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    Clinical Trial Results:
    A Phase 2, Proof-of-Concept, Randomized, Double-Blinded, Placebo-Controlled Study of ACH-0144471 Treatment for 6 Months in Patients with C3 Glomerulopathy (C3G), with an Open-label Extension

    Summary
    EudraCT number
    		 2017-000663-33
    Trial protocol
    		 GB  
    Global end of trial date
    18 Dec 2020

    Results information
    Results version number
    		 v2(current)
    This version publication date
    31 Aug 2022
    First version publication date
    25 Jun 2021
    Other versions
    		 v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    ACH471-204
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03369236
    WHO universal trial number (UTN)
    		 U1111-1203-9076
    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 7 87148158, clinicaltrials.eu@alexion.com
    Scientific contact
    Alexion Europe SAS European Clinical Trial Information, Alexion Pharmaceuticals Inc., +33 7 87148158, clinicaltrials.eu@alexion.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002310-PIP02-17
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    26 Mar 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Dec 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Dec 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy, relative to placebo, of 6 months of oral treatment with ACH-0144471 (also known as danicopan and ALXN2040) in participants with C3G based on: • Renal biopsy results • Improvement relative to baseline in proteinuria
    Protection of trial subjects
    This study was conducted in accordance with the ethical principles of Good Clinical Practice (GCP), according to the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Harmonized Tripartite Guideline, 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
    12 Jun 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 21 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 12
    Country: Number of subjects enrolled
    United Kingdom: 1
    Worldwide total number of subjects
    13
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    11
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 To enroll in the study, participants were required to have a biopsy confirmed diagnosis of C3G, with initial diagnosis made at least 3 months prior to dosing and significant proteinuria.

    Period 1
    Period 1 title
    Double-blind Treatment Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator
    Blinding implementation details
    Participants were randomized 1:1 to treatment with danicopan or placebo during the 6 month Blinded Treatment Period.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Danicopan (Double-blind), Followed by Danicopan (Open-label)
    Arm description
    		 Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    Other name
    ACH-4471, ACH4471, 4471, ACH-0144471 (formerly), ALXN2040
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Danicopan was administered TID.

    Arm title
    		 Placebo (Double-blind), Followed by Danicopan (Open-label)
    Arm description
    		 Placebo was administered TID during the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Matching placebo was administered TID.

    Number of subjects in period 1
    		Danicopan (Double-blind), Followed by Danicopan (Open-label) Placebo (Double-blind), Followed by Danicopan (Open-label)
    Started
    6
    7
    Received at least 1 dose of study drug
    6
    7
    Completed
    6
    6
    Not completed
    0
    1
         Accidental unblinding
    -
    1
    Period 2
    Period 2 title
    Open-label Extension period
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Danicopan (Double-blind), Followed by Danicopan (Open-label)
    Arm description
    		 Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    Other name
    ACH-4471, ACH4471, 4471, ACH-0144471 (formerly), ALXN2040
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Danicopan 200 mg was administered TID.

    Arm title
    		 Placebo (Double-blind), Followed by Danicopan (Open-label)
    Arm description
    		 Placebo was administered TID during the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Danicopan
    Investigational medicinal product code
    Other name
    ACH-4471, ACH4471, 4471, ACH-0144471 (formerly), ALXN2040
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Danicopan 200 mg was administered TID.

    Number of subjects in period 2
    		Danicopan (Double-blind), Followed by Danicopan (Open-label) Placebo (Double-blind), Followed by Danicopan (Open-label)
    Started
    6
    6
    Received at least 1 dose of study drug
    6
    6
    Completed
    1
    0
    Not completed
    5
    6
         Sponsor decision to close study
    3
    4
         Adverse event, non-fatal
    -
    1
         Lack of efficacy
    2
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Danicopan (Double-blind), Followed by Danicopan (Open-label)
    Reporting group description
    		 Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.

    Reporting group title
    Placebo (Double-blind), Followed by Danicopan (Open-label)
    Reporting group description
    		 Placebo was administered TID during the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.

    Reporting group values
    		 Danicopan (Double-blind), Followed by Danicopan (Open-label) Placebo (Double-blind), Followed by Danicopan (Open-label) Total
    Number of subjects
    		 6 7 13
    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)
    		 1 1 2
        Adults (18-64 years)
    		 5 6 11
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 25.5 ± 10.54 24.9 ± 4.85 -
    Gender categorical
    Units: Subjects
        Female
    		 2 2 4
        Male
    		 4 5 9
    Race
    Units: Subjects
        Asian
    		 0 2 2
        White
    		 5 4 9
        Other
    		 1 1 2
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 3 1 4
        Not Hispanic or Latino
    		 3 6 9

    End points

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    End points reporting groups
    Reporting group title
    Danicopan (Double-blind), Followed by Danicopan (Open-label)
    Reporting group description
    		 Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.

    Reporting group title
    Placebo (Double-blind), Followed by Danicopan (Open-label)
    Reporting group description
    		 Placebo was administered TID during the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.
    Reporting group title
    Danicopan (Double-blind), Followed by Danicopan (Open-label)
    Reporting group description
    		 Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.

    Reporting group title
    Placebo (Double-blind), Followed by Danicopan (Open-label)
    Reporting group description
    		 Placebo was administered TID during the 6-month Treatment Period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and received danicopan 200 mg TID.

    Subject analysis set title
    Danicopan (Double-blind Treatment Period)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.

    Subject analysis set title
    Placebo (Double-blind Treatment Period)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Placebo was administered TID during the 6-month treatment period.

    Subject analysis set title
    Total
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.

    Primary: Change From Baseline In Composite Biopsy Score At Week 28

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    End point title
    		 Change From Baseline In Composite Biopsy Score At Week 28 [1]
    End point description
    The composite biopsy score was based on a score incorporating changes in the activity index, glomerular C3c staining, and glomerular macrophage infiltration at the end of 6 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes. All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
    End point type
    Primary
    End point timeframe
    Baseline, Week 28
    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: Mean values and mean change from baseline were summarized descriptively by treatment group.
    End point values
    		 Danicopan (Double-blind Treatment Period) Placebo (Double-blind Treatment Period)
    Number of subjects analysed
    6
    6 [2]
    Units: score on a scale
    arithmetic mean (standard deviation)
        Baseline
    11.7 ± 4.23
    9.3 ± 3.50
        Week 28
    9.2 ± 4.87
    10.7 ± 3.39
        Change from Baseline
    -2.0 ± 1.87
    1.3 ± 2.88
    Notes
    [2] - Week 28: n=5 Change from Baseline: n=5
    No statistical analyses for this end point

    Primary: Participants With Reduction In Proteinuria At Week 28

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    End point title
    		 Participants With Reduction In Proteinuria At Week 28 [3]
    End point description
    Proteinuria reduction was defined as ≥ 30% decrease from baseline based on 24-hour urine protein (mg/day). All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
    End point type
    Primary
    End point timeframe
    Week 28
    Notes
    [3] - 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: Mean values and mean change from baseline were summarized descriptively by treatment group.
    End point values
    		 Danicopan (Double-blind Treatment Period) Placebo (Double-blind Treatment Period)
    Number of subjects analysed
    4
    5
    Units: participants
        number (not applicable)
    0
    1
    No statistical analyses for this end point

    Secondary: Change From Baseline In Proteinuria At Week 28

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    End point title
    		 Change From Baseline In Proteinuria At Week 28
    End point description
    Proteinuria was assessed based on 24-hour urine collections at baseline and Week 28. All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 Danicopan (Double-blind Treatment Period) Placebo (Double-blind Treatment Period)
    Number of subjects analysed
    6 [4]
    7 [5]
    Units: mg/day
    arithmetic mean (standard deviation)
        Baseline
    6137.67 ± 2904.359
    4274.47 ± 2992.819
        Week 28
    5301.75 ± 2984.445
    5186.80 ± 4069.279
        Change from Baseline
    302.00 ± 764.211
    182.20 ± 994.558
    Notes
    [4] - Week 28: n=4 Change from Baseline: n=4
    [5] - Week 28: n=5 Change from Baseline: n=5
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline In Proteinuria At Week 28

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    End point title
    		 Percent Change From Baseline In Proteinuria At Week 28
    End point description
    Proteinuria was assessed based on 24-hour urine collections at baseline and Week 28. All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 Danicopan (Double-blind Treatment Period) Placebo (Double-blind Treatment Period)
    Number of subjects analysed
    4
    5
    Units: percent change
        arithmetic mean (standard deviation)
    12.5 ± 31.11
    -10.4 ± 31.35
    No statistical analyses for this end point

    Secondary: Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To 6 Months

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    End point title
    		 Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To 6 Months
    End point description
    Slope of eGFR was estimated using a simple linear regression for each participant, including all data values from baseline until the end of the 6-month blinded treatment period, with eGFR as the dependent variable and time as the independent variable. All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
    End point type
    Secondary
    End point timeframe
    6 months
    End point values
    		 Danicopan (Double-blind Treatment Period) Placebo (Double-blind Treatment Period)
    Number of subjects analysed
    6
    7
    Units: mL/min/1.73 m^2 per month
        arithmetic mean (standard deviation)
    -2.26840 ± 1.790814
    -1.45421 ± 2.228395
    No statistical analyses for this end point

    Secondary: Slope Of Estimated Glomerular Filtration Rate (eGFR) After Open-label Danicopan Treatment

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    End point title
    		 Slope Of Estimated Glomerular Filtration Rate (eGFR) After Open-label Danicopan Treatment
    End point description
    Slope of eGFR was estimated using a simple linear regression for each participant, including all data values during the open-label extension period with eGFR as the dependent variable and time as the independent variable. All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Danicopan (Double-blind), Followed by Danicopan (Open-label) Placebo (Double-blind), Followed by Danicopan (Open-label) Total
    Number of subjects analysed
    5
    4
    9
    Units: mL/min/1.73 m^2 per month
        arithmetic mean (standard deviation)
    -1.02556 ± 1.389128
    -1.84935 ± 3.506810
    -1.39169 ± 2.401039
    No statistical analyses for this end point

    Secondary: Change From Baseline In eGFR At Week 28

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    End point title
    		 Change From Baseline In eGFR At Week 28
    End point description
    Change from baseline in eGFR at Week 28 is presented.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 Danicopan (Double-blind Treatment Period) Placebo (Double-blind Treatment Period)
    Number of subjects analysed
    6
    7 [6]
    Units: mL/min/1.73 m^2
    arithmetic mean (standard deviation)
        Baseline
    79.890 ± 44.4571
    68.381 ± 36.7249
        Week 28
    67.543 ± 47.8062
    50.874 ± 15.0517
        Change from Baseline
    -12.347 ± 10.8063
    -8.700 ± 16.0990
    Notes
    [6] - Week 28: n=5 Change from Baseline: n=5
    No statistical analyses for this end point

    Secondary: Participants With Significant Improvement In eGFR Relative To Baseline At Week 28

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    End point title
    		 Participants With Significant Improvement In eGFR Relative To Baseline At Week 28
    End point description
    Significant improvement relative to baseline was defined as a ≥ 20% increase from baseline in eGFR. All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 Danicopan (Double-blind Treatment Period) Placebo (Double-blind Treatment Period)
    Number of subjects analysed
    6
    5
    Units: participants
        number (not applicable)
    0
    0
    No statistical analyses for this end point

    Secondary: Participants With Significant Improvement In eGFR Relative To Baseline At Week 52

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    End point title
    		 Participants With Significant Improvement In eGFR Relative To Baseline At Week 52
    End point description
    Significant improvement relative to baseline was defined as a ≥ 20% increase from baseline in eGFR. All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 Danicopan (Double-blind), Followed by Danicopan (Open-label) Placebo (Double-blind), Followed by Danicopan (Open-label)
    Number of subjects analysed
    5
    5
    Units: participants
        number (not applicable)
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Danicopan (Double-blind Treatment Period)
    Reporting group description
    		 Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.

    Reporting group title
    Danicopan (Open-label Extension Period)
    Reporting group description
    		 All participants who received danicopan or placebo in the treatment period were to receive danicopan 200 mg TID during the open-label extension period.

    Reporting group title
    Placebo (Double-Blind Treatment Period)
    Reporting group description
    		 Placebo was administered TID during the 6-month treatment period.

    Serious adverse events
    		 Danicopan (Double-blind Treatment Period) Danicopan (Open-label Extension Period) Placebo (Double-Blind Treatment Period)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Danicopan (Double-blind Treatment Period) Danicopan (Open-label Extension Period) Placebo (Double-Blind Treatment Period)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 6 (83.33%)
    8 / 12 (66.67%)
    5 / 7 (71.43%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Orthostatic hypotension
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Pallor
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Dizziness
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    0
    Feeling cold
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Oedema peripheral
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    1
    Pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Face oedema
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Social circumstances
    Pregnancy of partner
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Reproductive system and breast disorders
    Perineal pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Nasal congestion
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    3
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    0
    Cough
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Epistaxis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Lower respiratory tract congestion
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Paranasal sinus discomfort
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Psychiatric disorders
    Nightmare
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    2
    Anxiety
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Insomnia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 12 (8.33%)
    1 / 7 (14.29%)
         occurrences all number
    2
    1
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    2 / 7 (28.57%)
         occurrences all number
    0
    0
    2
    Hand fracture
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Skin laceration
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    1
    Joint injury
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    2
    Dizziness
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    1
    Dizziness postural
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Lethargy
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Memory impairment
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Hypoaesthesia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Migraine
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Eye disorders
    Eye pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Vision blurred
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 12 (8.33%)
    2 / 7 (28.57%)
         occurrences all number
    2
    1
    2
    Vomiting
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 12 (8.33%)
    2 / 7 (28.57%)
         occurrences all number
    1
    1
    3
    Diarrhoea
         subjects affected / exposed
    1 / 6 (16.67%)
    2 / 12 (16.67%)
    0 / 7 (0.00%)
         occurrences all number
    1
    2
    0
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    0
    Macule
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    1
    0
    1
    Joint effusion
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Muscle spasms
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Muscular weakness
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Arthropathy
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Musculoskeletal stiffness
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Neck pain
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Pain in extremity
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Infections and infestations
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    3
    0
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0
    Pharyngitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 12 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    0
    1
    Nasopharyngitis
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 12 (16.67%)
    0 / 7 (0.00%)
         occurrences all number
    0
    3
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 12 (8.33%)
    0 / 7 (0.00%)
         occurrences all number
    0
    1
    0
    Metabolism and nutrition disorders
    Gout
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 12 (0.00%)
    0 / 7 (0.00%)
         occurrences all number
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Oct 2017
    • Allowed visits on Weeks 14, 18, 22, and 26 to be conducted by phone rather than in the clinic, at the discretion of the investigator, in order to reduce the burden on participants in the study.
    12 Dec 2017
    • Allowed investigators who wished to do so to collect measured glomerular filtration rate (GFR) in addition to the existing eGFR calculations. • The contraception requirements were modified to align with updated standard wording, and serious adverse event reporting contact information was updated.
    07 Mar 2018
    • Changed improvement in eGFR from a primary to a secondary objective and aligned endpoints with this change. • Adjusted inclusion/exclusion criteria related to complement components in order to facilitate enrollment of suitable participants. • Removed the planned collection pharmacokinetic profile samples on Day 3 in order to reduce the burden on participants. • The contraception requirements were modified to align with updated standard wording. • Vaccination procedures were updated to refer to national and/or local guidelines, minor wording changes are being made for clarity.
    15 Feb 2019
    • Made changes to the inclusion and exclusion criteria to better reflect the intended participant population and to facilitate enrollment. • Changed the dose escalation strategy so that all participants will escalate after 2 weeks. • Reduced sample collection and added flexibility to the collection schedule to reduce the burden on participants (including making collection of renal biopsies an optional sub-study).
    19 Jun 2019
    • Made changes to the inclusion and exclusion criteria to remove the allowance of adolescents in the study population. • Reduced sample collection and added flexibility to the collection schedule to reduce the burden on participants. • Removed the biopsy sub-study option (all participants had biopsy- confirmed diagnosis). • Extended the study to Week 104 (addition of a 12-month long- term follow-up period). • Removed Data and Safety Monitoring Board since the study was only blinded to the Investigator, site staff and participants (Sponsor not blinded). • Reduced the number of in-clinic study visits by having the ability to do visits as phone calls. • Reduced the number of expected participants to 20.
    15 May 2020
    • Increased duration of study treatment. • Allowed home and telephone visits, local laboratory testing, and study drug to be sent directly to participant’s home when clinic visits were not possible due to the coronavirus disease 2019 (COVID-19) global pandemic. • Allowed optional renal biopsy at Week 52 and Week 104 to be postponed due to COVID-19 global pandemic, and to be performed when possible. • Updated contraceptive language to align with most recent Investigational Brochure.

    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.
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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