Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

NCT05004129 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: AMO-02-MD-2-004
• Study Type: interventional
• Target: 76
• Locations: 4 countries
• Sites: 14

Brief Summary

This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

Conditions

Congenital Myotonic Dystrophy

Keywords

Tideglusib; AMO-02-MD-2-004; 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 (3)

• Safety (Adverse Events)

  The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from Screening to Enrolment (where applicable), from Enrolment to End of Treatment (52 Weeks), and End of Treatment to the End of Follow-up period.

  52 Weeks

• Safety (Adverse Events) - With Optional Expanded Access

  The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from End of Treatment to End of Optional Extended Access, and End of Optional Extended Access to the End of Follow-up period.

  Week 60 and every 8 weeks thereafter up until discontinuation or study closure, assessed up to Week 132

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

  The Clinician-completed Congenital DM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity of domains that are clinically relevant in Congenital DM1.

  52 Weeks

Secondary Outcomes (16)

• Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) - With Optional Expanded Access

  Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132

• Clinical Global Impressions Improvement Scale (CGI-I)

  54 Weeks

• Clinical Global Impressions Improvement Scale (CGI-I) - With Optional Expanded Access

  Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132

• Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score

  54 weeks

• Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score - With Optional Expanded Access

  Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132

Study Arms (1)

Tideglusib

EXPERIMENTAL

Weight adjusted or weight banded tideglusib, orally, once daily

Drug: Tideglusib

Interventions

Tideglusib DRUG

Tideglusib dosing will be weight-adjusted at 400 mg, 600 mg, or 1000 mg dose levels, or weight banded fixed doses of 400 mg, 600 mg, 800 mg or 1000 mg, with each subject starting at a weight-adjusted 400 mg dose level for 2 weeks, then up titrating to a weight-adjusted 600 mg dose level for the next 2 weeks.

Tideglusib

Eligibility Criteria

• Age: 6 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:
• Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
• Diagnosis must be genetically confirmed
• Subjects must be male or female aged ≥6 years to ≤45 years at Screening
• Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
• Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)
• 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
• Subjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:
• Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
• 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 (as required by local regulations)
• 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:

• 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 Eligibility/Baseline Visit
• Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
• 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 other than the AMO-02- MD-2-003 study
• Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or respiratory disease) that may impact the interpretability of the study results
• Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry

Sponsors & Collaborators

• AMO Pharma Limited: lead

Study Sites (14)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States

ENROLLING BY INVITATION

Stanford University

Palo Alto, California, 94304, United States

ENROLLING BY INVITATION

Lurie's Children's Hospital

Chicago, Illinois, 60611, United States

RECRUITING

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

RECRUITING

University of Rochester - Medical Center

Rochester, New York, 14642, United States

RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

University of Utah Clinical Neurosciences Center

Salt Lake City, Utah, 84132, United States

RECRUITING

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States

WITHDRAWN

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

Richmond, Virginia, 23219, United States

COMPLETED

The Bright Alliance

Randwick, New South Wales, 2031, Australia

RECRUITING

Children's Hospital London Health Sciences Centre (LHSC)

London, Ontario, N6A 4G5, Canada

ENROLLING BY INVITATION

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

RECRUITING

New Zealand Clinical Research (NZCR)

Auckland, 1010, New Zealand

COMPLETED

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

Study Officials

• Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM

  AMO Pharma

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2021
• First Posted: August 13, 2021
• Study Start: August 23, 2021
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: May 28, 2025

Record last verified: 2025-05

Locations

US (10); AU (1); NZ (1); CA (2)