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

PHASE2PHASE3RECRUITING

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.

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Study details:

This is an open-label study of weight-adjusted 1000 mg tideglusib, once daily for 52 weeks with an open-ended optional extended access period in children and adolescents with a diagnosis of Congenital DM1 who participated in the AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  • 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 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
  • 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
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    Eligibility

    Age eligible for study : 6 and older

    Healthy volunteers accepted : No

    Gender eligible for study: All

    Things to know

    Study dates

    Study start: 2021-08-23

    Primary completion: 2025-03-28

    Study completion finish: 2025-03-28

    study type

    Study type

    TREATMENT

    phase

    Phase

      PHASE2

      PHASE3

    trial

    Trial ID

    NCT05004129

    Intervention or treatment

    DRUG: Tideglusib

    Conditions

    • Congenital Myotonic Dystrophy

    Find a site

    Closest Location:

    The Bright Alliance

    Research sites nearby

    Select from list below to view details:

    • The Bright Alliance

      Randwick, New South Wales, Australia

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    Study Plan

    This section provides details of the study plan, including how the study is designed and what the study is measuring.

    How is the study designed?

    Participant Group/ArmIntervention/Treatment
    EXPERIMENTAL: Tideglusib
    • Weight adjusted tideglusib, orally, once daily
    DRUG: Tideglusib
    • All subjects will receive weight-adjusted 1000 mg tideglusib following a titration period of 2 weeks at a weight-adjusted dose of 400 mg and 2 weeks at a weight-adjusted dose of 600 mg tideglusib.

    What is the study measuring?

    Primary outcome

    Primary Outcome MeasurePrimary Outcome DescriptionPrimary Outcome Time Frame
    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 AccessThe 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 outcome

    Secondary Outcome MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
    Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) - With Optional Expanded AccessThe 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.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)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.54 Weeks
    Clinical Global Impressions Improvement Scale (CGI-I) - With Optional Expanded AccessThe 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.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) scoreThe 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.54 weeks
    Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score - With Optional Expanded AccessThe 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.Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
    Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)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.52 weeks
    Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) - With Optional Expanded AccessCC-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.Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
    Clinical Global Impressions Severity Scale (CGI-S)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.54 weeks
    Clinical Global Impressions Severity Scale (CGI-S) - With Optional Expanded AccessThe 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.Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
    Autism Behavior Inventory- Clinician (ABI-C)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.52 Weeks
    Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey InterviewThe 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.52 Weeks
    10-meter walk-run testThe 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.52 Weeks
    Plasma Troponin T levelsTroponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.52 Weeks
    Plasma Troponin T levels - With Optional Expanded AccessTroponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132

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    References

    Clinical Trials Gov: Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

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