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    Clinical Trial Results:
    Single-center, double-blind, randomized, placebo-controlled, 2-period/2-treatment crossover study investigating the effect of miglustat on the nasal potential difference in patients with cystic fibrosis homozygous for the ΔF508 mutation

    Summary
    EudraCT number
    		 2006-002049-35
    Trial protocol
    		 ES  
    Global end of trial date
    22 Feb 2008

    Results information
    Results version number
    		 v2(current)
    This version publication date
    07 Nov 2019
    First version publication date
    06 Aug 2015
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    Change of Sponsor

    Trial information

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    Trial identification
    Sponsor protocol code
    AC-056-201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Idorsia Pharmaceuticals Ltd
    Sponsor organisation address
    Hegenheimermattweg 91, Allschwil, Switzerland, 4123
    Public contact
    Clinical Trials Disclosure Desk, Idorsia Pharmaceuticals Ltd, clinical-trials-disclosure@idorsia.com
    Scientific contact
    Clinical Trials Disclosure Desk, Idorsia Pharmaceuticals Ltd, clinical-trials-disclosure@idorsia.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
    12 Feb 2009
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    22 Feb 2008
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Feb 2008
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To demonstrate that miglustat restores the function of the cystic fibrosis transmembrane conductance regulator (CFTR) in patients with cystic fibrosis homozygous for the ΔF508 mutation, as reflected in nasal potential difference (NPD)
    Protection of trial subjects
    Prior to the start of the trial the study center consulted an Independent Ethics Committee (IEC), i.e., a review panel that was responsible for ensuring the protection of the rights, safety, and well-being of human patients involved in a clinical investigation. The sponsor ensured that the IEC consulted was adequately constituted to provide assurance of that protection, and maintained a list of committee members and their qualifications. The protocol and any material provided to the patient (such as a subject information sheet or description of the study used to obtain informed consent) were reviewed and approved by the appropriate IEC before the study was started. This study was conducted in full conformance with the principles of the ‘Declaration of Helsinki’ and with the laws and regulations of the country in which the research was conducted. Both Actelion and the investigator had the right to terminate the study at any time, and in such a case, were responsible for protecting the patients’ interests. Written informed consent was obtained from each individual participating in the study prior to any study procedure and after adequate explanation of the aims, methods, objectives, and potential hazards of the study. It was made clear to each patient that he or she was completely free to refuse to enter the study, or to withdraw from it at any time for any reason. A description of any incentives to participate in the study was provided in the informed consent form.
    Background therapy
    		 Miglustat or placebo was given on top of standard care. Restrictions were applied for other investigational drugs and/or therapies, e.g., gene therapy.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    06 Nov 2007
    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: 3
    Worldwide total number of subjects
    3
    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)
    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
    		 One investigational center in Spain. The study was terminated prematurely after only 3 patients had participated in the study. One reason for premature study termination was low recruitment.

    Pre-assignment
    Screening details
    		 Screening examinations were done within 3 weeks and 3 days prior to the first administration of study medication.

    Period 1
    Period 1 title
    Baseline Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst
    Blinding implementation details
    The investigator and study staff, the patients, monitors, and the sponsor staff remained blinded to the treatment until study closure. The investigational drug and its matching placebo were indistinguishable and all patient kits were packaged in the same way.

    Arms
    Arm title
    		 Miglustat 200 mg t.i.d.
    Arm description
    		 Miglustat 200 mg t.i.d.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Miglustat 200 mg t.i.d.
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Miglustat 200 mg t.i.d. capsules, oral use

    Number of subjects in period 1
    		Miglustat 200 mg t.i.d.
    Started
    3
    Completed
    3
    Period 2
    Period 2 title
    Period 1
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst
    Blinding implementation details
    The investigator and study staff, the patients, monitors, and the sponsor staff remained blinded to the treatment until study closure. The investigational drug and its matching placebo were indistinguishable and all patient kits were packaged in the same way.

    Arms
    Arm title
    		 Miglustat 200 mg t.i.d.
    Arm description
    		 Miglustat 200 mg t.i.d. for 7 days
    Arm type
    		 Experimental

    Investigational medicinal product name
    Miglustat 200 mg t.i.d.
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Miglustat 200 mg t.i.d. capsules, oral use

    Number of subjects in period 2
    		Miglustat 200 mg t.i.d.
    Started
    3
    Completed
    3
    Period 3
    Period 3 title
    Period 2
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst
    Blinding implementation details
    The investigator and study staff, the patients, monitors, and the sponsor staff remained blinded to the treatment until study closure. The investigational drug and its matching placebo were indistinguishable and all patient kits were packaged in the same way.

    Arms
    Arm title
    		 Placebo
    Arm description
    		 Placebo 200 mg t.i.d.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo 200 mg t.i.d.
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Matching placebo 200 mg t.i.d. capsules, oral use

    Number of subjects in period 3
    		Placebo
    Started
    3
    Completed
    2
    Not completed
    1
         Consent withdrawn by subject
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Baseline Period
    Reporting group description
    		 Not applicable

    Reporting group values
    		 Baseline Period Total
    Number of subjects
    		 3
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 14.3 ± 4 -
    Gender categorical
    Units:
        Female
    		 1 1
        Male
    		 2 2
    Subject analysis sets

    Subject analysis set title
    All-treated set
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    This analysis set includes all randomized patients who received study drug.

    Subject analysis sets values
    		 All-treated set
    Number of subjects
    3
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    14.3 ± 4
    Gender categorical
    Units:
        Female
    1
        Male
    2

    End points

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    End points reporting groups
    Reporting group title
    Miglustat 200 mg t.i.d.
    Reporting group description
    		 Miglustat 200 mg t.i.d.
    Reporting group title
    Miglustat 200 mg t.i.d.
    Reporting group description
    		 Miglustat 200 mg t.i.d. for 7 days
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo 200 mg t.i.d.

    Subject analysis set title
    All-treated set
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    This analysis set includes all randomized patients who received study drug.

    Primary: Not applicable

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    End point title
    		 Not applicable [1]
    End point description
    Not applicable
    End point type
    Primary
    End point timeframe
    Not applicable
    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: Not applicable.
    End point values
    		 Miglustat 200 mg t.i.d. All-treated set
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: Not applicable
    Notes
    [2] - Not applicable
    [3] - Not applicable
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events from start of Period 1 (Day 1) to end of study (Day 28, Period 2). Serious adverse events from Day -21 until Day 1 (if related to study-mandated procedures) and from Day 1 to Day 56 (Follow up).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    9.0
    Reporting groups
    Reporting group title
    Overall Period
    Reporting group description
    		 Period 1 and 2

    Serious adverse events
    		 Overall Period
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 3 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Overall Period
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 3 (33.33%)
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2

    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.
    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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