Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

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Brief Title

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

Official Title

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

Brief Summary

      The purpose of this study is to determine whether Tideglusib is safe and efficacious in the
      treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy.
      The pharmacokinetics of tideglusib and its primary metabolite will also be investigated.

Study Phase

Phase 2

Study Type


Primary Outcome

Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.


Myotonic Dystrophy 1



Study Arms / Comparison Groups

 Cohort 1 - Tideglusib
Description:  1000 mg tideglusib, orally, once daily


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

July 20, 2016

Completion Date

January 2018

Primary Completion Date

January 2018

Eligibility Criteria

        Inclusion Criteria:

          -  Adolescents or adults with diagnosis of congenital or juvenile-onset type 1 myotonic
             dystrophy (DM-1)

          -  Diagnosis must be genetically confirmed

          -  Subjects must be male or female aged 12 years to 45 years

          -  Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 4 or
             greater at Screening and Run-in (V2)

          -  Subjects must be ambulatory and able to complete the 10 metre walk/run test (splints

          -  Subject's legally authorized 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

        Exclusion Criteria:

          -  Non-ambulatory (full time) wheel chair user

          -  Receiving stimulant medication

          -  Receiving other medications/therapies not stable (changed) within 4 weeks prior to
             Run-in (V2)

          -  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.

          -  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

          -  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

          -  Gastrointestinal disease which may interfere with the absorption, distribution,
             metabolism or excretion of the study medication and impact the interpretability of the
             study results

          -  Current clinically significant (as determined by the investigator) cardiovascular,
             renal, hepatic, endocrine or respiratory disease

          -  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

          -  A history of chronic liver disease with current out of range values for Alanine
             transaminase (ALT), clinically relevant hepatic steatosis or other clinical
             manifestations of ongoing liver disease

          -  A history of significant drug allergy (such as Steven-Johnson syndrome, anaphylaxis)

          -  A history of alcohol or substance use disorders




12 Years - 45 Years

Accepts Healthy Volunteers



Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP, , 

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

AMO Pharma Limited

Study Sponsor

Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP, Principal Investigator, Institute of Neuroscience, Newcastle University.

Verification Date

December 2018