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    Clinical Trial Results:
    A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400 mg Or 1000 mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy

    Summary
    EudraCT number
    2016-000067-16
    Trial protocol
    GB  
    Global end of trial date
    04 Jan 2018

    Results information
    Results version number
    v1(current)
    This version publication date
    19 Jul 2018
    First version publication date
    19 Jul 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AMO-02-MD-2-001
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    AMO Pharma Ltd
    Sponsor organisation address
    Throwsters, The Street, Wonersh, Surrey, United Kingdom, GU5 0PF
    Public contact
    General enquiries, AMO Pharma Ltd, +44 01483 898 448, clinicaltrials@amo-pharma.com
    Scientific contact
    General enquiries, AMO Pharma Ltd, +44 01483 898 448, clinicaltrials@amo-pharma.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
    01 Feb 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Jan 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Jan 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To investigate the safety and tolerability between baseline and end-of-treatment of tideglusib in adolescent and adult subjects with congenital or juvenile-onset myotonic dystrophy
    Protection of trial subjects
    The study was conducted in accordance with ICH principles of Good Clinical Practice (GCP), as required by European Directive 2001/20/EC and 2005/28/EC and local rules relevant to the use of new therapeutic agents within the United Kingdom, and adherence to the general principles that have their origins in the Declaration of Helsinki. Consideration was made of the fact that this study required recruitment of patients from 12 years of age who had been diagnosed with Type 1 Myotonic Dystrophy (DM-1). DM-1 patients have varying phenotypes but congenital and juvenile-onset DM-1 patients often suffer from intellectual disability. To address this, the primary Informed Consent was to be given by the subject's parent/caregiver/spouse/Legally Authorised Representative (LAR). The subject was to provide assent or informed consent depending on their age and on the investigator’s assessment of the subject’s developmental level. This was documented prior to any protocol-related activities being conducted.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    20 Jul 2016
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 16
    Worldwide total number of subjects
    16
    EEA total number of subjects
    16
    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)
    5
    Adults (18-64 years)
    11
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    Subjects were to be male or female aged 12 to 45 years with a diagnosis of genetically confirmed congenital or juvenile-onset type 1 myotonic dystrophy. Subjects were to have a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2) and were to be ambulatory and able to complete the 10-metre walk/run test.

    Pre-assignment period milestones
    Number of subjects started
    16
    Number of subjects completed
    16

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Single blind [1]
    Roles blinded
    Subject, Carer
    Blinding implementation details
    The study was single blinded, where the Sponsor/delegate and the Investigator site knew what treatment the subjects were receiving at each point during the study, but the subject and parent/caregiver/spouse/LAR were blinded as to whether the subject was receiving active or placebo. It is noted that all subjects and parents/caregivers/spouses/LARs were informed that the study subjects would receive the study drug at some time during the study.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Cohort 1
    Arm description
    Subjects were given a placebo run in (weeks -2 to 0) followed by tideglusib 1000 mg for 12 weeks (weeks 0 to 12)
    Arm type
    Experimental

    Investigational medicinal product name
    Tideglusib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    IMP was administered orally once daily by dispersing two packets in water. The product was to be consumed immediately after constitution. The product could be administered via a percutaneous endoscopic gastrostomy (PEG) if applicable. The IMP was to be administered in an overnight fasted state; food intake was to be prevented for at least 1 hour after taking the medication. The whole content of the packets was added to 100 mL of tap water in a glass and mixed with a spoon until dispersed. After drinking this constituted dispersion, another 50 mL of tap water was added to the glass and stirred with a spoon to disperse any remnants of the powder in the glass before drinking.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Administration of placebo is as per active

    Arm title
    Cohort 2
    Arm description
    Subjects were given a placebo run in (weeks -2 to 0) followed by tideglusib 400 mg for 12 weeks (weeks 0 to 12)
    Arm type
    Experimental

    Investigational medicinal product name
    Tideglusib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    IMP was administered orally once daily by dispersing one packet in water. The product was to be consumed immediately after constitution. The product could be administered via a percutaneous endoscopic gastrostomy (PEG) if applicable. The IMP was to be administered in an overnight fasted state; food intake was to be prevented for at least 1 hour after taking the medication. The whole content of the packet was added to 100 mL of tap water in a glass and mixed with a spoon until dispersed. After drinking this constituted dispersion, another 50 mL of tap water was added to the glass and stirred with a spoon to disperse any remnants of the powder in the glass before drinking.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Administration of placebo is as per active

    Notes
    [1] - The number of roles blinded appears inconsistent with a single blinded trial. It is expected that there will be one role blinded in a single blind trial.
    Justification: Due to the patient population, both the patient and carer were blinded to ensure results remained objective.
    Number of subjects in period 1
    Cohort 1 Cohort 2
    Started
    8
    8
    Completed
    8
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall trial (overall period)
    Reporting group description
    -

    Reporting group values
    Overall trial (overall period) Total
    Number of subjects
    16 16
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    5 5
        Adults (18-64 years)
    11 11
    Gender categorical
    Units: Subjects
        Female
    6 6
        Male
    10 10

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    Subjects were given a placebo run in (weeks -2 to 0) followed by tideglusib 1000 mg for 12 weeks (weeks 0 to 12)

    Reporting group title
    Cohort 2
    Reporting group description
    Subjects were given a placebo run in (weeks -2 to 0) followed by tideglusib 400 mg for 12 weeks (weeks 0 to 12)

    Primary: Safety

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    End point title
    Safety [1]
    End point description
    End point type
    Primary
    End point timeframe
    From start of active treatment to end of study
    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: An overall summary of TEAEs was produced, however no formal statistical analysis was required per protocol.
    End point values
    Cohort 1 Cohort 2
    Number of subjects analysed
    8
    8
    Units: Subjects
        Total no. of TEAEs
    8
    6
        Total no. of serious TEAEs
    0
    0
        Total no. TEAEs leading to discontinuation of IMP
    0
    0
        Total no. TEAEs leading to withdrawal
    0
    0
        Total no. TEAEs leading to death
    0
    0
        Severity: Mild
    3
    1
        Severity: Moderate
    5
    4
        Severity: Severe
    0
    1
        Unrelated to active treatment
    6
    6
        Related to active treatment
    2
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From start of active treatment to end of study
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    19.1
    Reporting groups
    Reporting group title
    Cohort 1 - 1000 mg
    Reporting group description
    -

    Reporting group title
    Cohort 2 - 400 mg
    Reporting group description
    -

    Serious adverse events
    Cohort 1 - 1000 mg Cohort 2 - 400 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Cohort 1 - 1000 mg Cohort 2 - 400 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 8 (100.00%)
    6 / 8 (75.00%)
    Injury, poisoning and procedural complications
    Back injury
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Fall
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Joint dislocation
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Laceration
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Limb injury
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 8 (12.50%)
         occurrences all number
    1
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Nervous system cyst
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Somnolence
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 8 (12.50%)
    2 / 8 (25.00%)
         occurrences all number
    1
    2
    Diarrhoea
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    Abdominal pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Post-tussive vomiting
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Erythema
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Rash papular
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Axillary mass
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Myalgia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Vitamin D deficiency
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    2 / 8 (25.00%)
    3 / 8 (37.50%)
         occurrences all number
    2
    4
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 8 (12.50%)
         occurrences all number
    1
    1
    Subcutaneous abscess
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Jan 2017
    Protocol version 4.0 included the following changes: - Change to exclusion criterion no. 16 relating to HbA1c to re-define the exclusion levels using absolute values to avoid ambiguity and to remove the need for further HbA1c value to be normal at run-in as each HbA1c test is applicable to a 2-3 month period. The value taken at screening was sufficient to determine eligibility. HbA1c was no longer collected at run-in. - Clarification that a "prothrombin time test" would be completed as part of the clinical lab tests collected in the event of elevated liver function values occurring as detailed in the Discontinuation Criteria. Prothrombin time tests had been stated as being completed at every visit in the schedule of assessments but not consistently throughout the protocol. - Correction of blood volume required for the GSK3B biomarker analysis. It was not possible to source a 3mL sodium citrate tube to collect this sample, therefore a 4 mL sodium citrate tube, requiring a 4mL blood sample was provided in all relevant kits. -Updated information on PK analysis methodology -Inclusion of analysis to be performed once all subjects in cohort 1 had completed or discontinued from active treatment in the study -Addition of exclusion criterion no. 29 where subjects were not allowed to enter the study if their BMI was less than 14.0 kg/m2 or greater than 40.0 kg/m2 to ensure that subjects were not over or under exposed to study drug respectively. -Inclusion of a text field in the CRF to record pertinent information that was reported by the subject and/or caregiver and not captured elsewhere in the CRF, that supported the CGI-S and CGI-I rating. -Clarification of the description of the establishment of the DSMC

    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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