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Trial record 1 of 1 for:    AMO Pharma
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Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

This study is currently recruiting participants.
Verified March 2017 by AMO Pharma Limited
Sponsor:
ClinicalTrials.gov Identifier:
NCT02858908
First Posted: August 8, 2016
Last Update Posted: March 16, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
AMO Pharma Limited
  Purpose
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 Intervention Phase
Myotonic Dystrophy 1 Drug: Tideglusib Phase 2

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by AMO Pharma Limited:

Primary Outcome Measures:
  • 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) ]

Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Detailed Description:
The study is currently open for enrolment at the Newcastle Upon Tyne Hospitals NHS Trust for subjects aged between 16-45 years
  Eligibility

Information from the National Library of Medicine

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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
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
United Kingdom
Newcastle-upon-Tyne Hospitals NHS Trust Recruiting
Newcastle Upon Tyne, Tyne and Wear, United Kingdom, NE1 4LP
Contact: Nikoletta Nikolenko    0191 241 8989    Nikoletta.nikolenko@ncl.ac.uk   
Sponsors and Collaborators
AMO Pharma Limited
Investigators
Principal Investigator: Hanns Lochmuller, MD, FAAN John Walton Muscular Dystrophy Research Centre, Newcastle University.
  More Information

Responsible Party: AMO Pharma Limited
ClinicalTrials.gov Identifier: NCT02858908     History of Changes
Other Study ID Numbers: AMO-02-MD-2-001
2016-000067-16 ( EudraCT Number )
First Submitted: August 4, 2016
First Posted: August 8, 2016
Last Update Posted: March 16, 2017
Last Verified: March 2017

Additional relevant MeSH terms:
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


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