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Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

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ClinicalTrials.gov Identifier: NCT03692312
Recruitment Status : Recruiting
First Posted : October 2, 2018
Last Update Posted : August 15, 2022
Information provided by (Responsible Party):
AMO Pharma Limited

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).

Condition or disease Intervention/treatment Phase
Congenital Myotonic Dystrophy Drug: Tideglusib Drug: Placebo Phase 2 Phase 3

Detailed Description:
This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 56 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : March 3, 2021
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : February 2023

Arm Intervention/treatment
Experimental: Tideglusib
Weight adjusted tideglusib, orally, once daily
Drug: Tideglusib
Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily

Drug: Placebo
Matching placebo formulation

Placebo Comparator: Placebo
Matching placebo, orally, once daily
Drug: Placebo
Matching placebo formulation

Primary Outcome Measures :
  1. Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) [ Time Frame: 22 weeks ]
    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.

Secondary Outcome Measures :
  1. Change in Clinical Global Impression- Improvement Scale (CGI-I) scores [ Time Frame: 22 weeks ]
    The clinician administered CGI-I rates how much the subject's illness has improved or worsened relative to a baseline state.

  2. Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score [ Time Frame: 22 Weeks ]
    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.

  3. Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) [ Time Frame: 22 weeks ]
    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.

  4. Clinical Global Impression - Severity Scale (CGI-S) [ Time Frame: 22 weeks ]
    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.

  5. 10-meter walk-run test [ Time Frame: 22 weeks ]
    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.

  6. Incidence of Adverse events (AEs), including serious adverse events (SAEs), between Screening to end of study. [ Time Frame: 22 to 28 weeks ]
    Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.

  7. Incidence of abnormal findings in objective assessments (e.g. laboratory values, ECGs, vital signs and bone mineral density) between Screening and end of study. [ Time Frame: 22 to 28 weeks ]
    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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Ages Eligible for Study:   6 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03692312

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United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Annette Guy    501-364-3380    guyea@archildrens.org   
Principal Investigator: Aravindhan Veerapandiyan, MD         
United States, California
University of California, Los Angeles (UCLA) Recruiting
Los Angeles, California, United States, 90095
Contact: Dennis Fernando    310-825-3264    DeFernando@mednet.ucla.edu   
Principal Investigator: Perry Shieh, MD, PhD         
Stanford University Recruiting
Palo Alto, California, United States, 94304
Contact: Stanford Neuromuscular Research    650-725-4341    neuromuscularresearch@stanford.edu   
Principal Investigator: John W Day, MD, PhD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Ishita Tejani    312-227-3019    itejani@luriechildrens.org   
Principal Investigator: Vamshi Rao, MD         
United States, Iowa
University of Iowa Hospitals and Clinics Recruiting
Iowa City, Iowa, United States, 52242
Contact: Chandra Miller    319-384-9618    chandra-miller@uiowa.edu   
Principal Investigator: Katherine Mathews, MD         
United States, New York
University of Rochester Medical Center Recruiting
Rochester, New York, United States, 14642
Contact: James Hilbert    585-273-5590    james_hilbert@URMC.Rochester.edu   
Contact: Jeanne Dekdebrun    585-276-4611    jeanne_Dekdebrun@URMC.Rochester.edu   
Sub-Investigator: Johanna Hamel, MD         
Sub-Investigator: Emma Ciafaloni, MD         
Principal Investigator: Bo Hoon Lee, MD         
United States, Pennsylvania
University of Pittsburgh Medical Center Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Jennifer Monahan    412-692-5176    jennifer.monahan@chp.edu   
Contact: Casey Matthews    412-692-5480    casey.nguyen@chp.edu   
Principal Investigator: Hoda Abdel-Hamid, MD         
United States, Utah
University of Utah Hospital Recruiting
Salt Lake City, Utah, United States, 84112
Contact: Lauren Branigan    845-544-3978    lauren.branigan@hsc.utah.edu   
Principal Investigator: Stephanie Manberg, DO         
United States, Virginia
Children's Hospital of the King's Daughters Withdrawn
Norfolk, Virginia, United States, 23507
Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program. Recruiting
Richmond, Virginia, United States, 23219
Contact: Jodie Howell    804-828-6011    Jodie.Howell@vcuhealth.org   
Contact: Jessica St Romain    804-828-7887    Mary.StRomain@vcuhealth.org   
Principal Investigator: Nicholas Johnson, MD         
Australia, New South Wales
The Bright Alliance Recruiting
Randwick, New South Wales, Australia, 2031
Contact: Sandra Holland    02 9382 5584    Sandra.Holland@health.nsw.gov.au   
Principal Investigator: Michelle Farrar, MD         
Canada, Ontario
Children's Hospital London Health Sciences Centre (LHSC) Recruiting
London, Ontario, Canada, N6A4G5
Contact: Rhiannon Hicks    519-685-8441    rhiannon.hicks@lhsc.on.ca   
Principal Investigator: Craig Campbell, MD, MSc, FRCPC         
Children's Hospital of Eastern Ontario Recruiting
Ottawa, Ontario, Canada, K1H 8L1
Contact: Emilie Hill Smith    613-737-7600 ext 4014    EHillSmith@cheo.on.ca   
Principal Investigator: Hanns Lochmuller, MD         
New Zealand
New Zealand Clinical Research (NZCR) Recruiting
Auckland, New Zealand, 1010
Contact: Teri Hodgson    64 9 373 3474 ext 109    reach@nzcr.co.nz   
Principal Investigator: Gina O'Grady, MD         
United Kingdom
Newcastle University Not yet recruiting
Newcastle Upon Tyne, United Kingdom, NE2 4HH
Contact: Angela Green    0191 208 3105    Angela.Green@newcastle.ac.uk   
Principal Investigator: Grainne Gorman, MRCP         
Sponsors and Collaborators
AMO Pharma Limited
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Study Director: Joseph P Horrigan, MD AMO Pharma
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Responsible Party: AMO Pharma Limited
ClinicalTrials.gov Identifier: NCT03692312    
Other Study ID Numbers: AMO-02-MD-2-003
2016-004623-23 ( EudraCT Number )
First Posted: October 2, 2018    Key Record Dates
Last Update Posted: August 15, 2022
Last Verified: August 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by AMO Pharma Limited:
Congenital Myotonic Dystrophy
Myotonic Dystrophy
Dystrophia Myotonica
Myotonia Atrophica
Myotonia Dystrophica
Myotonic Dystrophy, Congenital
Steinert Disease
Steinert Myotonic Dystrophy
Steinert's Disease
Additional relevant MeSH terms:
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Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn