Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

December 20, 2018 updated by: AMO Pharma Limited

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

16

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
    • Tyne And Wear
      • Newcastle Upon Tyne, Tyne And Wear, United Kingdom, NE1 4LP
        • Newcastle-upon-Tyne Hospitals NHS Trust

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 45 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Single

Arms and Interventions

Participant Group / 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,

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
14 weeks (baseline through end of study)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AMO Pharma Limited

Investigators

  • Principal Investigator: Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP, Institute of Neuroscience, Newcastle University.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 20, 2016

Primary Completion (Actual)

January 1, 2018

Study Completion (Actual)

January 1, 2018

Study Registration Dates

First Submitted

August 4, 2016

First Submitted That Met QC Criteria

August 4, 2016

First Posted (Estimate)

August 8, 2016

Study Record Updates

Last Update Posted (Actual)

December 27, 2018

Last Update Submitted That Met QC Criteria

December 20, 2018

Last Verified

December 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AMO-02-MD-2-001
  • 2016-000067-16 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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