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    Clinical Trial Results:
    AN OPEN-LABEL PROOF OF CONCEPT PHASE IIA TRIAL OF ALXN1007 FOR THE TREATMENT OF NON-CRITERIA MANIFESTATIONS OF ANTIPHOSPHOLIPID SYNDROME

    Summary
    EudraCT number
    		 2013-003588-73
    Trial protocol
    		 GB   IT   ES  
    Global end of trial date
    20 Jun 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    02 Jul 2017
    First version publication date
    02 Jul 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ALXN1007-APS-201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02128269
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals Inc.
    Sponsor organisation address
    100 College Street, New Haven, United States, 06510
    Public contact
    European Clinical Trial Information, Alexion Europe SAS, +33 153643848, clinicaltrials.eu@alxn.com
    Scientific contact
    European Clinical Trial Information, Alexion Europe SAS, +33 153643848, clinicaltrials.eu@alxn.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
    14 Apr 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Jun 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Jun 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Safety and tolerability of intravenous (IV) ALXN1007 in persistently antiphospholipid (aPL) positive patients with at least 1 of the following non-criteria manifestations of antiphospholipid syndrome (APS): aPL nephropathy, skin ulcers, and/or thrombocytopenia.
    Protection of trial subjects
    All patients must be vaccinated against N. meningitidis if not already vaccinated within the time period of active coverage specified by the vaccine manufacturer.  Patients must be vaccinated at least 14 days prior to receiving the first dose of ALXN1007, or  Patients must be vaccinated and receive treatment with appropriate antibiotics until 14 days after the vaccination.
    Background therapy
    		 Patients may continue on their APS medications, but every effort should be made to keep the patients’ concomitant medications stable through the Week 24 visit.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Dec 2013
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Japan: 2
    Country: Number of subjects enrolled
    United States: 4
    Worldwide total number of subjects
    9
    EEA total number of subjects
    3
    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)
    7
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Screening discontinued early due to slow patient enrollment

    Pre-assignment
    Screening details
    		 22 total patients screened

    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 ALXN1007
    Arm description
    		 ALXN1007: 10 mg/kg IV q 2 weeks x 12 doses
    Arm type
    		 Experimental

    Investigational medicinal product name
    ALXN1007
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    ALXN1007: 10 mg/kg IV q 2 weeks x 12 doses

    Number of subjects in period 1
    		ALXN1007
    Started
    9
    Completed
    7
    Not completed
    2
         Physician decision
    1
         Adverse event, non-fatal
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Trial
    Reporting group description
    		 All patients (included those who discontinued prematurely) were followed for 12 weeks after last infusion

    Reporting group values
    		 Overall Trial Total
    Number of subjects
    		 9 9
    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)
    		 7 7
        From 65-84 years
    		 2 2
        85 years and over
    		 0 0
    Gender categorical
    Units: Subjects
        Female
    		 3 3
        Male
    		 6 6

    End points

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    End points reporting groups
    Reporting group title
    ALXN1007
    Reporting group description
    		 ALXN1007: 10 mg/kg IV q 2 weeks x 12 doses

    Primary: Safety and Tolerability

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    End point title
    		 Safety and Tolerability [1]
    End point description
    Measured by Percentage of Patients Reporting Adverse Events
    End point type
    Primary
    End point timeframe
    24 Weeks
    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: No comparative analysis was done. Only percentage of patients experiencing adverse events was reported based on all patients with at least one dose of ALXN1007. Adverse events are summarized for 24 week treatment period and 12 week follow up period combined.
    End point values
    		 ALXN1007
    Number of subjects analysed
    9
    Units: Patient numbers
    8
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Treatment Period (24 weeks) and Follow-up Period (12 weeks)
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.0
    Reporting groups
    Reporting group title
    ALXN1007
    Reporting group description
    		 ALXN1007: 10 mg/kg IV q 2 weeks x 12 doses

    Serious adverse events
    		 ALXN1007
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 9 (22.22%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Injury, poisoning and procedural complications
    Lumbar vertebral fracture
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Wrist fracture
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Abortion missed
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholestasis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Liver Cytolysis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Lumbar spinal stenosis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 ALXN1007
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 9 (88.89%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Squamous cell carcinoma
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Vascular disorders
    Varicose vein
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nervous system disorders
    Carpal tunnel syndrome
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Dizziness
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Hypoaesthesia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Restless legs syndrome
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Antiphospholipid syndrome
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Thrombocytopenia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Enterocolitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Haemorrhoids
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Hypoaesthesia oral
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Colitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Epistaxis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Cystitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Labyrinthitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Oral herpes
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Paronychia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Pharyngitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Staphylococcal infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Tracheitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 9 (44.44%)
         occurrences all number
    8
    Urethritis
         subjects affected / exposed
    1 / 9 (11.11%)
         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
    14 Nov 2013
    Amendment included changes to laboratory safety assessments, acceptable birth control methods, antibody testing, study drug storage conditions, and the adverse event severity assessment grading scale.
    24 Jan 2014
    The Amendment revised the required use of contraception to 6 months after receiving the last dose of study drug for both female and male patients.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    31 Aug 2015
    Screening for the study was stopped due to slow patient recruitment. Initially enrolled patients completed the study.
    -

    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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    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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