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    Clinical Trial Results:
    Treatment of congenital nephrogenic diabetes insipidus with a guanylate cyclase stimulator, riociguat or a phosphodiesterase type 5 inhibitor, sildenafil

    Summary
    EudraCT number
    		 2016-001591-30
    Trial protocol
    		 DK  
    Global end of trial date
    06 Jun 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Jun 2020
    First version publication date
    04 Jun 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    S-20150201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Claus Bistrup
    Sponsor organisation address
    J.B. Winsløws Vej 4, Odense C, Denmark, 5000
    Public contact
    Dept. of Nephrology, Odense University Hospital, 45 65411106, ode.y@rsyd.dk
    Scientific contact
    Dept. of Nephrology, Odense University Hospital, 45 65411106, ode.y@rsyd.dk
    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
    01 Nov 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    06 Jun 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Jun 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The aim of the study is to test the hypothesis that treatment with riociguat or sildenafil in patients with NDI caused by AVP2R mutation improves the symptoms by reducing diuresis
    Protection of trial subjects
    Patients were hospitalized for the first two days of each intervention to enable close monitoring of side-effects. For the rest of each intervention, patients had daily check-ups where symptoms and side-effects were monitored and handled.
    Background therapy
    		 None
    Evidence for comparator
    		 Not applicable. The effect of the two interventions was evaluated by comparing baseline values to similar values after each intervention.
    Actual start date of recruitment
    20 Feb 2019
    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
    Denmark: 2
    Worldwide total number of subjects
    2
    EEA total number of subjects
    2
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    2
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The two subjects were included on the 20th of February 2019 after oral and written information. Written informed consent was obtained before any study related procedures.

    Pre-assignment
    Screening details
    		 The two subjects fulfilling the elegibility criteria were recruited from the outpatient clinic at the Department of Nephrology, Odense University Hospital.

    Period 1
    Period 1 title
    Sildenafil
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Sildenafil
    Arm description
    		 Intervention
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sildenafil
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    50 mg x 3/day for 7 days

    Number of subjects in period 1
    		Sildenafil
    Started
    2
    Completed
    2
    Period 2
    Period 2 title
    Riociguat
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Riociguat
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Riociguat
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 mg 3 times per day

    Number of subjects in period 2
    		Riociguat
    Started
    2
    Completed
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Sildenafil
    Reporting group description
    		 -

    Reporting group values
    		 Sildenafil Total
    Number of subjects
    		 2 2
    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)
    		 2 2
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        median (full range (min-max))
    		 28 (28 to 28) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0
        Male
    		 2 2
    Bodyweight
    Units: kg
        arithmetic mean (full range (min-max))
    		 143.5 (142.4 to 144.6) -
    Height
    Units: cm
        arithmetic mean (full range (min-max))
    		 182.5 (182.5 to 182.5) -

    End points

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    End points reporting groups
    Reporting group title
    Sildenafil
    Reporting group description
    		 Intervention
    Reporting group title
    Riociguat
    Reporting group description
    		 -

    Primary: Diureses per hour

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    End point title
    		 Diureses per hour [1]
    End point description
    End point type
    Primary
    End point timeframe
    During water deprivation test
    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: N=2, therefor, no statistic analysis has been performed
    End point values
    		 Sildenafil Riociguat
    Number of subjects analysed
    2
    2
    Units: mL
        arithmetic mean (full range (min-max))
    1103.67 (809 to 1685)
    971 (764 to 1335)
    No statistical analyses for this end point

    Primary: water intake

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    End point title
    		 water intake [2]
    End point description
    End point type
    Primary
    End point timeframe
    During intervention periode
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Water intake was not measured. No statistical analysis performed.
    End point values
    		 Sildenafil Riociguat
    Number of subjects analysed
    2 [3]
    2 [4]
    Units: mL
        arithmetic mean (full range (min-max))
    0 (0 to 0)
    0 (0 to 0)
    Notes
    [3] - Not measured
    [4] - Not measured
    No statistical analyses for this end point

    Post-hoc: Diureses per 24h

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    End point title
    		 Diureses per 24h
    End point description
    End point type
    Post-hoc
    End point timeframe
    During intervention periode
    End point values
    		 Sildenafil Riociguat
    Number of subjects analysed
    2
    2
    Units: mL
        arithmetic mean (full range (min-max))
    10817.33 (8480 to 13320)
    13177.83 (9240 to 18140)
    No statistical analyses for this end point

    Post-hoc: Urinary osmolality

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    End point title
    		 Urinary osmolality
    End point description
    End point type
    Post-hoc
    End point timeframe
    During intervention period
    End point values
    		 Sildenafil Riociguat
    Number of subjects analysed
    2
    2
    Units: mosm/kg
        arithmetic mean (full range (min-max))
    92.58 (84 to 99)
    88.08 (75 to 98)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    During interventions
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 SNOMED CT
    Dictionary version
    2019AB
    Reporting groups
    Reporting group title
    Sildnafil
    Reporting group description
    		 -

    Reporting group title
    Riociguat
    Reporting group description
    		 -

    Serious adverse events
    		 Sildnafil Riociguat
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Sildnafil Riociguat
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    2 / 2 (100.00%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    2
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 2 (100.00%)
    1 / 2 (50.00%)
         occurrences all number
    2
    2
    Gastrointestinal disorders
    Gastritis
         subjects affected / exposed
    2 / 2 (100.00%)
    1 / 2 (50.00%)
         occurrences all number
    3
    1
    Abdominal discomfort
    Additional description: Lower abdomen
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Myalgia
    Additional description: Slight
         subjects affected / exposed
    2 / 2 (100.00%)
    0 / 2 (0.00%)
         occurrences all number
    2
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

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

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