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Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02858908
Recruitment Status : Completed
First Posted : August 8, 2016
Last Update Posted : December 27, 2018
Sponsor:
Information provided by (Responsible Party):
AMO Pharma Limited

Study Description
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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.

Condition or disease Intervention/treatment Phase
Myotonic Dystrophy 1 Drug: Tideglusib Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
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
Actual Study Start Date : July 20, 2016
Actual Primary Completion Date : January 2018
Actual Study Completion Date : January 2018

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1 - Tideglusib
1000 mg tideglusib, orally, once daily
 Drug: Tideglusib
Tideglusib for oral suspension,
Experimental: Cohort 2 - Tideglusib
400 mg tideglusib, orally, once daily
 Drug: Tideglusib
Tideglusib for oral suspension,


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study. [ Time Frame: 14 weeks (baseline through end of study) ]
    Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.


Eligibility Criteria
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Ages Eligible for Study:   12 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02858908


Locations
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United Kingdom
Newcastle-upon-Tyne Hospitals NHS Trust
Newcastle Upon Tyne, Tyne And Wear, United Kingdom, NE1 4LP
Sponsors and Collaborators
AMO Pharma Limited
Investigators
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Principal Investigator: Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP Institute of Neuroscience, Newcastle University.
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: AMO Pharma Limited
ClinicalTrials.gov Identifier: NCT02858908    
Other Study ID Numbers: AMO-02-MD-2-001
2016-000067-16 ( EudraCT Number )
First Posted: August 8, 2016    Key Record Dates
Last Update Posted: December 27, 2018
Last Verified: December 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
 Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn