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

AMO Pharma Ltd.0 sites16 target enrollmentMay 13, 2016

Overview

No summary available.

Registry

who.int

Start Date

May 13, 2016

End Date

TBD

Last Updated

7 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•1\. Subjects under study must be adolescent or adults with a diagnosis of congenital or juvenile\-onset type 1 myotonic dystrophy (DM\-1\). For the purposes of this study, the following definitions apply:
•Congenital: in addition to the genetic confirmation of DM\-1, one or more of the following signs or symptoms was evident within the first week after birth:
•a) Hypotonia
•b) Generalized weakness
•c) Respiratory insufficiency
•d) Feeding difficulties
•e) Clubfoot or another musculoskeletal deformity
•Childhood/juvenile\-onset: in addition to the genetic confirmation of DM\-1, at least 2 signs or symptoms (not caused by another, unrelated condition) were evident prior to 12 years of age that can be clearly assigned to DM\-1, for example:
•a) Muscle weakness
•b) Myotonia (delayed muscle relaxation)

•1\. Non\-ambulatory (full time) wheel chair user
•2\. Receiving stimulant medication
•3\. Receiving other medications/therapies not stable (changed) within 4 weeks prior to Run\-in (V2\)
•4\. 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
•5\. 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
•6\. 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
•7\. Men, if engaged in sexual relations with a female of child\-bearing potential, not using an acceptable contraceptive method if not surgically sterile
•8\. Gastrointestinal disease which 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) cardiovascular, renal, hepatic, endocrine or respiratory disease
•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