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   43839   clinical trials with a EudraCT protocol, of which   7280   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 2a Proof-of-Mechanism, Open-Label Study to Determine the Effect of ACH-0144471 on C3 Levels in Participants With Low C3 Levels Due to Either C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

    Summary
    EudraCT number
    2016-003525-42
    Trial protocol
    BE   NL  
    Global end of trial date
    09 Jan 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    07 Jul 2021
    First version publication date
    07 Jul 2021
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    ACH471-201
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03124368
    WHO universal trial number (UTN)
    U1111-1203-9016
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals, Inc
    Sponsor organisation address
    121 Seaport Boulevard, Boston, MA, United States, 02210
    Public contact
    European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +33 147100606, clinicaltrials.eu@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Pharmaceuticals, Inc., +33 147100606, clinicaltrials.eu@alexion.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    09 Jan 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    21 Dec 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    09 Jan 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to determine whether ACH-0144471 (danicopan) increases blood C3 complement protein (C3) levels in participants with low C3 levels due to either C3G or IC-MPGN.
    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
    11 Jul 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Australia: 4
    Worldwide total number of subjects
    6
    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)
    6
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    Participants were recruited from 5 study centers total in Australia, Belgium, and The Netherlands. Only 3 study centers, 1 in each country, treated participants.

    Period 1
    Period 1 title
    Overall (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Group 1: Danicopan 100 mg TID (Sentinel)
    Arm description
    Participants received 100 milligrams (mg) of danicopan three times daily (TID) during the Treatment Period.
    Arm type
    Experimental

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    Other name
    ACH-0144471, ACH-4471, ACH4471, 4471, ALXN2040
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Participants received study drug for 14 days (Treatment Period), followed by a taper over the next 7 days (Taper Period).

    Arm title
    Group 2: Danicopan up to 200 mg TID
    Arm description
    Participants received not more than 200 mg of danicopan TID during the Treatment Period.
    Arm type
    Experimental

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    Other name
    ACH-0144471, ACH-4471, ACH4471, 4471, ALXN2040
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Participants received study drug for 14 days (Treatment Period), followed by a taper over the next 7 days (Taper Period).

    Number of subjects in period 1
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID
    Started
    2
    4
    Received At Least 1 Dose Of Study Drug
    2
    4
    Completed
    2
    4

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Group 1: Danicopan 100 mg TID (Sentinel)
    Reporting group description
    Participants received 100 milligrams (mg) of danicopan three times daily (TID) during the Treatment Period.

    Reporting group title
    Group 2: Danicopan up to 200 mg TID
    Reporting group description
    Participants received not more than 200 mg of danicopan TID during the Treatment Period.

    Reporting group values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects
    2 4 6
    Age categorical
    Units: Subjects
        In utero
    0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0
        Newborns (0-27 days)
    0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0
        Children (2-11 years)
    0 0 0
        Adolescents (12-17 years)
    0 0 0
        Adults (18-64 years)
    2 4 6
        From 65-84 years
    0 0 0
        85 years and over
    0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    25.50 ± 7.85 30.00 ± 12.68 -
    Gender categorical
    Units: Subjects
        Female
    0 1 1
        Male
    2 3 5
    Race
    Units: Subjects
        American Indian or Alaska Native
    0 0 0
        Asian
    2 1 3
        Native Hawaiian or Other Pacific Islander
    0 0 0
        Black or African American
    0 0 0
        White
    0 3 3
        More than one race
    0 0 0
        Unknown or Not Reported
    0 0 0

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Group 1: Danicopan 100 mg TID (Sentinel)
    Reporting group description
    Participants received 100 milligrams (mg) of danicopan three times daily (TID) during the Treatment Period.

    Reporting group title
    Group 2: Danicopan up to 200 mg TID
    Reporting group description
    Participants received not more than 200 mg of danicopan TID during the Treatment Period.

    Subject analysis set title
    Total
    Subject analysis set type
    Full analysis
    Subject analysis set description
    All participants who received at least 1 dose of ALXN2040.

    Subject analysis set title
    Group 1: Danicopan 100 mg TID
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Participants received 100 mg of danicopan TID during the Treatment Period.

    Subject analysis set title
    Group 2: Danicopan 150 mg TID
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Participants received 150 mg of danicopan TID during the Treatment Period.

    Subject analysis set title
    Group 2: Danicopan 200 mg TID
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Participants received 200 mg of danicopan TID during the Treatment Period.

    Primary: Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15

    Close Top of page
    End point title
    Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 [1]
    End point description
    Serum C3 levels were measured by conventional Roche immunoturbidimetric assay method. Change from Baseline = Serum C3 levels on Day 15 - Baseline Serum C3 levels.
    End point type
    Primary
    End point timeframe
    Baseline, Day 15
    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: Quantitative statistical analysis was not performed for this end point, per protocol. Descriptive statistics are included (mean and standard deviation).
    End point values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects analysed
    2
    4
    6
    Units: g/L
    arithmetic mean (standard deviation)
        Baseline
    0.32 ± 0.060
    0.56 ± 0.230
    0.48 ± 0.220
        Day 15
    0.35 ± 0.060
    0.70 ± 0.350
    0.58 ± 0.330
        Change from Baseline
    0.03 ± 0.000
    0.14 ± 0.140
    0.11 ± 0.120
    No statistical analyses for this end point

    Primary: Change From Baseline In Plasma Intact C3 Level On Day 15

    Close Top of page
    End point title
    Change From Baseline In Plasma Intact C3 Level On Day 15 [2]
    End point description
    Plasma Intact C3 level were measured by a novel multiplex assay method. Change from Baseline = Plasma Intact C3 levels on Day 15 - Baseline Plasma Intact C3 levels.
    End point type
    Primary
    End point timeframe
    Baseline, Day 15
    Notes
    [2] - 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: Quantitative statistical analysis was not performed for this end point, per protocol. Descriptive statistics are included (mean and standard deviation).
    End point values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects analysed
    2
    4
    6
    Units: μg/mL
    arithmetic mean (standard deviation)
        Baseline
    39.50 ± 0.710
    58.25 ± 47.910
    52.00 ± 38.360
        Day 15
    54.30 ± 17.390
    33.50 ± 9.040
    40.43 ± 15.000
        Change from Baseline
    14.80 ± 18.100
    -24.75 ± 50.970
    -11.57 ± 45.180
    No statistical analyses for this end point

    Secondary: Change From Baseline In Total Complement Classical Pathway (CP) Activity On Day 14

    Close Top of page
    End point title
    Change From Baseline In Total Complement Classical Pathway (CP) Activity On Day 14
    End point description
    Serum CP activity was measured by a functional immunoassay method. Change from Baseline = Total Complement CP Activity on Day 14 - Baseline Total Complement CP Activity
    End point type
    Secondary
    End point timeframe
    Baseline, Day 14
    End point values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects analysed
    2
    4
    6
    Units: CAE Unit
    arithmetic mean (standard deviation)
        Baseline
    31.00 ± 21.210
    91.00 ± 41.370
    71.00 ± 45.570
        Day 14
    41.50 ± 23.330
    96.75 ± 23.680
    78.33 ± 35.490
        Change from Baseline
    10.50 ± 2.120
    5.75 ± 34.250
    7.33 ± 26.660
    No statistical analyses for this end point

    Secondary: Change From Baseline In Total Complement Alternative Pathway (AP) Functional Activity (AP Wieslab) On Day 15

    Close Top of page
    End point title
    Change From Baseline In Total Complement Alternative Pathway (AP) Functional Activity (AP Wieslab) On Day 15
    End point description
    Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Total Complement AP Functional Activity on Day 15 - Baseline Total Complement AP Functional Activity
    End point type
    Secondary
    End point timeframe
    Baseline, Day 15
    End point values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects analysed
    2
    4
    6
    Units: percentage
    arithmetic mean (standard deviation)
        Baseline
    8.350 ± 10.508
    31.073 ± 19.779
    23.498 ± 19.862
        Day 15
    1.635 ± 2.3122
    0.993 ± 1.1101
    1.207 ± 1.3852
        Change from Baseline
    -6.715 ± 8.1954
    -30.08 ± 19.176
    -22.29 ± 19.484
    No statistical analyses for this end point

    Secondary: Time To Achieving Peak Serum C3 Levels

    Close Top of page
    End point title
    Time To Achieving Peak Serum C3 Levels
    End point description
    End point type
    Secondary
    End point timeframe
    From The First Day Of Dosing through Day 14
    End point values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects analysed
    2
    4
    6
    Units: days
        arithmetic mean (standard deviation)
    2.5 ± 2.12
    10.5 ± 4.43
    7.8 ± 5.46
    No statistical analyses for this end point

    Secondary: Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation

    Close Top of page
    End point title
    Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation
    End point description
    An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), 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.
    End point type
    Secondary
    End point timeframe
    Up to Day 49
    End point values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects analysed
    2
    4
    6
    Units: Participants
        TEAEs
    2
    3
    5
        SAEs
    0
    1
    1
        TEAEs ≥Grade 3
    0
    0
    0
        TEAEs leading to discontinuation
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Postdose (AUC0-8)

    Close Top of page
    End point title
    Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Postdose (AUC0-8)
    End point description
    Serial blood samples were collected predose and up to 8 hours postdose on Days 1 and 7.
    End point type
    Secondary
    End point timeframe
    Days 1 and 7
    End point values
    Group 1: Danicopan 100 mg TID Group 2: Danicopan 150 mg TID Group 2: Danicopan 200 mg TID
    Number of subjects analysed
    2
    1
    3
    Units: h*ng/mL
    geometric mean (geometric coefficient of variation)
        Day 1
    871 ± 38.1
    2060 ± 0
    1640 ± 42.8
        Day 7
    1120 ± 44.4
    2470 ± 0
    1760 ± 24.2
    No statistical analyses for this end point

    Secondary: PK: Maximum Plasma Concentration (Cmax)

    Close Top of page
    End point title
    PK: Maximum Plasma Concentration (Cmax)
    End point description
    Serial blood samples were collected predose and up to 8 hours postdose on Days 1 and 7.
    End point type
    Secondary
    End point timeframe
    Days 1 and 7
    End point values
    Group 1: Danicopan 100 mg TID Group 2: Danicopan 150 mg TID Group 2: Danicopan 200 mg TID
    Number of subjects analysed
    2
    1
    3
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Day 1
    199 ± 6.75
    563 ± 0
    427 ± 46.3
        Day 7
    270 ± 41.2
    579 ± 0
    385 ± 39.1
    No statistical analyses for this end point

    Secondary: PK: Time To Maximum Concentration (Tmax)

    Close Top of page
    End point title
    PK: Time To Maximum Concentration (Tmax)
    End point description
    Serial blood samples were collected predose and up to 8 hours postdose on Days 1 and 7.
    End point type
    Secondary
    End point timeframe
    Days 1 and 7
    End point values
    Group 1: Danicopan 100 mg TID Group 2: Danicopan 150 mg TID Group 2: Danicopan 200 mg TID
    Number of subjects analysed
    2
    1
    3
    Units: hours
    median (full range (min-max))
        Day 1
    1.00 (1.00 to 1.00)
    2.50 (2.50 to 2.50)
    2.00 (1.50 to 2.00)
        Day 7
    2.75 (1.50 to 4.00)
    2.50 (2.50 to 2.50)
    1.50 (1.00 to 4.00)
    No statistical analyses for this end point

    Secondary: Change From Baseline In Bb Fragment Of Complement Factor B (Bb) At Day 15

    Close Top of page
    End point title
    Change From Baseline In Bb Fragment Of Complement Factor B (Bb) At Day 15
    End point description
    Plasma Bb was measured by enzyme-linked immunosorbent assay (ELISA). Change from Baseline = Complement Bb on Day 15 - Baseline
    End point type
    Secondary
    End point timeframe
    Baseline, Day 15
    End point values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects analysed
    2
    4
    6
    Units: µg/mL
    arithmetic mean (standard deviation)
        Baseline
    1.786 ± 1.2116
    1.229 ± 0.4729
    1.416 ± 0.7152
        Day 15
    1.511 ± 0.9781
    0.622 ± 0.3349
    0.918 ± 0.6854
        Change from baseline
    -0.278 ± 0.2335
    -0.607 ± 0.1790
    -0.497 ± 0.2430
    No statistical analyses for this end point

    Secondary: Change From Baseline In Soluble Terminal Complement Complex (sC5b-9) At Day 15

    Close Top of page
    End point title
    Change From Baseline In Soluble Terminal Complement Complex (sC5b-9) At Day 15
    End point description
    Plasma sC5b-9 was measured by ELISA. Change from Baseline = sC5b-9 on Day 15 - Baseline sC5b-9
    End point type
    Secondary
    End point timeframe
    Baseline, Day 15
    End point values
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID Total
    Number of subjects analysed
    2
    4
    6
    Units: ng/mL
    arithmetic mean (standard deviation)
        Baseline
    1002.0 ± 684.4794
    613.750 ± 225.2397
    743.167 ± 405.3874
        Day 15
    1105.5 ± 622.9611
    563.850 ± 302.4446
    744.400 ± 459.0596
        Change from Baseline
    103.500 ± 61.5183
    -49.900 ± 237.1917
    1.233 ± 201.9602
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    After the first dose of study medication (following Day 0) through the follow-up visit at Day 49.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    12.1
    Reporting groups
    Reporting group title
    Group 1: Danicopan 100 mg TID (Sentinel)
    Reporting group description
    Participants received 100 mg of danicopan TID during the Treatment Period.

    Reporting group title
    Group 2: Danicopan up to 200 mg TID
    Reporting group description
    Participants received not more than 200 mg of danicopan TID during the Treatment Period.

    Serious adverse events
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 4 (25.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Nervous system disorders
    Presyncope
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Group 1: Danicopan 100 mg TID (Sentinel) Group 2: Danicopan up to 200 mg TID
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    3 / 4 (75.00%)
    Vascular disorders
    Hypotension
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Nervous system disorders
    Dizziness
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Headache
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 2 (50.00%)
    1 / 4 (25.00%)
         occurrences all number
    1
    1
    General disorders and administration site conditions
    Crepitations
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Pain
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Diarrhoea
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Prerenal failure
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Infections and infestations
    Upper respiratory tract infection
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Hyperkalaemia
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 4 (25.00%)
         occurrences all number
    0
    1
    Hypokalaemia
    alternative dictionary used: MedDRA 20.0
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 4 (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
    07 Apr 2017
    • Dose was increased based on current PK modeling and clinical safety in healthy volunteers. In Group 1, participants were given 100 mg TID instead of 75 mg TID. For Group 2, the maximum dose was changed from 125 mg TID to 150 mg TID. • Circumstances under which an early dosing taper would be initiated were modified. Two consecutive C3 levels >125% upper limit of normal (ULN) or >3× a participant’s baseline value and greater than or equal to the lower limit of normal triggered initiation of the taper period, rather than >125% ULN after 7 days. • Potential long-term follow-up visits were added to allow collection of longitudinal observational data. Participants were asked, but not required, to visit the clinic every 45 days for up to 1 year. • AE definitions were clarified. • Grading of AEs was changes from Division of AIDS criteria to CTCAE. • The phase of the study was changed from 1b to 2a.
    09 Jun 2017
    • Dose levels were updated based on current PK modeling and clinical safety in healthy volunteers. The maximum dose level for Group 2 was changed from 150 mg to 200 mg TID. • The eGFR calculation method was revised. The Modified Diet for Renal Disease equation was used for participants ≥18 years of age, and the Schwartz equation was used for participants <18 years of age. • Blood volumes for clinical laboratory tests were updated to include blood drawn at the optional long-term follow-up visits. • AE definitions were further clarified. • The previous human experience was updated.

    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.
    Sample size was based on very limited clinical cases of C3G and IC-MPGN. Plasma intact C3 values should be regarded with caution as they were generated using a new multiplex assay method with limited historical experience and unexplained variation.
    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 © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA