period 2
    		Danicopan + Eculizumab
    Started
    11
    Completed
    11

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Danicopan + Eculizumab
    Reporting group description
    		 Participants were administered 100, 150, or 200 milligrams (mg) danicopan three times daily (TID) in combination with eculizumab for 24 weeks. Danicopan dose may have been increased within each participant, to a maximum of 200 mg TID based on safety and efficacy. After completing the 24-Week Treatment Period, participants who received clinical benefit (as assessed by the Investigator based on improvement in hemoglobin) continued into the LTE and received the same dose of danicopan plus eculizumab treatment as that received at the end of the 24-Week Treatment Period.

    Reporting group values
    		 Danicopan + Eculizumab Total
    Number of subjects
    		 12 12
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 10 10
        From 65-84 years
    		 2 2
        85 years and over
    		 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 45.59 ( 16.385 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 10 10
        Male
    		 2 2
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 12 12
        Unknown or Not Reported
    		 0 0
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 7 7
        Asian
    		 1 1
        Black or African American
    		 3 3
        Other
    		 1 1

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Danicopan + Eculizumab
    Reporting group description
    		 Participants were administered 100, 150, or 200 milligrams (mg) danicopan three times daily (TID) in combination with eculizumab for 24 weeks. Danicopan dose may have been increased within each participant, to a maximum of 200 mg TID based on safety and efficacy. After completing the 24-Week Treatment Period, participants who received clinical benefit (as assessed by the Investigator based on improvement in hemoglobin) continued into the LTE and received the same dose of danicopan plus eculizumab treatment as that received at the end of the 24-Week Treatment Period.
    Reporting group title
    Danicopan + Eculizumab
    Reporting group description
    		 Participants were administered 100, 150, or 200 milligrams (mg) danicopan three times daily (TID) in combination with eculizumab for 24 weeks. Danicopan dose may have been increased within each participant, to a maximum of 200 mg TID based on safety and efficacy. After completing the 24-Week Treatment Period, participants who received clinical benefit (as assessed by the Investigator based on improvement in hemoglobin) continued into the LTE and received the same dose of danicopan plus eculizumab treatment as that received at the end of the 24-Week Treatment Period.

    Subject analysis set title
    24-Week Treatment Period: Danicopan + Eculizumab
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Participants were administered 100, 150, or 200 mg danicopan TID in combination with eculizumab for 24 weeks. Danicopan dose may have been increased within each participant, to a maximum of 200 mg TID based on safety and efficacy.

    Subject analysis set title
    LTE Period: Danicopan + Eculizumab
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    After completing the 24-Week Treatment Period, participants who received clinical benefit (as assessed by the Investigator based on improvement in hemoglobin) continued into the LTE and received the same dose of danicopan plus eculizumab treatment as that received at the end of the 24-Week Treatment Period.

    Primary: Change from Baseline In Hemoglobin At Week 24

    		 Close Top of page
    End point title
    		 Change from Baseline In Hemoglobin At Week 24 [1]
    End point description
    Efficacy Population: All treated participants who received at least 4 weeks of danicopan.
    End point type
    Primary
    End point timeframe
    Baseline, Week 24
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Statistical analysis was not planned for this endpoint.
    End point values
    		 Danicopan + Eculizumab
    Number of subjects analysed
    11
    Units: g/dL
    arithmetic mean (standard deviation)
        Baseline
    7.94 ( 1.425 )
        Week 24
    10.33 ( 1.661 )
        Change from Baseline
    2.39 ( 1.333 )
    No statistical analyses for this end point

    Secondary: Change From Baseline In Lactate Dehydrogenase At Week 24

    		 Close Top of page
    End point title
    		 Change From Baseline In Lactate Dehydrogenase At Week 24
    End point description
    Efficacy Population: All treated participants who received at least 4 weeks of danicopan.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Danicopan + Eculizumab
    Number of subjects analysed
    11
    Units: IU/L
    arithmetic mean (standard deviation)
        Baseline
    244.5 ( 74.40 )
        Week 24
    239.5 ( 48.48 )
        Change from Baseline
    -5.0 ( 48.60 )
    No statistical analyses for this end point

    Secondary: Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment

    		 Close Top of page
    End point title
    		 Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment
    End point description
    Efficacy Population: All treated participants who received at least 4 weeks of danicopan.
    End point type
    Secondary
    End point timeframe
    Within 24 weeks prior to first dose and during 24-week treatment period
    End point values
    		 Danicopan + Eculizumab
    Number of subjects analysed
    11
    Units: participants
        Within 24 Weeks Prior to First Dose
    1
        During 24-Week Treatment Period
    10
    No statistical analyses for this end point

    Secondary: Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment

    		 Close Top of page
    End point title
    		 Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment
    End point description
    Efficacy Population: All treated participants who received at least 4 weeks of danicopan.
    End point type
    Secondary
    End point timeframe
    Within 24 weeks prior to first dose and during 24-week treatment period
    End point values
    		 Danicopan + Eculizumab
    Number of subjects analysed
    11
    Units: RBC units
    arithmetic mean (standard deviation)
        Within 24 Weeks Prior to First Dose
    4.5 ( 3.96 )
        During 24-Week Treatment Period
    0.2 ( 0.60 )
    No statistical analyses for this end point

    Secondary: Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug

    		 Close Top of page
    End point title
    		 Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug
    End point description
    An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Reported Adverse Events module. Participants who received at least 1 dose of danicopan were included in the safety assessment.
    End point type
    Secondary
    End point timeframe
    Day 1 (after dosing) through end of study (maximum exposure: 1631 days)
    End point values
    		 24-Week Treatment Period: Danicopan + Eculizumab LTE Period: Danicopan + Eculizumab
    Number of subjects analysed
    12
    11
    Units: participants
        SAEs
    2
    7
        Grade 3 AEs
    4
    6
        Grade 4 AEs
    1
    1
        AEs Leading to Discontinuation of Study Drug
    1
    0
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Day 1 (after dosing) through end of study (maximum exposure: 1631 days)
    Adverse event reporting additional description
    Participants who received at least 1 dose of danicopan were included in the safety assessment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    LTE Period: Danicopan + Eculizumab
    Reporting group description
    		 After completing the 24-Week Treatment Period, participants who received clinical benefit (as assessed by the Investigator based on improvement in hemoglobin) continued into the LTE and received the same dose of danicopan plus eculizumab treatment as that received at the end of the 24-Week Treatment Period.

    Reporting group title
    24-Week Treatment Period: Danicopan + Eculizumab
    Reporting group description
    		 Participants were administered 100, 150, or 200 mg danicopan TID in combination with eculizumab for 24 weeks. Danicopan dose may have been increased within each participant, to a maximum of 200 mg TID based on safety and efficacy.

    Serious adverse events
    		 LTE Period: Danicopan + Eculizumab 24-Week Treatment Period: Danicopan + Eculizumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    7 / 11 (63.64%)
    2 / 12 (16.67%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Schwannoma
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Febrile neutropenia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Haemolysis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Pancreatitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary oedema
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Haemoglobinuria
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Device related infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyelonephritis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tracheobronchitis viral
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 LTE Period: Danicopan + Eculizumab 24-Week Treatment Period: Danicopan + Eculizumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    11 / 11 (100.00%)
    10 / 12 (83.33%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Melanocytic naevus
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Thirst
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Feeling abnormal
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Fatigue
         subjects affected / exposed
    3 / 11 (27.27%)
    2 / 12 (16.67%)
         occurrences all number
    4
    2
    Pyrexia
         subjects affected / exposed
    4 / 11 (36.36%)
    1 / 12 (8.33%)
         occurrences all number
    5
    1
    Vaccination site pain
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Pain
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Chest discomfort
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Chills
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 12 (16.67%)
         occurrences all number
    1
    2
    Influenza like illness
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Immune system disorders
    Seasonal allergy
    alternative dictionary used: MedDRA 18.1
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Immunisation reaction
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Menorrhagia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Dysphonia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Nasal congestion
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Oropharyngeal pain
    alternative dictionary used: MedDRA 18.1
         subjects affected / exposed
    2 / 11 (18.18%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Cough
    alternative dictionary used: MedDRA 18.1
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Dyspnoea
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Depression
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Insomnia
         subjects affected / exposed
    2 / 11 (18.18%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Investigations
    Blood bilirubin increased
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    2
    2
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 12 (16.67%)
         occurrences all number
    1
    2
    Fibrin D dimer increased
    alternative dictionary used: MedDRA 18.1
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    SARS-CoV-2 test positive
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Blood creatinine increased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Blood alkaline phosphatase increased
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Contusion
    alternative dictionary used: MedDRA 18.1
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Vaccination complication
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Transfusion-associated dyspnoea
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Transfusion reaction
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Febrile nonhaemolytic transfusion reaction
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Accidental overdose
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Cardiac disorders
    Sinus tachycardia
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Palpitations
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    5 / 11 (45.45%)
    3 / 12 (25.00%)
         occurrences all number
    9
    8
    Dizziness
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Anosmia
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Ageusia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Lethargy
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Memory impairment
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Restless legs syndrome
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Sciatica
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Taste disorder
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Disturbance in attention
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Blood and lymphatic system disorders
    Anaemia
    alternative dictionary used: MedDRA 18.1
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Neutropenia
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 12 (16.67%)
         occurrences all number
    1
    3
    Thrombocytopenia
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    4
    0
    Haemolysis
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Eustachian tube dysfunction
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Tinnitus
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Vertigo
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Periorbital swelling
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Tongue ulceration
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Nausea
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Lip swelling
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Diarrhoea
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Abdominal pain
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 12 (16.67%)
         occurrences all number
    2
    3
    Abdominal discomfort
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Abdominal pain upper
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    1
    2
    Dysphagia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Gingival discomfort
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Mouth ulceration
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Oral pain
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Periodontal disease
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Saliva altered
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Vomiting
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Hepatobiliary disorders
    Hyperbilirubinaemia
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    1
    2
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Dry skin
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Dermal cyst
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Alopecia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Hyperhidrosis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Haemoglobinuria
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    2 / 11 (18.18%)
    2 / 12 (16.67%)
         occurrences all number
    4
    2
    Neck pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    2
    Musculoskeletal pain
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 12 (16.67%)
         occurrences all number
    1
    2
    Arthralgia
    alternative dictionary used: MedDRA 18.1
         subjects affected / exposed
    2 / 11 (18.18%)
    2 / 12 (16.67%)
         occurrences all number
    3
    2
    Limb discomfort
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    3
    0
    Back pain
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Flank pain
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Muscle spasms
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Myalgia
         subjects affected / exposed
    2 / 11 (18.18%)
    1 / 12 (8.33%)
         occurrences all number
    2
    1
    Infections and infestations
    COVID-19
         subjects affected / exposed
    6 / 11 (54.55%)
    0 / 12 (0.00%)
         occurrences all number
    6
    0
    Upper respiratory tract infection
         subjects affected / exposed
    5 / 11 (45.45%)
    5 / 12 (41.67%)
         occurrences all number
    7
    5
    Nasopharyngitis
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Conjunctivitis
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Sinusitis
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Cellulitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Hordeolum
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Oral herpes
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Pharyngitis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Vulvovaginal candidiasis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Metabolic acidosis
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Hypocalcaemia
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Dec 2017
    This amendment: - Updated the contraception section to include definitions requested by Health Authorities. - Updated the contact information for SAE reporting.
    17 Jan 2018
    This amendment provided wording permitting the conducting of patient reported outcomes interviews as questionnaires where required.
    13 Mar 2018
    This amendment: - Specified that vaccination against bacterial infections should be performed, when necessary based on vaccination history, according to national and/or local guidelines. - Updated and clarified requirements for “acceptable” and “highly effective” methods of contraception. - Reduced the time window for definition of a missed dose from 6 hours to 4 hours.
    15 Feb 2019
    - Amended the exclusion criteria to allow enrollment of participants with a history of hematopoietic stem cell transplant (HSCT) if HSCT engraftment has failed. - Amended the exclusion criteria to allow enrollment of participants with direct bilirubin > 1.5 × upper limit of normal if the elevated bilirubin is due to extravascular hemolysis, in the opinion of the investigator. - Permitted participants to switch from eculizumab to an approved eculizumab biosimilar or ravulizumab after completion of the primary endpoint at Week 24. - Expanded potential enrolment to a maximum of 14 participants.
    19 Feb 2019
    - Shortened the time (from 12 to 8 weeks) for participants to receive a stable dose of eculizumab prior to study entry.
    04 Jun 2021
    This amendment: - Updated the dose taper instructions and schedule to remove the 75 mg dose since the 75 mg tablets used in this study would expire and no longer be manufactured. - Added an option for participants to enter another appropriate Alexion clinical study, if available. - Added guidance on dose tapering for participants who enter other clinical studies. - Removed PK and PD samples at the Long term Extension Period clinic visits, Taper Visits, and Follow up Visits. - Added guidance for collection of a predose PK sample for participants who had dose escalation after Week 12. - Updated the language for use of prophylactic antibiotics to recommend use as per local requirements, if applicable. - Added language regarding COVID 19 risk assessment and COVID 19 vaccination risk assessment as per MHRA requirements.

    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
    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-Sun Apr 28 00:04:03 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA