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    Clinical Trial Results:
    CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS)

    Summary
    EudraCT number
    		 2014-002596-28
    Trial protocol
    		 DE   GB  
    Global end of trial date
    20 Jul 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    31 Mar 2018
    First version publication date
    31 Mar 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PTC124-GD-024-MPS
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    PTC Therapeutics Inc.
    Sponsor organisation address
    100 Corporate Court, South Plainfield, United States, NJ 07080
    Public contact
    Joseph McIntosh, MD, PTC Therapeutics Inc., 1 908-912-9138,
    Scientific contact
    Joseph McIntosh, MD, PTC Therapeutics Inc., 1 908-912-9138,
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Sep 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Jul 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Jul 2017
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objectives of the studies were to: - Characterize the safety profile of ataluren in patients with nonsense mutation mucopolysaccharidosis type 1 (nmMPS 1), and - Evaluate the cerebrospinal fluid (CSF) and plasma pharmacokinetics (PK) of ataluren in patients with nmMPS 1.
    Protection of trial subjects
    This study was conducted in conformance with Good Clinical Practice standards and applicable country and/or local statutes and regulations regarding ethical committee review, informed consent, and the protection of human subjects participating in biomedical research.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Sep 2015
    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
    Germany: 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)
    2
    Adolescents (12-17 years)
    1
    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
    		 The study included a 14-day screening period.

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

    Arms
    Arm title
    		 "Non-treated Patients" (Period 1)
    Arm description
    		 All patients participated in treatment Period 1 and received 12 weeks of ataluren. Patients who were to be enrolled included: - patients on enzyme replacement therapy (ERT) at study entry (referred to as “ERT patients”), - patients not on ERT at study entry but who had previously received bone marrow transplantation/hematopoietic stem cell transplantation (BMT/HSCT) (referred to as “post-BMT/HSCT patients”), and - patients not on ERT at study entry and who had not previously received BMT/HSCT (referred to as “non-treated patients”). In this study, the 3 patients enrolled were in the "non-treated" arm, and no "ERT" or "post-BMT/HSCT" patients were enrolled.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ataluren
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Ataluren was administered 3 times a day: 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening; during 12 weeks.

    Number of subjects in period 1
    		"Non-treated Patients" (Period 1)
    Started
    3
    Completed
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    All patients
    Reporting group description
    		 -

    Reporting group values
    		 All patients Total
    Number of subjects
    		 3 3
    Age categorical
    Units: Subjects
        Children (2-11 years)
    		 2 2
        Adolescents (12-17 years)
    		 1 1
    Gender categorical
    Units: Subjects
        Female
    		 1 1
        Male
    		 2 2

    End points

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    End points reporting groups
    Reporting group title
    "Non-treated Patients" (Period 1)
    Reporting group description
    		 All patients participated in treatment Period 1 and received 12 weeks of ataluren. Patients who were to be enrolled included: - patients on enzyme replacement therapy (ERT) at study entry (referred to as “ERT patients”), - patients not on ERT at study entry but who had previously received bone marrow transplantation/hematopoietic stem cell transplantation (BMT/HSCT) (referred to as “post-BMT/HSCT patients”), and - patients not on ERT at study entry and who had not previously received BMT/HSCT (referred to as “non-treated patients”). In this study, the 3 patients enrolled were in the "non-treated" arm, and no "ERT" or "post-BMT/HSCT" patients were enrolled.

    Primary: Ataluren Cerebrospinal Fluid Concentrations

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    End point title
    		 Ataluren Cerebrospinal Fluid Concentrations [1]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    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: Descriptive statistics only due to the small number of patients in the study. General conclusions regarding the safety and PK of ataluren in patients with nmMPS 1 cannot be made. The results of this study indicate that ataluren crosses the blood-brain barrier.
    End point values
    		 "Non-treated Patients" (Period 1)
    Number of subjects analysed
    2
    Units: nanogram(s)/millilitre
    arithmetic mean (standard deviation)
        Pre-dose
    79.40 ( 87.116 )
    No statistical analyses for this end point

    Primary: Ataluren Plasma Concentrations

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    End point title
    		 Ataluren Plasma Concentrations [2]
    End point description
    End point type
    Primary
    End point timeframe
    Week 12
    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: Descriptive statistics only due to the small number of patients in the study. General conclusions regarding the safety and PK of ataluren in patients with nmMPS 1 cannot be made.
    End point values
    		 "Non-treated Patients" (Period 1)
    Number of subjects analysed
    3
    Units: microgram(s)/millilitre
    arithmetic mean (standard deviation)
        Pre-dose
    4.023 ( 2.4918 )
        Hour 2
    10.297 ( 1.3580 )
        Hour 4
    4.720 ( 1.2210 )
        Hour 6
    2.940 ( 0.4530 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline to Week 12
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.0
    Reporting groups
    Reporting group title
    Safety Population
    Reporting group description
    		 -

    Serious adverse events
    		 Safety Population
    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
    		 Safety Population
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    Investigations
    Weight increased
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Gastrointestinal disorders
    Flatulence
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Infections and infestations
    Rash pustular
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 3 (33.33%)
         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
    04 Dec 2015
    · Secondary Objectives and endpoints were modified to delete the urinary glycosaminoglycan (GAG) level assessed by the conventional method and the urine filter paper method. · Several inclusion and exclusion criteria were amended to ensure consistency throughout the protocol. · The schedule of assessments was updated. · The Metabolic Urinary GaG value was updated.

    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.
    PTC124-GD-024-MPS was terminated early due to low enrolment. Due to the small number of patients in the study, general conclusions regarding the safety and PK of ataluren in patients with nmMPS 1 cannot be made.
    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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