Skip to main content
OpenTrials
Recruiting

NCT Number: NCT05004129

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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Congenital Myotonic Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases

Age range

6 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

The Bright Alliance, Randwick, New South Wales, Australia

Loading trial locations.

About this study

This is an open-label study of either a weight-adjusted 1000 mg fixed dose or a weight banded fixed dose of tideglusib across a 52-week treatment period with an open-ended optional extended access period. The subjects are children and adolescents with Congenital DM1 who participated in the antecedent AMO-02-MD-2-003 study or individuals with either Congenital or Childhood onset DM1 who are treatment naïve.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Key Exclusion Criteria:

  • 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

Treatment and study plan

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.

Primary outcomes

  1. Safety (Adverse Events)

    Time frame: 52 Weeks

    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.

  2. Safety (Adverse Events) - With Optional Expanded Access

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

    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.

  3. Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)

    Time frame: 52 Weeks

    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.

Secondary outcomes

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

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

    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.

  2. Clinical Global Impressions Improvement Scale (CGI-I)

    Time frame: 54 Weeks

    The CGI-I requires the clinician to rate how much the subject's illness has changed (improved, worsened or stayed the same) relative to a baseline state.

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

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

    The CGI-I requires the clinician to rate how much the subject's illness has changed (improved, worsened or stayed the same) relative to a baseline state.

  4. Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score

    Time frame: 54 weeks

    The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study.

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

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

    The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study.

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

    Time frame: 52 weeks

    CC-CDM1-RS provides a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 symptoms that the caregiver is asked to rate from 0 to 4 based on overall severity.

  7. Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) - With Optional Expanded Access

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

    CC-CDM1-RS provides a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 symptoms that the caregiver is asked to rate from 0 to 4 based on overall severity.

  8. Clinical Global Impressions Severity Scale (CGI-S)

    Time frame: 54 weeks

    The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis.

  9. Clinical Global Impressions Severity Scale (CGI-S) - With Optional Expanded Access

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

    The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis.

  10. Autism Behavior Inventory- Clinician (ABI-C)

    Time frame: 52 Weeks

    ABI-C is a 14 item rating scale requires the clinician to assess the core features of Autism Spectrum Disorder as well as common associated behaviors.

  11. Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey Interview

    Time frame: 52 Weeks

    The Vineland Adaptive Behavior Scales require the clinician to measure personal and social skills needed for everyday living. It provides a targeted assessment of adaptive behavior via a semi-structured parent or caregiver interview.

  12. 10-meter walk-run test

    Time frame: 52 Weeks

    The 10-meter walk/run test is a performance measure used to assess walking speed in meters per second over a short distance. It can be used as an assessment of functional mobility.

  13. Plasma Troponin T levels

    Time frame: 52 Weeks

    Troponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.

  14. Plasma Troponin T levels - With Optional Expanded Access

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

    Troponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.

  15. Blood Leukocytes CTG Repeats

    Time frame: Baseline and Week 52

    A genomic blood sample will be taken from all subjects to confirm the average number of repeats at sampling and estimate the number of leukocyte CTG repeats inherited for an individual subject.

  16. Blood Leukocytes CTG Repeats - With Optional Expanded Access

    Time frame: Week 52 or later

    A genomic blood sample will be taken from all subjects to confirm the average number of repeats at sampling and estimate the number of leukocyte CTG repeats inherited for an individual subject. For subjects that have already passed Week 52 at implementation of this endpoint, a single genomic blood sample will taken at the next in-clinic OEA visit.

Sponsors and collaborators

Lead sponsor

AMO Pharma Limited

Industry

Registry information

Official study title

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)

Acronym: REACH CDM X

Important dates

Study start
2021
Primary completion
2026
Study completion
2026
First posted
Aug 13, 2021
Registry last updated
May 28, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.