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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05004129
Registration number
NCT05004129
Ethics application status
Date submitted
5/08/2021
Date registered
13/08/2021
Date last updated
8/11/2023
Titles & IDs
Public title
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
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Scientific 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)
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Secondary ID [1]
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AMO-02-MD-2-004
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Universal Trial Number (UTN)
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Trial acronym
REACH CDM X
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Congenital Myotonic Dystrophy
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Tideglusib
Experimental: Tideglusib - Weight adjusted tideglusib, orally, once daily
Treatment: Drugs: 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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Safety (Adverse Events)
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Assessment method [1]
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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.
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Timepoint [1]
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52 Weeks
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Primary outcome [2]
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Safety (Adverse Events) - With Optional Expanded Access
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Assessment method [2]
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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.
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Timepoint [2]
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Week 60 and every 8 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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Primary outcome [3]
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Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
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Assessment method [3]
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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.
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Timepoint [3]
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52 Weeks
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Secondary outcome [1]
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Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) - With Optional Expanded Access
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Assessment method [1]
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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.
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Timepoint [1]
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Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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Secondary outcome [2]
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Clinical Global Impressions Improvement Scale (CGI-I)
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Assessment method [2]
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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.
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Timepoint [2]
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54 Weeks
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Secondary outcome [3]
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Clinical Global Impressions Improvement Scale (CGI-I) - With Optional Expanded Access
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Assessment method [3]
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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.
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Timepoint [3]
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Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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Secondary outcome [4]
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Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score
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Assessment method [4]
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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.
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Timepoint [4]
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54 weeks
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Secondary outcome [5]
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Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score - With Optional Expanded Access
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Assessment method [5]
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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.
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Timepoint [5]
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Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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Secondary outcome [6]
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Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
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Assessment method [6]
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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.
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Timepoint [6]
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52 weeks
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Secondary outcome [7]
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Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) - With Optional Expanded Access
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Assessment method [7]
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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.
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Timepoint [7]
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Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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Secondary outcome [8]
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Clinical Global Impressions Severity Scale (CGI-S)
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Assessment method [8]
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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.
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Timepoint [8]
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54 weeks
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Secondary outcome [9]
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Clinical Global Impressions Severity Scale (CGI-S) - With Optional Expanded Access
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Assessment method [9]
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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.
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Timepoint [9]
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Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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Secondary outcome [10]
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Autism Behavior Inventory- Clinician (ABI-C)
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Assessment method [10]
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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.
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Timepoint [10]
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52 Weeks
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Secondary outcome [11]
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Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey Interview
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Assessment method [11]
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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.
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Timepoint [11]
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52 Weeks
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Secondary outcome [12]
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10-meter walk-run test
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Assessment method [12]
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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.
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Timepoint [12]
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52 Weeks
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Secondary outcome [13]
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Plasma Troponin T levels
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Assessment method [13]
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Troponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.
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Timepoint [13]
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52 Weeks
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Secondary outcome [14]
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Plasma Troponin T levels - With Optional Expanded Access
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Assessment method [14]
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Troponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.
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Timepoint [14]
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Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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Eligibility
Key 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:
1. Subjects under study must be individuals with a diagnosis of Congenital or Childhood
Onset DM1.
2. Diagnosis must be genetically confirmed
3. Subjects must be male or female aged =6 years to =45 years at Screening
4. Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or
greater at Screening (V-1)
5. 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)
6. Subject's caregiver must be willing and able to support participation for duration of
study
7. 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:
1. Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
2. 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)
3. Subject's caregiver must be willing and able to support participation for duration of
study
4. Subject must be willing and able to comply with the required food intake restrictions
as outlined per protocol
Key
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Minimum age
6
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Maximum age
45
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
2. New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline
Visit
3. Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors
(eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole,
nefazodone, idinavir and ritonavir)
4. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g.
warfarin and digitoxin)
5. 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
6. 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
7. Hypersensitivity to tideglusib or any components of its formulation including allergy
to strawberry
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2/Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
23/08/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
28/03/2025
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Actual
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Sample size
Target
76
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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The Bright Alliance - Randwick
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Recruitment postcode(s) [1]
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2031 - Randwick
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Arkansas
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Country [2]
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United States of America
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State/province [2]
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California
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Country [3]
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United States of America
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State/province [3]
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Illinois
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Country [4]
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United States of America
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State/province [4]
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Iowa
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Country [5]
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United States of America
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State/province [5]
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New York
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Country [6]
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United States of America
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State/province [6]
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Pennsylvania
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Country [7]
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United States of America
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State/province [7]
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Utah
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Country [8]
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United States of America
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State/province [8]
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Virginia
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Country [9]
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Canada
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State/province [9]
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Ontario
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Country [10]
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New Zealand
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State/province [10]
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Auckland
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
AMO Pharma Limited
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05004129
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Joseph P Horrigan, MD
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Address
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AMO Pharma
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05004129
Download to PDF