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    Clinical Trial Results:
    A Phase IIA Prospective, Single-Centre, Open Label Clinical Trial to Evaluate the Safety, Tolerability and Pharmacodynamic Effects of Ambroxol in Patients with Parkinson Disease: Ambroxol in Disease Modification in Parkinson Disease

    Summary
    EudraCT number
    		 2015-002571-24
    Trial protocol
    		 GB  
    Global end of trial date
    26 Apr 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    24 Jul 2019
    First version publication date
    24 Jul 2019
    Other versions
    Summary report(s)
    		 AiM-PD Summary Upload

    Trial information

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    Trial identification
    Sponsor protocol code
    15/0118
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02941822
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Univeristy College London
    Sponsor organisation address
    Gower Street, London, United Kingdom, WC1E 6BT
    Public contact
    Joshua Elflein, Leonard Wolfson Experimental Neurology Centre Clinical Research Facility , +44 02034484541, joshua.elflein@ucl.ac.uk
    Scientific contact
    Joshua Elflein, Leonard Wolfson Experimental Neurology Centre Clinical Research Facility , +44 2034484541, joshua.elflein@ucl.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
    26 Apr 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    26 Apr 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Apr 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    • To assess the central nervous system (CNS), cerebrospinal fluid (CSF) penetration and binding to GCase of ambroxol by the parameters outline (modulation of GCase activity & ambroxol level) at 5 intra-participant dose escalations from day 1 to day 186 at 60 mg TID (day 1-7), 120 mg TID (day 8-14), 180 mg TID (day 15-21), 300 mg TID (day 22-28) and 420 mg TID (day 29-186). • To assess the safety and tolerability of the Glucocerebrosidase (GCase) modulating chaperone ambroxol in Parkinson disease participants with and without Gaucher gene (GBA) mutation at 5 intra-participant dose escalations from day 1 to 186. • To measure the pharmacodynamic effects of ambroxol on GCase activity in blood and CSF following ambroxol oral administration at 5 intra-participant dose escalations from day 1 to 186. • To quantify the effect of ambroxol on biomarkers of Parkinson and neurodegeneration at 5 intra-participant dose escalations from day 1 to 186.
    Protection of trial subjects
    Data will be reported in accordance to the protocol and local reporting regulations (REC/MHRA) which is covered by Good Pharmacovigilance Practice. There will be regular Trial Management Group (TMG) which will meet regularly throughout the course of the study to assess the study progress and all AEs and the minutes will be accessed and recorded. The protocol also specifies criterias for the following: - IMP discontinuations - Participant stopping criteria - Participant Termination criteria The purpose of the TMG is to ensure participant safety is not compromised during the course of the study.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Jul 2016
    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
    United Kingdom: 24
    Worldwide total number of subjects
    24
    EEA total number of subjects
    24
    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)
    19
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This study was conducted at the Leonard Wolfson Experimental Neurology Centre Clinical Research Facility, which is based at the National Hospital for Neurology and Neurosurgery and is part of the University College London Hospitals NHS Foundation Trust. All participants recruited to this study had a confirmed diagnosis of Parkinson Disease.

    Pre-assignment
    Screening details
    		 Twenty participants to be recruited in total, 10 with a GBA positive status and 10 with a GBA negative status. Informed consent, Medical history, Physical and neurological examinations, Screening genotyping, Vital signs, Height and weight, ECG, pregnancy test, blood collection, Adverse event review, Concomitant medication review, etc.

    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    N/A

    Arms
    Arm title
    		 Interventional
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ambroxol hydrochloride
    Investigational medicinal product code
    Other name
    Ambrosan
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Participants will be instructed to self-administer 5 intra-participant dose escalations at 60 mg TID (day 1-7), 120 mg TID (day 8-14), 180 mg TID (day 15-21), 300 mg TID (day 22-28) and 420 mg TID (day 29-186). The study drug should be administered with or without a meal and rinsed down with sufficient amount of fluid for each daily morning, afternoon and evening in accordance to the instructions provided by the Investigator.

    Number of subjects in period 1
    		Interventional
    Started
    24
    Completed
    18
    Not completed
    6
         Consent withdrawn by subject
    3
         Screen Failures
    3

    Baseline characteristics

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    End points

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    End points reporting groups
    Reporting group title
    Interventional
    Reporting group description
    		 -

    Primary: To assess the central nervous system (CNS), cerebrospinal fluid (CSF) penetration and binding to GCase of ambroxol by the parameters outline (modulation of GCase activity & ambroxol level)

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    End point title
    		 To assess the central nervous system (CNS), cerebrospinal fluid (CSF) penetration and binding to GCase of ambroxol by the parameters outline (modulation of GCase activity & ambroxol level) [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline to Day 186
    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: Comprehensive information regarding the statistical analysis can be referenced in the attached trial summary.
    End point values
    		 Interventional
    Number of subjects analysed
    18
    Units: ng/mL
    156
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    All AEs assessed upon reporting.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21
    Frequency threshold for reporting non-serious adverse events: 5%
    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: All AEs reported in attached AiM-PD Trial Summary.

    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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