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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03692312
Registration number
NCT03692312
Ethics application status
Date submitted
16/03/2018
Date registered
2/10/2018
Date last updated
8/09/2023
Titles & IDs
Public title
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
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Scientific title
A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM)
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Secondary ID [1]
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2016-004623-23
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Secondary ID [2]
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AMO-02-MD-2-003
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Universal Trial Number (UTN)
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Trial acronym
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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
Treatment: Drugs - Placebo
Experimental: Tideglusib - Weight adjusted tideglusib, orally, once daily
Placebo comparator: Placebo - Matching placebo, orally, once daily
Treatment: Drugs: Tideglusib
Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily
Treatment: Drugs: Placebo
Matching placebo formulation
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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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Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
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Assessment method [1]
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The Clinician-Completed Congenital DM1 Scale is an 11-item rating scale completed by the clinician that scores the symptom severity of domains that are clinically relevant in Congenital DM1.
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Timepoint [1]
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22 weeks
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Secondary outcome [1]
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Change in Clinical Global Impression- Improvement Scale (CGI-I) scores
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Assessment method [1]
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The clinician administered CGI-I rates how much the subject's illness has improved or worsened relative to a baseline state.
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Timepoint [1]
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22 weeks
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Secondary outcome [2]
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Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score
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Assessment method [2]
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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 [2]
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22 Weeks
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Secondary outcome [3]
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Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
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Assessment method [3]
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The Caregiver-Completed Congenital DM1 Scale is a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 clinically relevant symptoms that the caregiver is asked to rate the severity of.
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Timepoint [3]
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22 weeks
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Secondary outcome [4]
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Clinical Global Impression - Severity Scale (CGI-S)
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Assessment method [4]
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The Clinical Global Impression - Severity Scale (CGI-S) is a 7-point 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 [4]
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22 weeks
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Secondary outcome [5]
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10-meter walk-run test
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Assessment method [5]
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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 [5]
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22 weeks
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Secondary outcome [6]
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Incidence of Adverse events (AEs), including serious adverse events (SAEs), between Screening to end of study.
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Assessment method [6]
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Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.
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Timepoint [6]
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22 to 28 weeks
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Secondary outcome [7]
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Incidence of abnormal findings in objective assessments (e.g. laboratory values, ECGs, vital signs and bone mineral density) between Screening and end of study.
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Assessment method [7]
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Abnormal laboratory findings (e.g. hematology, liver function, biochemistry, urinalysis) or other abnormal assessments (e.g. ECGs, vital signs) that are judged by the Investigator as clinically significant will be recorded as AEs or SAEs if they meet the definition of an AE. The Investigator will exercise his or her medical and scientific judgment in deciding whether an abnormal laboratory finding or other abnormal assessment is clinically significant.
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Timepoint [7]
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22 to 28 weeks
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Eligibility
Key inclusion criteria
1. Male or female children and adolescents aged =6 years and =16 years
2. Diagnosis of Congenital DM1 (also known as Steinert's disease)
* Diagnosis must be genetically confirmed
* One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth:
* Hypotonia
* Generalized weakness
* Respiratory insufficiency
* Feeding difficulties
* Clubfoot or another musculoskeletal deformity
3. Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
4. 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
5. Subject's caregiver must be willing and able to support participation for duration of study
6. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
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Minimum age
6
Years
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Maximum age
16
Years
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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. Not able to walk; (full time wheel chair use)
2. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
3. New or change in medications/therapies within 4 weeks prior to Screening
4. Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
5. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
6. 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
7. Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
8. Hypersensitivity to tideglusib and its excipients including allergy to strawberry
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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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
3/03/2021
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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
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Actual
4/04/2023
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Sample size
Target
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Accrual to date
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Final
56
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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
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United States of America
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State/province [1]
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Arkansas
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United States of America
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California
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United States of America
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Illinois
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Iowa
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United States of America
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New York
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United States of America
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Pennsylvania
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Utah
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Virginia
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Canada
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Ontario
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New Zealand
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State/province [10]
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Auckland
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Country [11]
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United Kingdom
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State/province [11]
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Newcastle Upon Tyne
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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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Ethics approval
Ethics application status
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Summary
Brief summary
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
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Trial website
https://clinicaltrials.gov/study/NCT03692312
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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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Phone
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Fax
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Email
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Contact person for public queries
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03692312
Download to PDF