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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.
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.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
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
TREATMENT
Phase
PHASE2
PHASE3
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
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/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: Tideglusib
| DRUG: Tideglusib
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary 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 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 outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) - With Optional Expanded Access | 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. | 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 Access | 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. | 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 | 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. | 54 weeks |
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score - With Optional Expanded Access | 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. | 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 Access | 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. | 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 Access | 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. | 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 Interview | 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. | 52 Weeks |
10-meter walk-run test | 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. | 52 Weeks |
Plasma Troponin T levels | Troponin 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 Access | Troponin 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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