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    Summary
    EudraCT Number:2016-000067-16
    Sponsor's Protocol Code Number:AMO-02-MD-2-001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-05-13
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2016-000067-16
    A.3Full title of the trial
    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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to investigate how safe and effective tideglusib is, as treatment for adolescents and adults with myotonic dystrophy diagnosed before they were 12 years old.
    A.4.1Sponsor's protocol code numberAMO-02-MD-2-001
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAMO Pharma Ltd.
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAMO Pharma Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAMO Pharma Ltd
    B.5.2Functional name of contact pointGeneral enquiries
    B.5.3 Address:
    B.5.3.1Street AddressThrowsters, The Street
    B.5.3.2Town/ cityWonersh, Surrey
    B.5.3.3Post codeGU5 0PF
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+4401483 898 448
    B.5.6E-mailclinicaltrials@amo-pharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTideglusib
    D.3.4Pharmaceutical form Powder for oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTideglusib
    D.3.9.3Other descriptive name4-Benzyl-2-naphthalen-1-yl-1,2,4- thiadiazolidine-3,5-dione
    D.3.9.4EV Substance CodeSUB32020
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number1000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for oral suspension
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Treatment of adolescent and adult congenital and juvenile onset myotonic dystrophy
    E.1.1.1Medical condition in easily understood language
    Treatment of myotonic dystrophy in adults and adolescents
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.1
    E.1.2Level PT
    E.1.2Classification code 10068871
    E.1.2Term Myotonic dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main 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
    E.2.2Secondary objectives of the trial
    To investigate the blood pharmacokinetics of tideglusib in adolescent and adult subjects with congenital or juvenile-onset myotonic dystrophy.

    To investigate differences in outcomes reflective of efficacy, between baseline and end-of treatment, with tideglusib in adolescent and adult subjects with congenital or juvenile-onset myotonic dystrophy.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects under study must be adolescent or adults with a diagnosis of congenital or juvenile-onset type 1 myotonic dystrophy (DM-1). For the purposes of this study, the following definitions apply:
    Congenital: in addition to the genetic confirmation of DM-1, one or more of the following signs or symptoms was evident within the first week after birth:
    a) Hypotonia
    b) Generalized weakness
    c) Respiratory insufficiency
    d) Feeding difficulties
    e) Clubfoot or another musculoskeletal deformity

    Childhood/juvenile-onset: in addition to the genetic confirmation of DM-1, at least 2 signs or symptoms (not caused by another, unrelated condition) were evident prior to 12 years of age that can be clearly assigned to DM-1, for example:
    a) Muscle weakness
    b) Myotonia (delayed muscle relaxation)
    c) Difficulty using hands, including fine motor problems
    d) Excessive daytime sleepiness
    e) Problems with upper or lower gastrointestinal functioning
    f) Problems with concentration or focusing (including symptoms of attention-deficit/hyperactivity disorder)
    g) Learning difficulties (including dyslexia)

    2. Diagnosis must be genetically confirmed
    3. Subjects must be male or female aged 12 years to 45 years (the first 3 subjects in Cohort 1 must be ≥18 years)
    4. Subjects must have a Clinical Global Impression – Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2)
    5. Subjects must be ambulatory and able to complete the 10 metre walk/run test (splints allowed)
    6. Subject’s legally authorised representative (LAR) must provide written informed consent and there must be written consent or assent (as age applicable and developmentally appropriate) by the subject before any study-related procedures are conducted
    7. Subject’s caregiver must be willing and able to support subject’s participation for duration of study and if different from the LAR, must consent to this in writing
    E.4Principal exclusion criteria
    1. Non-ambulatory (full time) wheel chair user
    2. Receiving stimulant medication
    3. Receiving other medications/therapies not stable (changed) within 4 weeks prior to Run-in (V2)
    4. Medical illness or other concern which would cause investigator to conclude subjects will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
    5. Current enrolment in a clinical trial of an investigational drug or enrolment in a clinical trial of an investigational drug in the last 6 months
    6. Women of child bearing potential who are pregnant, lactating or not willing to use a protocol-defined acceptable* contraception method if sexually active and not surgically sterile
    7. Men, if engaged in sexual relations with a female of child-bearing potential, not using an acceptable contraceptive method if not surgically sterile
    8. Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication and impact the interpretability of the study results
    9. Current clinically significant (as determined by the investigator) cardiovascular, renal, hepatic, endocrine or respiratory disease
    10. Clinically significant heart disease (in the opinion of the investigator) or second or third degree heart block, atrial flutter, atrial fibrillation, ventricular arrhythmias, or is receiving medication for treatment of a cardiac arrhythmia
    11. Average QTcF value of >450msec at screening
    12. Kidney disease requiring ongoing treatment
    13. A history of chronic liver disease with current out of range value for ALT, clinically relevant hepatic steatosis or other clinical manifestations of ongoing liver disease
    14. Current ALT value > 2X the upper limit of the normal reference range at Screening or Run-in (V2) (may repeat to confirm)
    15. Current total bilirubin value greater than the upper limit of the normal reference range at Screening or Run-in (V2) (unless due to Gilbert’s syndrome) (may repeat to confirm)
    16. HbA1c values greater than 6% or 42.0mmol/mol at Screening (may repeat to confirm)
    17. TSH values outside of the normal reference range at Screening or Run-in (V2) (may repeat to confirm)
    18. Serum creatinine >150 umol/L and creatinine clearance ≤ 60 mL/m (according to Cockcroft-Gault formula) at Screening (may repeat to confirm)
    19. Clinical history of hepatitis, previous or current positive serological evidence for hepatitis B or C
    20. Serological evidence of Hepatitis A at Screening or in the 6 months preceding Screening
    21. A history of significant drug allergy (such as Steven-Johnson syndrome, anaphylaxis)
    22. A history of alcohol or substance use disorders
    23. Current malignancy or any history of malignancy except for surgically-cured skin cancer or pilomatricoma (benign tumor of the hair follicle that is associated with DM-1).
    24. Severe arthritis or other medical condition (besides DM-1) that would significantly impact ambulation
    25. Use within 4 weeks prior to baseline (V3) of strong CYP3A4 inhibitors e.g. clarithyromycin, telithromycin, ketoconazole, itarconazole, posaconazole, nefazadone, indinavir, ritonavir
    26. Concurrent use of drugs metabolised by CYP3A4 with a narrow therapeutic window e.g. warfarin and digitoxin
    27. Judged clinically to be at risk of suicide (suicidal ideation, severe depression, or other factors) over the last three months, as assessed by the Investigator.
    28. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry
    29. BMI of less than 14.0 kg/m2 or greater than 40.0 kg/m2 at Screening
    * Acceptable contraception is considered to be using one of the following birth control methods:
    • Surgical sterilisation of either the female subject in study (e.g., bilateral tubal ligation) or of her male partner (vasectomy with documented azoospermia) if he is the sole partner of that subject
    • Established hormonal contraception (implantable, patch, oral or intramuscular [IM]) administered for at least one month prior to study medication administration
    • An intrauterine device (IUD) or intrauterine system (IUS) with failure rate of less than 1% per year inserted by qualified physician at least one month prior to study medication administration
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of adverse events (AE), including Serious adverse events (SAE).

    The incidence of abnormal findings in objective assessments (e.g. laboratory values, ECGs, and vital signs, weight, physical examination, urinalysis) during the course of the study will also be assessed.
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs will be evaluated between baseline and Follow-up Visit, while SAEs will be evaluated between screening and the last clinic visit or until resolution, whenever possible.

    The incidence of abnormal findings in objective assessments (e.g. laboratory values, ECGs, and vital signs, weight, physical examination, urinalysis) during the course of the study will also be assessed.
    E.5.2Secondary end point(s)
    • Blood pharmacokinetics (PK) of tideglusib
    • 10 metre walk/run test (self-selected and fastest velocity)
    • Computerised handgrip myometer measure of grip strength and muscle relaxation time
    • Respiratory Forced vital capacity (FVC)
    • Clinical Global Impressions– Severity and –Improvement (CGI-S and CGI-I)
    • Actigraphy (measure daily averages of: number of bouts of activity, number of steps taken and energy expenditure )
    • Nine Hole Peg Test (NHPT)
    • Dual-energy X-ray absorptiometry (DXA) whole body scan of lean muscle mass (g) legs, arms, and total
    • Top 3 Concerns VAS (caregiver-completed and where possible, subject-completed also)
    • OSU Autism Rating Scale (OARS)
    • OSU Autism CGI
    • Clinician-completed Domain Specific Cause for Concern VAS: Myotonic Dystrophy
    • Peabody Picture Vocabulary Test
    • Biomarker - lymphocyte GSK3β levels and activity
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout study: Grip strength, Actigraphy (except for screening).
    Screening, run-in, baseline, week 6, EOT: 10 metre walk/run and Pulmonary function tests [(FVC) and at follow up]
    Screening, run-in, baseline, week 2, 8, EOT: Nine Hole Peg Test
    Screening, run-in, baseline, EOT, follow up: CGI-S
    Baseline, weeks 2, 4, 6, 8, 10, EOT, follow up: CGI-I
    Run-in, baseline, week 6, EOT, follow up: Clinician-completed Domain Specific Cause for Concern VAS: Myotonic Dystrophy and Top 3 Concerns
    Run-in, baseline and EOT: OSU CGI-S
    Baseline, week 6, EOT: OSU CGI-I
    Run-in, baseline, EOT: OSU Autism Rating Scale (and week 6), Peabody Picture Vocabulary Test
    Week 2, end of trial: PK samples
    Run-in, baseline, week 2, EOT: biomarker and RNA sample
    Baseline and EOT: DXA
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind Yes
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 8
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    The patient will provide assent or informed consent depending on the patient’s age and the investigator’s assessment of what is developmentally appropriate. Informed consent will also be provided by the caregiver/legally authorised representative.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 16
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-02-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-04-20
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-01-04
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