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    Clinical Trial Results:
    A Phase 3 Study to Evaluate the Safety and Efficacy of OMS721 for the Treatment of Atypical Hemolytic Uremic Syndrome (aHUS) in Adults and Adolescents.

    Summary
    EudraCT number
    		 2017-002057-11
    Trial protocol
    		 LT   PL  
    Global end of trial date
    27 Nov 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Jun 2026
    First version publication date
    28 Jun 2026
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    OMS721-HUS-002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03205995
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Omeros Corporation
    Sponsor organisation address
    201 Elliott Avenue West, Seattle, United States, 98119
    Public contact
    Steve Whitaker, Omeros Corporation, 206 676-5000,
    Scientific contact
    Steve Whitaker, Omeros Corporation, 206 676-5000,
    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
    02 Feb 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    02 Feb 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Nov 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this study is to evaluate the effect of OMS721 in subjects with aHUS on: • Platelet count change from baseline
    Protection of trial subjects
    N/A
    Background therapy
    		 N/A
    Evidence for comparator
    		 N/A
    Actual start date of recruitment
    06 Sep 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
    Poland: 3
    Country: Number of subjects enrolled
    Lithuania: 3
    Worldwide total number of subjects
    6
    EEA total number of subjects
    6
    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

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    Recruitment
    Recruitment details
    		 The study was conducted globally at different sites between 02 May 2018 and 27 Nov 2023.

    Pre-assignment
    Screening details
    		 Overall, 6 subjects were enrolled and randomized into this uncontrolled, open-label study.

    Period 1
    Period 1 title
    Treatment Induction Period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    N/A

    Arms
    Arm title
    		 Narsoplimab
    Arm description
    		 Patients received narsoplimab 370 mg IV on Days 1 and 4. Beginning on the day of the first dose (Day 1), patients also began treatment with narsoplimab 150 mg subcutaneously (SC) once daily. If a patient received plasma therapy during the Treatment Induction Period, the patient received supplemental narsoplimab 185 mg IV. If the patient received plasma therapy on Day 1 or Day 4 of the Treatment Induction Period, the regularly scheduled narsoplimab dose was administered within 1 hour after plasma exchange or within 1 hour before plasma infusion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Narsoplimab
    Investigational medicinal product code
    Other name
    OMS721
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received narsoplimab 370 mg IV on Days 1 and 4. Beginning on the day of the first dose (Day 1), patients also began treatment with narsoplimab 150 mg subcutaneously (SC) once daily. If a patient received plasma therapy during the Treatment Induction Period, the patient received supplemental narsoplimab 185 mg IV. If the patient received plasma therapy on Day 1 or Day 4 of the Treatment Induction Period, the regularly scheduled narsoplimab dose was administered within 1 hour after plasma exchange or within 1 hour before plasma infusion.

    Number of subjects in period 1
    		Narsoplimab
    Started
    6
    Completed
    0
    Not completed
    6
         Physician decision
    2
         Adverse event, non-fatal
    1
         Death
    1
         Protocol deviation
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Treatment Induction Period
    Reporting group description
    		 -

    Reporting group values
    		 Treatment Induction Period Total
    Number of subjects
    		 6 6
    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)
    		 6 6
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Gender categorical
    Units: Subjects
        Female
    		 5 5
        Male
    		 1 1
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 6 6
        Not Reported/Unknown
    		 0 0
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 0 0
        Black or African American
    		 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0
        White
    		 6 6
        Other
    		 0 0
    Child Bearing Potential,
    Units: Subjects
        Yes
    		 5 5
        No
    		 0 0
        Not Applicable
    		 1 1
    Baseline Weight
    Units: kg
        arithmetic mean (standard deviation)
    		 65.8 ( 13.78 ) -
    Baseline BMI
    Units: kg/m2)
        arithmetic mean (standard deviation)
    		 23.3 ( 4.66 ) -
    Baseline systolic blood pressure
    Units: mmHg
        arithmetic mean (standard deviation)
    		 148 ( 13.8 ) -
    Baseline diastolic blood pressure
    Units: mmHg
        arithmetic mean (standard deviation)
    		 87.5 ( 10.15 ) -

    End points

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    End points reporting groups
    Reporting group title
    Narsoplimab
    Reporting group description
    		 Patients received narsoplimab 370 mg IV on Days 1 and 4. Beginning on the day of the first dose (Day 1), patients also began treatment with narsoplimab 150 mg subcutaneously (SC) once daily. If a patient received plasma therapy during the Treatment Induction Period, the patient received supplemental narsoplimab 185 mg IV. If the patient received plasma therapy on Day 1 or Day 4 of the Treatment Induction Period, the regularly scheduled narsoplimab dose was administered within 1 hour after plasma exchange or within 1 hour before plasma infusion.

    Primary: Platelet Count (10^9 Platelets/L) Change From Baseline at Week 26

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    End point title
    		 Platelet Count (10^9 Platelets/L) Change From Baseline at Week 26 [1]
    End point description
    The primary outcome to be measured is platelet count change from baseline.
    End point type
    Primary
    End point timeframe
    Week 26
    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: As the endpoint is descriptive in nature, no statistical analysis is provided.
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: 10^9 platelets/L
        median (full range (min-max))
    42 (-21 to 115.5)
    No statistical analyses for this end point

    Secondary: Safety as Measured by Incidences of Adverse Events, Vital Signs, ECG, and Clinical Laboratory Tests

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    End point title
    		 Safety as Measured by Incidences of Adverse Events, Vital Signs, ECG, and Clinical Laboratory Tests
    End point description
    Assessment of safety of OMS721 (narsoplimab) in participants with aHUS by incidence of Adverse Events, clinically significant vital sign abnormalities, ECG abnormalities, and clinical laboratory test abnormalities
    End point type
    Secondary
    End point timeframe
    Pre-dose and up to 771 days post-dose
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: Participants
    6
    No statistical analyses for this end point

    Secondary: Thrombotic Microangiopathies (TMA) Response

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    End point title
    		 Thrombotic Microangiopathies (TMA) Response
    End point description
    Complete TMA response defined as normalization of platelet count, normalization of serum lactate dehydrogenase (LDH), and > 25% decrease in serum creatinine by at least 2 consecutive measures over at least 4 consecutive weeks, within the initial 26-week period
    End point type
    Secondary
    End point timeframe
    26 weeks
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: Participants
    0
    No statistical analyses for this end point

    Secondary: TMA Event-free Status

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    End point title
    		 TMA Event-free Status
    End point description
    No decrease in platelet count of > 25% from baseline, no plasma exchange or plasma infusion, and no initiation of new dialysis over at least 12 consecutive weeks, within the initial 26-week period
    End point type
    Secondary
    End point timeframe
    26 weeks
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: Count of Participants
    2
    No statistical analyses for this end point

    Secondary: Increase in Estimated Glomerular Filtration Rate (eGFR)

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    End point title
    		 Increase in Estimated Glomerular Filtration Rate (eGFR)
    End point description
    Increase of greater than 15 ml/min/1.73 m2 in eGFR calculated by the modification of diet in renal disease (MDRD) Equation
    End point type
    Secondary
    End point timeframe
    26 weeks
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: Count of Participants
    0
    No statistical analyses for this end point

    Secondary: Hematological Normalization

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    End point title
    		 Hematological Normalization
    End point description
    Normalization of platelet count and normalization of serum LDH by 2 consecutive measurements over at least 4 weeks, within the initial 26-week period
    End point type
    Secondary
    End point timeframe
    26 weeks
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: Count of Participants
    1
    No statistical analyses for this end point

    Secondary: TMA Remission

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    End point title
    		 TMA Remission
    End point description
    Platelet count greater than or equal to 150,000/μL on at least 2 consecutive measures over at least 2 consecutive weeks, within the initial 26-week period
    End point type
    Secondary
    End point timeframe
    26 weeks
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6 [2]
    Units: Count of Participants
    2
    Notes
    [2] - Any patient who received drug
    No statistical analyses for this end point

    Secondary: Incidence of Antidrug Antibodies (ADA)

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    End point title
    		 Incidence of Antidrug Antibodies (ADA)
    End point description
    Incidences of ADA in participants with aHUS, administered OMS721 (narsoplimab)
    End point type
    Secondary
    End point timeframe
    771 days post-dose
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6 [3]
    Units: Count of Participants
    1
    Notes
    [3] - Any patient who received drug
    No statistical analyses for this end point

    Secondary: Change From Baseline in Serum Creatinine (mg/dL)

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    End point title
    		 Change From Baseline in Serum Creatinine (mg/dL)
    End point description
    Assessment of subject's change from baseline in serum creatinine.
    End point type
    Secondary
    End point timeframe
    26 weeks
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6 [4]
    Units: mg/dL
        median (full range (min-max))
    1.53 (-0.98 to 3.16)
    Notes
    [4] - Any patient who received drug
    No statistical analyses for this end point

    Secondary: Change From Baseline in Serum LDH (U/L)

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    End point title
    		 Change From Baseline in Serum LDH (U/L)
    End point description
    Assessment of subject's change from baseline in serum LDH
    End point type
    Secondary
    End point timeframe
    26 weeks
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6 [5]
    Units: U/L
        median (full range (min-max))
    -12 (-112 to 389)
    Notes
    [5] - Any patient who received drug
    No statistical analyses for this end point

    Secondary: Change From Baseline in Haptoglobin (mg/dL)

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    End point title
    		 Change From Baseline in Haptoglobin (mg/dL)
    End point description
    Assessment of subject's change from baseline in haptoglobin
    End point type
    Secondary
    End point timeframe
    26 weeks
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6 [6]
    Units: mg/dL
        median (full range (min-max))
    30 (4 to 56)
    Notes
    [6] - Any patient who received drug.
    No statistical analyses for this end point

    Secondary: Pharmacokinetics (PK): Trough Plasma Concentration, Lower Limit of Quantification (LLOQ)

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    End point title
    		 Pharmacokinetics (PK): Trough Plasma Concentration, Lower Limit of Quantification (LLOQ)
    End point description
    Pharmacokinetics (PK): Trough plasma concentration, lower limit of quantification (LLOQ)
    End point type
    Secondary
    End point timeframe
    Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (RT) (if occurs): RT Days 1-4; Follow-Up at Day 771
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: ng/mL
        geometric mean (standard deviation)
    15477.2 ( 6471.62 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetics (PK): Maximum Plasma Concentrations (Cmax)

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    End point title
    		 Pharmacokinetics (PK): Maximum Plasma Concentrations (Cmax)
    End point description
    Pharmacokinetics (PK): Maximum plasma concentrations (Cmax)
    End point type
    Secondary
    End point timeframe
    Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: ng/mL
        median (full range (min-max))
    48350 (25100 to 86300)
    No statistical analyses for this end point

    Secondary: Pharmacokinetics (PK): Area Under Time-concentration Curve (AUC)

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    End point title
    		 Pharmacokinetics (PK): Area Under Time-concentration Curve (AUC)
    End point description
    Pharmacokinetics (PK): Area under time-concentration curve (AUC) - Outcome measure data for this secondary outcome measure were not reported because no PK modeling could be performed due to insufficient data collection due to early study termination; therefore, there are no data or results to summarize.
    End point type
    Secondary
    End point timeframe
    Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6 [7]
    Units: ng/mL
        median (full range (min-max))
    0 (0 to 0)
    Notes
    [7] - PK modeling for calculating the AUC was not performed because the study was not completed.
    No statistical analyses for this end point

    Secondary: Pharmacodynamics (PD): Inhibition of C3 Activity (%)

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    End point title
    		 Pharmacodynamics (PD): Inhibition of C3 Activity (%)
    End point description
    Pharmacodynamics (PD): Inhibition of C3 activity
    End point type
    Secondary
    End point timeframe
    Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6 [8]
    Units: Inhibition of C3 activity (%)
        median (full range (min-max))
    84.4 (1.7 to 101.4)
    Notes
    [8] - Any patient who received drug and measurable C3 activity.
    No statistical analyses for this end point

    Secondary: Pharmacodynamics (PD): Inhibition of C4 Activity (%)

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    End point title
    		 Pharmacodynamics (PD): Inhibition of C4 Activity (%)
    End point description
    Pharmacodynamics (PD): Inhibition of C4 activity
    End point type
    Secondary
    End point timeframe
    Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771
    End point values
    		 Narsoplimab
    Number of subjects analysed
    6
    Units: Inhibition of C4 activity (%
        median (full range (min-max))
    91.9 (11.9 to 99.9)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the signing informed consent form to end of follow up
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19
    Reporting groups
    Reporting group title
    Narsoplimab 370 mg IV + 150 mg SC
    Reporting group description
    		 Patients received narsoplimab 370 mg IV on Days 1 and 4. Beginning on the day of the first dose (Day 1), patients also began treatment with narsoplimab 150 mg subcutaneously (SC) once daily. If a patient received plasma therapy during the Treatment Induction Period, the patient received supplemental narsoplimab 185 mg IV. If the patient received plasma therapy on Day 1 or Day 4 of the Treatment Induction Period, the regularly scheduled narsoplimab dose was administered within 1 hour after plasma exchange or within 1 hour before plasma infusion

    Serious adverse events
    		 Narsoplimab 370 mg IV + 150 mg SC
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 6 (83.33%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    0
    Injury, poisoning and procedural complications
    Arteriovenous fistula aneurysm
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Shunt blood flow excessive
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Cardiac failure
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Cerebral haematoma
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Blood and lymphatic system disorders
    Haemolysis
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Leukopenia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Immune system disorders
    Chronic allograft nephropathy
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Kidney transplant rejection
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Hepatic failure
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary hypertension
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Purpura
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Chronic kidney disease
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Escherichia sepsis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Pneumonia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal graft infection
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Narsoplimab 370 mg IV + 150 mg SC
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    Vascular disorders
    Alopecia
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Hypertension
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Purpura
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    3
    Injection site extravasation
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Injection site pain
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Injection site haematoma
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Oedema peripheral
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Immune system disorders
    Hepatic failure
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Reproductive system and breast disorders
    Chronic kidney disease
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Leukocyturia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Amenorrhoea
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Psychiatric disorders
    Cerebral haematoma
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Epilepsy
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Headache
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Neurological symptom
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Investigations
    Electrocardiogram QT prolonged
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Human chorionic gonadotropin abnormal
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Arteriovenous fistula aneurysm
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Post procedural haematoma
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Shunt blood flow excessive
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Subdural haematoma
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Bacterial infection
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Clostridium difficile infection
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Escherichia sepsis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Molluscum contagiosum
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Pneumonia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Ureaplasma infection
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Urinary tract infection enterococcal
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Congenital, familial and genetic disorders
    Hypertrophic cardiomyopathy
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Cardiac disorders
    Bradycardia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Cardiac failure
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Nervous system disorders
    Back pain
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Pain in extremity
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Haemolysis
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Leukopenia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Thrombocytopenia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Blood cholesterol increased
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Gastrointestinal disorders
    Ascites
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Enterocolitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Oesophageal ulcer
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Pulmonary hypertension
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Aggression
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Anxiety
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Insomnia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Endocrine disorders
    Adrenal insufficiency
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Hyperthyroidism
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Hypocalcaemia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Infections and infestations
    Arteriovenous fistula site infection
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Infection
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Renal graft infection
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Urinary tract infection
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Chronic allograft nephropathy
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Hypersensitivity
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Kidney transplant rejection
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    3
    Hyperphosphataemia
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Hyperuricaemia
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    C-reactive protein increased
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Treponema test positive
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 May 2017
    Amendment #1: - Change to Sections 2, 6.2, 7.3.2. Rationale: To update the study’s secondary objectives and secondary endpoints with these event times, which will be used in evaluating the efficacy of OMS721 in the treatment of aHUS subjects in the study. Added three secondary study objectives and secondary endpoints: * Time to TMA event-free status measured by the time to reach the first measurement of TMA event-free status from the first OMS721 dose * Time to eGFR increase of > 15 ml/min/1.73 m2 measured by the time to reach the first measurement increase in eGFR of > 15 ml/min/1.73 m2 from the first OMS721 dose * Time to hematological normalization measured by the time to reach the first measurement of hematological normalization from the first OMS721 dose

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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