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    Clinical Trial Results:
    Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients

    Summary
    EudraCT number
    		 2013-003228-35
    Trial protocol
    		 ES  
    Global end of trial date
    23 Jul 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    02 Jul 2021
    First version publication date
    02 Jul 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    18072013
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    VHIR
    Sponsor organisation address
    Passeig Vall Hebron 119-129, Barcelona, Spain, 08035
    Public contact
    Study coordinator, VHIR, +34 934893166, nmartin@vhebron.net
    Scientific contact
    Study coordinator, VHIR, +34 934893166, joaquin.lopez.soriano@vhir.org
    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
    23 Jul 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Jul 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Jul 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of the study is to assess the efficacy of 0.55% cysteamine formulated in viscous solution compared with saline solution at the same dose, in corneal crystal deposition.
    Protection of trial subjects
    Patients and parents were trained and informed about drop administration and possible adverse effects. Since there was no changes in the administered drug, no other measures were needed to implement.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    15 Nov 2013
    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
    Spain: 5
    Worldwide total number of subjects
    5
    EEA total number of subjects
    5
    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)
    2
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 5
    Number of subjects completed
    		 5

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

    Arms
    Arm title
    		 Viscous eyedrops
    Arm description
    		 Comparison of cysteamine saline solution versus viscous solution
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mercaptamine
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Ear/eye/nasal drops, solution
    Routes of administration
    Ophthalmic use
    Dosage and administration details
    0.55% viscous solution of cysteamine according to previous prescription for 3 months. Followed by 0.55% viscous solution of cysteamine four times a day for 3 months

    Number of subjects in period 1
    		Viscous eyedrops
    Started
    5
    Completed
    5

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cysteamine viscous solutions
    Reporting group description
    		 -

    Reporting group values
    		 Cysteamine viscous solutions Total
    Number of subjects
    		 5 5
    Age categorical
    Units: Subjects
        children 5-17y
    		 5 5
    Gender categorical
    Units: Subjects
        Female
    		 1 1
        Male
    		 4 4
    Cystinosis disease
    Cystinosis diagnostic
    Units: Subjects
        Disease
    		 5 5
    Subject analysis sets

    Subject analysis set title
    Efficiency and safety of cysteamine drops
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Children with cystinosis

    Subject analysis sets values
    		 Efficiency and safety of cysteamine drops
    Number of subjects
    5
    Age categorical
    Units: Subjects
        children 5-17y
    5
    Age continuous
    Units:
        
    ±
    Gender categorical
    Units: Subjects
        Female
    1
        Male
    4
    Cystinosis disease
    Cystinosis diagnostic
    Units: Subjects
        Disease
    5

    End points

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    End points reporting groups
    Reporting group title
    Viscous eyedrops
    Reporting group description
    		 Comparison of cysteamine saline solution versus viscous solution

    Subject analysis set title
    Efficiency and safety of cysteamine drops
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Children with cystinosis

    Primary: Corneal cystine crystal deposits

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    End point title
    		 Corneal cystine crystal deposits [1]
    End point description
    End point type
    Primary
    End point timeframe
    At the end of 6 months treatment
    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: 0.55% cysteamine viscous solution showed similar effects on corneal cistine crystals than saline solution. No changes were observed by modyfing frequency of instillations. Low number of enrolled patients limits the conclusions of the study. Collaboration of patients was a problem to obtain objective results
    End point values
    		 Viscous eyedrops
    Number of subjects analysed
    5
    Units: Number of crystals
    5
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    During the extension of the study
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    2
    Reporting groups
    Reporting group title
    Total adverse events
    Reporting group description
    		 -

    Serious adverse events
    		 Total adverse events
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 5 (40.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Nervous system disorders
    Intracranial pressure increased
         subjects affected / exposed
    2 / 5 (40.00%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Transplant dysfunction
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Total adverse events
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    Eye disorders
    Itching
         subjects affected / exposed
    5 / 5 (100.00%)
         occurrences all number
    5
    Vision blurred
         subjects affected / exposed
    3 / 5 (60.00%)
         occurrences all number
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Jan 2015
    Dr Marisol Lopez Moreno was included as researcher in the study
    12 Jan 2015
    Confocal microscopy was done at Hospital Clínic (barcelona) instead of Casa Maternitat because the equipment was moved

    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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    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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