Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

NCT02858908 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: AMO-02-MD-2-0012016-000067-16
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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.

Conditions

Myotonic Dystrophy 1

Outcome Measures

Primary Outcomes (1)

• Safety (Adverse Events)

  Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.

  12 weeks

Secondary Outcomes (19)

• Plasma Concentration of Tideglusib

  12 weeks

• Blood Pharmacokinetics of Tideglusib

  12 weeks

• Area Under the Plasma Concentration vs. Time Curve of Tideglusib

  12 weeks

• 10 Metre Walk/Run Test

  12 weeks

• Computerised Handgrip Myometer Measure of Grip Strength and Muscle Relaxation Time

  12 weeks

Study Arms (2)

Cohort 1 - Tideglusib

EXPERIMENTAL

1000 mg tideglusib, orally, once daily

Drug: Tideglusib

Cohort 2 - Tideglusib

EXPERIMENTAL

400 mg tideglusib, orally, once daily

Drug: Tideglusib

Interventions

Tideglusib DRUG

Tideglusib for oral suspension,

Cohort 1 - Tideglusib; Cohort 2 - Tideglusib

Eligibility Criteria

• Age: 12 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• AMO Pharma Limited: lead

Study Sites (1)

Newcastle-upon-Tyne Hospitals NHS Trust

Newcastle upon Tyne, Tyne and Wear, NE1 4LP, United Kingdom

Location

Related Publications (1)

• Horrigan J, Gomes TB, Snape M, Nikolenko N, McMorn A, Evans S, Yaroshinsky A, Della Pasqua O, Oosterholt S, Lochmuller H. A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1). Pediatr Neurol. 2020 Nov;112:84-93. doi: 10.1016/j.pediatrneurol.2020.08.001. Epub 2020 Aug 5.

  PMID: 32942085 DERIVED

MeSH Terms

Conditions

Myotonic Dystrophy

Interventions

tideglusib

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Dr. Mike Snape, Chief Scientific Officer
• Organization: AMO Pharma Ltd.

mike.snape@amo-pharma.com

+44 (0) 7775915639

Study Officials

• Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP

  Institute of Neuroscience, Newcastle University.

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: August 4, 2016
• First Posted: August 8, 2016
• Study Start: July 20, 2016
• Primary Completion: January 1, 2018
• Study Completion: January 1, 2018
• Last Updated: September 11, 2025
• Results First Posted: September 11, 2025

Record last verified: 2025-08

Locations

GB (1)