Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT03692312 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: AMO-02-MD-2-0032016-004623-23
• Study Type: interventional
• Target: 56
• Locations: 5 countries
• Sites: 14

Brief Summary

This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).

Conditions

Congenital Myotonic Dystrophy

Keywords

Tideglusib; AMO-02-MD-2-003; Congenital Myotonic Dystrophy; Myotonic Dystrophy; Dystrophia Myotonica; Myotonia Atrophica; Myotonia Dystrophica; Myotonic Dystrophy, Congenital; Steinert Disease; Steinert Myotonic Dystrophy; Steinert's Disease

Outcome Measures

Primary Outcomes (1)

• Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)

  The Clinician-Completed Congenital DM1 Scale is an 11-item rating scale completed by the clinician that scores the symptom severity of domains that are clinically relevant in Congenital DM1. The severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe.

  Baseline and week 20

Secondary Outcomes (10)

• Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores

  Baseline and week 20

• Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score

  Baseline and week 20

• Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)

  Baseline and week 20

• Clinical Global Impression - Severity Scale (CGI-S)

  Baseline and week 20

• 10-meter Walk-run Test

  20 weeks

Study Arms (2)

Tideglusib

EXPERIMENTAL

Weight adjusted tideglusib, orally, once daily

Drug: Tideglusib; Drug: Placebo

Placebo

PLACEBO COMPARATOR

Matching placebo, orally, once daily

Drug: Placebo

Interventions

Tideglusib DRUG

Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily

Tideglusib

Placebo DRUG

Matching placebo formulation

Placebo; Tideglusib

Eligibility Criteria

• Age: 6 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female children and adolescents aged ≥6 years and ≤16 years
• Diagnosis of Congenital DM1 (also known as Steinert's disease)
• Diagnosis must be genetically confirmed
• One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth:
• Hypotonia
• Generalized weakness
• Respiratory insufficiency
• Feeding difficulties
• Clubfoot or another musculoskeletal deformity
• Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
• Written, voluntary informed consent must be obtained before any study related procedures are conducted.
• Where a parent or LAR provides consent, there must also be assent from the subject
• Subject's caregiver must be willing and able to support participation for duration of study
• Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol

You may not qualify if:

• Not able to walk; (full time wheel chair use)
• Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
• New or change in medications/therapies within 4 weeks prior to Screening
• Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
• Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
• Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months
• Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
• Hypersensitivity to tideglusib and its excipients including allergy to strawberry

Sponsors & Collaborators

• AMO Pharma Limited: lead

Study Sites (14)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Utah Hospital

Salt Lake City, Utah, 84112, United States

Location

Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program.

Richmond, Virginia, 23219, United States

Location

The Bright Alliance

Randwick, New South Wales, 2031, Australia

Location

Children's Hospital London Health Sciences Centre (LHSC)

London, Ontario, N6A4G5, Canada

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Location

New Zealand Clinical Research (NZCR)

Auckland, 1010, New Zealand

Location

Newcastle University

Newcastle upon Tyne, NE2 4HH, United Kingdom

Location

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

Limitations and Caveats

None reported

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

• Joseph P Horrigan, MD

  AMO Pharma

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: March 16, 2018
• First Posted: October 2, 2018
• Study Start: March 3, 2021
• Primary Completion: April 4, 2023
• Study Completion: April 4, 2023
• Last Updated: October 8, 2025
• Results First Posted: September 11, 2025

Record last verified: 2025-09

Locations

GB (1); US (9); AU (1); NZ (1); CA (2)