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
Interventional
Primary Outcome
Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.
Condition
Myotonic Dystrophy 1
Intervention
Tideglusib
Study Arms / Comparison Groups
Cohort 1 - Tideglusib
Description: 1000 mg tideglusib, orally, once daily
Publications
* 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
Drug
Estimated Enrollment
16
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 allowed) - 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 conducted 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 sterile. - 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
Gender
All
Ages
12 Years - 45 Years
Accepts Healthy Volunteers
No
Contacts
Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP, ,
Location Countries
United Kingdom
Location Countries
United Kingdom
Administrative Informations
NCT ID
NCT02858908
Organization ID
AMO-02-MD-2-001
Secondary IDs
2016-000067-16
Responsible Party
Sponsor
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