Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
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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.
Condition or disease
Myotonic Dystrophy 1
The study is currently open for enrolment at the Newcastle Upon Tyne Hospitals NHS Trust for subjects aged between 16-45 years
Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across two dose levels of tideglusib. SAEs and AEs will be examined throughout the study. [ Time Frame: 14 weeks (baseline through end of study) ]
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Ages Eligible for Study:
12 Years to 45 Years (Child, Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
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
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)