Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-07-20

Study Completion Date

2018-01-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Myotonic Dystrophy 1

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

Study Groups

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Cohort 1 - Tideglusib

1000 mg tideglusib, orally, once daily

Group Type EXPERIMENTAL

Tideglusib

Intervention Type DRUG

Tideglusib for oral suspension,

Cohort 2 - Tideglusib

400 mg tideglusib, orally, once daily

Group Type EXPERIMENTAL

Tideglusib

Intervention Type DRUG

Tideglusib for oral suspension,

Interventions

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Tideglusib

Tideglusib for oral suspension,

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

12 Years

Maximum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No