Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02858908
• Date of registration: 04/08/2016
• Prospective Registration: No
• Primary sponsor: AMO Pharma Limited
• Public title: Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
• Scientific 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
• Date of first enrolment: July 20, 2016
• Target sample size: 16
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02858908
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Single (Participant).
• Phase: Phase 2

Countries of recruitment

United Kingdom

Contacts

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

Key inclusion & exclusion 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



Age minimum: 12 Years
Age maximum: 45 Years
Gender: All

Health Condition(s) or Problem(s) studied

Myotonic Dystrophy 1

Intervention(s)

Drug: Tideglusib

Primary Outcome(s)

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)]

Secondary ID(s)

2016-000067-16

AMO-02-MD-2-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors