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    Clinical Trial Results:
    Assessing the therapeutic efficacy and safety of an 11β-hydroxysteroid dehydrogenase type 1 inhibitor (AZD4017) in idiopathic intracranial hypertension (IIH).

    Summary
    EudraCT number
    		 2013-003643-31
    Trial protocol
    		 GB  
    Global end of trial date
    10 Feb 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    29 Aug 2019
    First version publication date
    29 Aug 2019
    Other versions
    Summary report(s)
    		 11β-Hydroxysteroid Dehydrogenase Type 1 inhibition in Idiopathic Intracranial

    Trial information

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    Trial identification
    Sponsor protocol code
    RG_13-022
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02017444
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    University of Birmingham
    Sponsor organisation address
    Edgbaston, Birmingham, United Kingdom, B15 2TT
    Public contact
    Dr Birgit Whitman, University of Birmingham, +44 1214158011, B.Whitman@bham.ac.uk
    Scientific contact
    Dr Birgit Whitman, University of Birmingham, +44 1214158011, B.Whitman@bham.ac.uk
    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
    25 Jan 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    19 Dec 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    10 Feb 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study is to evaluate whether 12 weeks of a 11β-hydroxysteroid dehydrogenase inhibitor (AZD4017) is effective and safe for reducing the raised intracranial pressure (pressure of fluid around the brain) observed in patients with idiopathic intracranial hypertension, compared to a placebo ('dummy' drug with no active properties). In the original grant application and early versions of the protocol, the primary outcome measure was stated as the change in ICP between baseline and 12 weeks. Following adoption of the study by the University of Birmingham Clinical Trials Unit, the primary outcome was changed to ICP at 12 weeks, with adjustment for baseline ICP in the analysis. This change was made blind to any data analysis.
    Protection of trial subjects
    The trial was discussed with potential participants and written information presented detailing the exact nature of the trial and the potential risks involved. It was clearly stated that participants were free to withdraw from the trial at any time and for any reason, with no obligation to give the reason for withdrawal and without affecting their future care. Since the effects of AZD4017 on unborn children are unknown, participants had pregnancy tests before randomisation and at intervals throughout the trial. Participants were also required to use one form of highly effective contraception. Informed by earlier trials investigating AZD4017, a panel of safety bloods were monitored throughout the trial, including renal function, liver function, thyroid function and creatine kinase. An independent Data Monitoring and Ethics Committee reviewed data including Adverse Events and safety blood results.
    Background therapy
    		 Although weight loss is generally advised for patients with IIH, the management of IIH varies considerably owing to a lack of supporting evidence. During trial design and registration, there was no evidence supporting the use of any particular medical treatment for IIH. For progressive or acute deterioration of vision in IIH, surgical techniques such as cerebrospinal fluid (CSF) shunting, optic nerve sheath fenestration, or venous sinus stenting have been used to prevent blindness. However, there is limited evidence to support these surgical interventions.
    Evidence for comparator
    		 AZD4017 was compared to placebo rather than a current medical treatment of IIH due to the lack of evidence for any other active treatment noted above.
    Actual start date of recruitment
    25 Apr 2014
    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
    United Kingdom: 31
    Worldwide total number of subjects
    31
    EEA total number of subjects
    31
    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)
    31
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 31 participants were recruited from 3 UK NHS Trusts between April 2014 and August 2016.

    Pre-assignment
    Screening details
    		 Patients were eligible for pre-screening (slit lamp examination for papilloedema Frisen grading ≥1 and a blood test) if they were female, ≥18 years old, with a confirmed diagnosis of active IIH (Modified Dandy criteria). Screening before randomisation then involved a lumbar puncture to confirm raised ICP and a urine pregnancy test.

    Period 1
    Period 1 title
    Treatment period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Assessor, Subject
    Blinding implementation details
    The trial drug and placebo was blinded, randomised, and packaged by Almac, contract manufacturing organisation on behalf of AstraZeneca. If unblinding was required, unblinding codes were held in the Pharmacy Departments of each Trust, as well as by the Trial Statistician with reasons for unblinding to be recorded.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 AZD4017
    Arm description
    		 An oral selective 11β-HSD1 inhibitor, AZD4017, at 400mg twice daily for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    AZD4017
    Investigational medicinal product code
    n/a
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    400mg twice daily (am and pm) for 12 weeks

    Arm title
    		 Placebo arm
    Arm description
    		 A matched placebo 400mg twice daily for 12 weeks.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    n/a
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    400mg twice daily (am and pm) for 12 weeks

    Number of subjects in period 1
    		AZD4017 Placebo arm
    Started
    17
    14
    Completed
    17
    12
    Not completed
    0
    2
         Physician decision
    -
    1
         Protocol deviation
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    AZD4017
    Reporting group description
    		 An oral selective 11β-HSD1 inhibitor, AZD4017, at 400mg twice daily for 12 weeks.

    Reporting group title
    Placebo arm
    Reporting group description
    		 A matched placebo 400mg twice daily for 12 weeks.

    Reporting group values
    		 AZD4017 Placebo arm Total
    Number of subjects
    		 17 14 31
    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)
    		 17 14 31
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age continuous
    Age in years
    Units: years
        arithmetic mean (standard deviation)
    		 30.1 ( 5.9 ) 32.4 ( 8 ) -
    Gender categorical
    Units: Subjects
        Female
    		 17 14 31
        Male
    		 0 0 0
    Ethnicity
    Units: Subjects
        White British
    		 16 13 29
        Asian/Asian British - Pakistani
    		 1 0 1
        Asian/Asian British - Other Asian
    		 0 1 1
    Number on acetazolamide
    Units: Subjects
        Number on acetazolamide
    		 6 4 10
        Not on acetazolamide
    		 11 10 21
    IIH Symptoms
    Headache
    Units: Subjects
        Headache
    		 16 14 30
        No headache
    		 1 0 1
    Frisén Grading, number
    Worst eye only
    Units: Subjects
        Frisén Grade 1
    		 4 2 6
        Frisén Grade 2
    		 9 5 14
        Frisén Grade 3
    		 0 3 3
        Frisén Grade 4
    		 2 1 3
        Frisén Grade 5
    		 1 0 1
        Not recorded
    		 1 3 4
    IIH Symptoms
    Visual loss
    Units: Subjects
        Visual loss
    		 4 8 12
        No visual loss
    		 13 6 19
    IIH Symptoms
    Pulsatile tinnitus
    Units: Subjects
        Pulsatile tinnitus
    		 12 13 25
        No Pulsatile tinnitus
    		 5 1 6
    IIH Symptoms
    Diplopia
    Units: Subjects
        Diplopia
    		 7 5 12
        No Diplopia
    		 10 9 19
    IIH Symptoms
    Transient visual obscurations
    Units: Subjects
        Transient visual obscurations
    		 6 6 12
        No Transient visual obscurations
    		 11 8 19
    Opening LP pressure, cmCSF
    Units: cmCSF
        arithmetic mean (standard deviation)
    		 33.7 ( 6.3 ) 32.7 ( 4.8 ) -
    Weight, kg
    Units: kilograms
        arithmetic mean (standard deviation)
    		 97.9 ( 21.3 ) 108.4 ( 42.3 ) -
    BMI (weight (kg)/ height (m2)
    Units: kg/m2
        arithmetic mean (standard deviation)
    		 37.3 ( 7.2 ) 41.2 ( 16.6 ) -
    HIT-6 score
    Units: HIT-6
        arithmetic mean (standard deviation)
    		 63.8 ( 8.2 ) 63.4 ( 8.1 ) -
    Perimetric mean deviation
    Worst eye only
    Units: dB
        arithmetic mean (standard deviation)
    		 -6.1 ( 5.4 ) -3.4 ( 6.8 ) -
    Log visual acuity
    Worst eye only
    Units: LVA
        arithmetic mean (standard deviation)
    		 0.08 ( 0.23 ) 0.13 ( 0.22 ) -
    Log contrast sensitivity
    Worst eye only
    Units: LCS
        arithmetic mean (standard deviation)
    		 1.63 ( 0.22 ) 1.63 ( 0.16 ) -
    OCT, thickness in μm
    Average retinal nerve fibre layer, worst eye only
    Units: μm
        arithmetic mean (standard deviation)
    		 152 ( 68.7 ) 158.4 ( 83 ) -
    OCT, thickness in μm
    Maximum retinal nerve fibre, worst eye only
    Units: μm
        arithmetic mean (standard deviation)
    		 320.2 ( 117.2 ) 290 ( 102.4 ) -
    Average Frisén grading
    Worst eye only
    Units: Frisén grading
        arithmetic mean (standard deviation)
    		 2.19 ( 1.17 ) 2.27 ( 0.9 ) -

    End points

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    End points reporting groups
    Reporting group title
    AZD4017
    Reporting group description
    		 An oral selective 11β-HSD1 inhibitor, AZD4017, at 400mg twice daily for 12 weeks.

    Reporting group title
    Placebo arm
    Reporting group description
    		 A matched placebo 400mg twice daily for 12 weeks.

    Primary: Primary clinical outcome, mean ICP at 12 weeks

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    End point title
    		 Primary clinical outcome, mean ICP at 12 weeks
    End point description
    End point type
    Primary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 AZD4017 Placebo arm
    Number of subjects analysed
    16 [1]
    12 [2]
    Units: cmCSF
        arithmetic mean (standard deviation)
    29.7 ( 5.2 )
    31.3 ( 6.7 )
    Notes
    [1] - 1 participant unable to complete LP for ICP at week 12
    [2] - 2 participants withdrawn before timepoint
    Statistical analysis title
    		 Primary outcome
    Statistical analysis description
    The primary outcome is to examine the effect of AZD4017 on ICP, as measured by lumbar puncture in cmCSF, from baseline to 12 weeks. The primary outcome measure is the difference in ICP at 12 weeks. Analysis is by intention-to-treat. A linear regression model will be used to compare the ICP at 12 weeks between the two arms, adjusting for baseline ICP.
    Comparison groups
    Placebo arm v AZD4017
    Number of subjects included in analysis
    28
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [3]
    P-value
    		 = 0.2
    Method
    		 Regression, Linear
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -2.8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -7.1
         upper limit
    		 1.5
    Variability estimate
    Standard deviation
    Notes
    [3] - Negative values in the adjusted mean difference between treatment arms favour AZD4017.

    Secondary: Secondary clinical outcome: Log Visual Acuity

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    End point title
    		 Secondary clinical outcome: Log Visual Acuity
    End point description
    Worst eye only
    End point type
    Secondary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 AZD4017 Placebo arm
    Number of subjects analysed
    16
    12
    Units: LogMAR
        arithmetic mean (standard deviation)
    0.06 ( 0.15 )
    0.09 ( 0.18 )
    Statistical analysis title
    		 Visual acuity
    Comparison groups
    AZD4017 v Placebo arm
    Number of subjects included in analysis
    28
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [4]
    P-value
    		 = 0.5
    Method
    		 Regression, Linear
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.03
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.12
         upper limit
    		 0.07
    Variability estimate
    Standard deviation
    Notes
    [4] - Negative values in the adjusted mean difference between treatment arms favour AZD4017.

    Secondary: Secondary clinical outcome: Log contrast sensitivity

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    End point title
    		 Secondary clinical outcome: Log contrast sensitivity
    End point description
    worst eye only
    End point type
    Secondary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 AZD4017 Placebo arm
    Number of subjects analysed
    13
    10
    Units: LCS
        arithmetic mean (standard deviation)
    1.65 ( 0.15 )
    1.66 ( 0.12 )
    Statistical analysis title
    		 Log Contrast Sensitivity
    Comparison groups
    AZD4017 v Placebo arm
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [5]
    P-value
    		 = 0.7
    Method
    		 Regression, Linear
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.02
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.15
         upper limit
    		 0.11
    Variability estimate
    Standard deviation
    Notes
    [5] - Negative values in the adjusted mean difference between treatment arms favour AZD4017.

    Secondary: Secondary clinical outcome: Perimetric mean deviation

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    End point title
    		 Secondary clinical outcome: Perimetric mean deviation
    End point description
    worst eye only
    End point type
    Secondary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 AZD4017 Placebo arm
    Number of subjects analysed
    17
    12
    Units: dB
        arithmetic mean (standard deviation)
    -3.4 ( 3.2 )
    -2.2 ( 3.1 )
    Statistical analysis title
    		 Perimetric mean deviation
    Comparison groups
    AZD4017 v Placebo arm
    Number of subjects included in analysis
    29
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [6]
    P-value
    		 = 0.8
    Method
    		 Regression, Linear
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2
         upper limit
    		 2.7
    Variability estimate
    Standard deviation
    Notes
    [6] - Negative values in the adjusted mean difference between treatment arms favour AZD4017.

    Secondary: Secondary clinical outcome: OCT retinal nerve fibre layer average

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    End point title
    		 Secondary clinical outcome: OCT retinal nerve fibre layer average
    End point description
    Worst eye only
    End point type
    Secondary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 AZD4017 Placebo arm
    Number of subjects analysed
    15
    11
    Units: μm
        arithmetic mean (standard deviation)
    139.7 ( 56.3 )
    143.2 ( 78.7 )
    Statistical analysis title
    		 OCT RNFL average
    Comparison groups
    AZD4017 v Placebo arm
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [7]
    P-value
    		 = 1
    Method
    		 Regression, Linear
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -34
         upper limit
    		 34.1
    Variability estimate
    Standard deviation
    Notes
    [7] - Negative values in the adjusted mean difference between treatment arms favour AZD4017.

    Secondary: Secondary clinical outcome: OCT retinal nerve fibre layer maxium

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    End point title
    		 Secondary clinical outcome: OCT retinal nerve fibre layer maxium
    End point description
    Worst eye only
    End point type
    Secondary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 AZD4017 Placebo arm
    Number of subjects analysed
    15
    11
    Units: μm
        arithmetic mean (standard deviation)
    305.5 ( 122.3 )
    277 ( 133.1 )
    Statistical analysis title
    		 OCT RNFL maximum
    Comparison groups
    AZD4017 v Placebo arm
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [8]
    P-value
    		 = 0.9
    Method
    		 Regression, Linear
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -4.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -68.1
         upper limit
    		 59.1
    Variability estimate
    Standard deviation
    Notes
    [8] - Negative values in the adjusted mean difference between treatment arms favour AZD4017.

    Secondary: Secondary clinical outcome: Average Frisén grading

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    End point title
    		 Secondary clinical outcome: Average Frisén grading
    End point description
    Worst eye only
    End point type
    Secondary
    End point timeframe
    Baseline to 12 weeks
    End point values
    		 AZD4017 Placebo arm
    Number of subjects analysed
    16
    12
    Units: Frisén grading
        arithmetic mean (standard deviation)
    1.56 ( 0.96 )
    2.25 ( 0.87 )
    Statistical analysis title
    		 Average Frisén grading
    Statistical analysis description
    Negative values in the adjusted mean difference between treatment arms favour AZD4017.
    Comparison groups
    AZD4017 v Placebo arm
    Number of subjects included in analysis
    28
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [9]
    P-value
    		 = 0.06
    Method
    		 Regression, Linear
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.7
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.4
         upper limit
    		 0.03
    Variability estimate
    Standard deviation
    Notes
    [9] - Negative values in the adjusted mean difference between treatment arms favour AZD4017.

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All AEs and SAEs were reported from the signing of the consent form to the end of the follow-up period at week 16. SAEs were to be reported within 24 hours of the site becoming aware of it.
    Adverse event reporting additional description
    AEs were collected whether or not related to the IMP.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    14
    Reporting groups
    Reporting group title
    AZD4017
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 AZD4017 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 14 (7.14%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Nervous system disorders
    Deterioration of IIH symptoms
    Additional description: Expected progression of condition
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 14 (7.14%)
         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
    		 AZD4017 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    16 / 17 (94.12%)
    11 / 14 (78.57%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Neoplasia
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Cardiac disorders
    Cardiovascular
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 14 (7.14%)
         occurrences all number
    1
    1
    Nervous system disorders
    Neurological
         subjects affected / exposed
    7 / 17 (41.18%)
    3 / 14 (21.43%)
         occurrences all number
    18
    6
    General disorders and administration site conditions
    Genito-urinary
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 14 (7.14%)
         occurrences all number
    2
    1
    Allergies
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 14 (0.00%)
         occurrences all number
    1
    0
    Other
    Additional description: Reasons for other included: tiredness, hot sweats, flu like symptoms, disrupted sleep, toothache/infection, breast pain, mentrual problems for more than 3 weeks, mouth ulcers, a cold, transient nausea, and height headaches.
         subjects affected / exposed
    7 / 17 (41.18%)
    5 / 14 (35.71%)
         occurrences all number
    9
    6
    Ear and labyrinth disorders
    Eyes, ear, nose, Throat
         subjects affected / exposed
    12 / 17 (70.59%)
    6 / 14 (42.86%)
         occurrences all number
    22
    14
    Immune system disorders
    Immunological
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 14 (7.14%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Gastrointestinal
         subjects affected / exposed
    8 / 17 (47.06%)
    3 / 14 (21.43%)
         occurrences all number
    22
    3
    Respiratory, thoracic and mediastinal disorders
    Respiratory
         subjects affected / exposed
    4 / 17 (23.53%)
    3 / 14 (21.43%)
         occurrences all number
    4
    3
    Skin and subcutaneous tissue disorders
    Dermatological
         subjects affected / exposed
    2 / 17 (11.76%)
    5 / 14 (35.71%)
         occurrences all number
    4
    8
    Psychiatric disorders
    Psychological
         subjects affected / exposed
    4 / 17 (23.53%)
    4 / 14 (28.57%)
         occurrences all number
    8
    8
    Endocrine disorders
    Endocrine
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 14 (7.14%)
         occurrences all number
    1
    1
    Musculoskeletal and connective tissue disorders
    Musculoskeletal
         subjects affected / exposed
    6 / 17 (35.29%)
    7 / 14 (50.00%)
         occurrences all number
    13
    10

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Nov 2014
    Substantial Amendment 1: To improve recruitment: Introducing patient compensation; switching subset of follow up visits to telephone visits to ease patient burden.
    13 Aug 2015
    Substantial Amendment 2: Change from single- to multi-centre trial; Removal of eligibility criteria requiring patients to be within 6 months of confirmed IIH diagnosis.
    26 Oct 2015
    Substantial Amendment 3: Correction of stratification text in randomisation section of protocol to reflect changing from single- to multi-centre trial.
    17 Nov 2015
    Substantial Amendment 4: Clarification of safety bloods required and their reporting/review timelines
    07 Jul 2016
    Substantial Amendment 5: Clarification to IIH symptom recording
    04 Jan 2017
    Substantial Amendment 6: Change to statistical analysis proposed in protocol (from change over 12 weeks to mean difference between arms)

    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.
    The pre-print of the results has not been peer-reviewed as of July 2019.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/28923789
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