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    Summary
    EudraCT Number:2016-004623-23
    Sponsor's Protocol Code Number:AMO-02-MD-2-003
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-02-08
    Trial 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 number2016-004623-23
    A.3Full title of the trial
    A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy
    A.3.2Name or abbreviated title of the trial where available
    Efficacy & Safety of Tideglusib in Congenital Myotonic Dystrophy
    A.4.1Sponsor's protocol code numberAMO-02-MD-2-003
    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+4401483382444
    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 IMPGastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTideglusib
    D.3.9.1CAS number 865854-05-3
    D.3.9.2Current sponsor codeAMO-02
    D.3.9.3Other descriptive name4-benzyl-2-naphthalen-1-yl-1,2,4-thiadiazolidine-3,5-dione
    D.3.9.4EV Substance CodeAS1
    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 child and adolescent congenital myotonic dystrophy.
    E.1.1.1Medical condition in easily understood language
    Treatment of myotonic dystrophy in children 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 20.0
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    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 evaluate the efficacy, between start and end of treatment, of tideglusib compared to placebo in children and adolescents with Congenital Myotonic Dystrophy (DM1).
    E.2.2Secondary objectives of the trial
    To evaluate the safety and tolerability of tideglusib compared to placebo in children and adolescents with Congenital DM1

    To evaluate the blood levels of tideglusib in children and adolescents with Congenital DM1.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The subject will not be considered eligible for the study without meeting all of the criteria below:
    1. Subjects under study must be children or adolescents with a diagnosis of DM1. For the purposes of this study, the following definitions apply.
    In addition to the genetic confirmation of DM1, one or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first week after birth:
    • Hypotonia
    • Generalized weakness
    • Respiratory insufficiency
    • Feeding difficulties
    • Clubfoot or another musculoskeletal deformity
    2. Diagnosis must be genetically confirmed
    3. Subjects must be male or female children and adolescents aged ≥6 years and ≤16 years at Screening
    4. Subjects must have a Clinical Global Impression – Severity (CGI-S) score of 4 or greater at Screening and start of Run-in (V2)
    5. Subjects must be ambulatory and able to complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
    6. Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations)
    7. Subject’s caregiver must be willing and able to support participation for duration of study
    8. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
    E.4Principal exclusion criteria
    1. Non-ambulatory (full time) wheel chair use
    2. BMI less than 13.5 kg/m² or greater than 40 kg/m²
    3. Receiving other medications/therapies not stable (i.e. changed) within 4 weeks prior to Screening. For each enrollee, every effort should be made to maintain stable regimens (i.e. dose, as applicable, and frequency) of allowed concomitant medications (e.g. concomitant mexiletine or stimulants) and allowed non-medicine based therapies (e.g. occupational or physiotherapy) throughout the course of the study, from the time of commencement of Screening until the last study assessment
    4. Use within 4 weeks prior to Baseline (V3) of strong CYP3A4 inhibitors. Examples include clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir
    5. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window e.g. warfarin and digitoxin
    6. Medical illness or other concern which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
    7. Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months
    8. Gastrointestinal disease which, in the opinion of the investigator, 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) neurological, cardiovascular, renal, hepatic, endocrine or respiratory disease that may impact the interpretability of the study results
    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. Implantation of a cardiac pacemaker within the 12 months preceding Screening
    12. Average QTcF value of >450 msec at Screening or at Run-In (V2) (may repeat to confirm)
    13. Clinically significant abnormalities in safety laboratory tests, vital signs or ECG, as determined by the Investigator at Screening or Run-In (V2) as applicable (may repeat to confirm)
    14. Females 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
    15. Males, engaged in sexual relations with a female of child-bearing potential, not using an acceptable contraceptive method if not surgically sterile
    16. Kidney disease requiring ongoing treatment
    17. A history of chronic liver disease with current out of range values for ALT, clinically relevant hepatic steatosis or other clinical manifestations of liver disease
    18. ALT value > 2X ULN reference range at Screening (may repeat to confirm)
    19. Total bilirubin value greater than the upper limit of the normal reference range at Screening (unless due to Gilbert’s syndrome) (may repeat to confirm). For subjects with a well known/well documented diagnosis of Gilbert's syndrome a total bilirubin value >2x ULN reference range at Screening (may repeat to confirm)
    20. HbA1c values greater than 6% or 42.0 mmol/mol at Screening (may repeat to confirm)
    21. TSH values outside of the normal reference range at Screening (may repeat to confirm)
    22. Serum creatinine >1.7 mg/dL (>150 micromole/L) or creatinine clearance ≤ 60 mL/min (according to Cockcroft-Gault formula) at Screening (may repeat to confirm)
    23. Clinical history of hepatitis or previous or current positive serological evidence for Hep B or C
    24. Serological evidence of Hep A at Screening or in the 6 months preceding Screening
    25. A history of significant drug allergy (such as Steven-Johnson syndrome, anaphylaxis)
    26. A history of alcohol or substance use disorders
    27. 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 Congenital DM1)
    28. Severe arthritis or other medical condition (besides Congenital DM1) that would significantly impact ambulation or completion of myometric assessments
    29. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry
    30. Unable to swallow liquids or may have trouble swallowing liquids (in the opinion of the investigator), unless medication to be administered by gastrostomy tube
    E.5 End points
    E.5.1Primary end point(s)
    Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
    E.5.1.1Timepoint(s) of evaluation of this end point
    A blinded sample size re-estimation, based on the observed variance in the primary outcome measure, will occur when approximately 50% of subjects are enrolled and have completed week 20 (Visit 11). The variability of key secondary and secondary endpoints may be assessed as well, if feasible.

    This endpoint will be fully evaluated once a full data set is acquired.
    E.5.2Secondary end point(s)
    Key Secondary Efficacy Endpoint:
    • Clinical Global Impression- Improvement Scale (CGI-I)
    Secondary Efficacy Endpoints:
    • Top 3 Caregiver Concerns VAS score
    • Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
    • Clinical Global Impression - Severity Scale (CGI-S)
    • DXA Scan measurement of total body lean/muscle mass
    E.5.2.1Timepoint(s) of evaluation of this end point
    When approximately 50% of subjects are enrolled and have completed week 20 (Visit 11). The variability of key secondary and secondary endpoints may be assessed, if feasible.

    All secondary endpoints will be evaluated once a full data set is acquired.
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    United States
    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
    End of the study is defined as - Last subject's last study assessment. This is conducted 2 weeks post treatment during the follow up (Visit 12) for those subjects not entering the extension protocol. If the subject has entered into the extension protocol, the end of the trial is the final study assessment (end of treatment, Visit 11).
    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 months0
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days30
    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: 56
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 28
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 28
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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
    6-15 years olds will assent and parent/LAR will consent.

    16 year olds will consent if they have mental capacity. If they do not have mental capacity then the parent/LAR will consent and they will assent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 56
    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)
    It is currently unclear if tideglusib will render clinical benefits to the participants. It is not appropriate to promise continuation of study medication to participants until more substantial information is available indicating that tideglusib renders an acceptable prospect of benefit for the participants. However, for subjects who have completed this study and meet eligibility criteria, a longer term extension study will be offered to provide access to the medication for a further 32 weeks.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-03-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-11-19
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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