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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

TREATMENT

Phase

PHASE2

PHASE3

Trial ID

NCT05004129

Intervention or treatment

DRUG: Tideglusib

Conditions

• Congenital Myotonic Dystrophy

Image related to Congenital Myotonic Dystrophy

Condition: Congenital Myotonic Dystrophy
DRUG: Tideglusib
Randwick, New South Wales, Australia
Sponsor: AMO Pharma Limited

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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 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 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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References

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