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Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03692312
Recruitment Status : Not yet recruiting
First Posted : October 2, 2018
Last Update Posted : September 21, 2020
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
Estimated Study Start Date : November 2020
Estimated Primary Completion Date : February 2022
Estimated Study Completion Date : March 2022

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. 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.

  6. 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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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 week 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 identifier (NCT number): NCT03692312

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United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Contact: Annette Guy    501-364-3380   
Principal Investigator: Aravindhan Veerapandiyan, MD         
United States, California
University of California, Los Angeles (UCLA)
Los Angeles, California, United States, 90095
Contact    310-825-3264      
Principal Investigator: Perry Shieh, MD, PhD         
Stanford University
Palo Alto, California, United States, 94304
Contact: Stanford Neuromuscular Research    650-725-4341   
Principal Investigator: John W Day, MD, PhD         
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
Contact: Bryn Miller, BSN, RN    319-335-5836   
Principal Investigator: Katherine Mathews, MD         
United States, New York
University of Rochester Medical Center
Rochester, New York, United States, 14642
Contact: James Hilbert    585-273-5590   
Contact: Jeanne Dekdebrun    585-276-4611   
Principal Investigator: Johanna Hamel, MD         
Sub-Investigator: Emma Ciafaloni, MD         
United States, Pennsylvania
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15213
Contact: Jennifer Monahan    412-692-5176   
Principal Investigator: Hoba Abdel-Hamid, MD         
United States, Virginia
Children's Specialty Group, PLLC, Division of Child & Adolescent Neurology, Children's Hospital of the King's Daughters
Norfolk, Virginia, United States, 23510
Contact: Terrie Conklin, RN, CCRC    757-668-9356    terrie.conklin@CHKD.ORG   
Principal Investigator: Crystal Proud, MD         
Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program.
Richmond, Virginia, United States, 23219
Contact: Danielle Frazier    804-277-9244   
Principal Investigator: Nicholas Johnson, MD         
Canada, Ontario
Children's Hospital London Health Sciences Centre (LHSC)
London, Ontario, Canada, N6A4G5
Contact: Rhiannon Hicks    519-685-8441   
Principal Investigator: Craig Campbell, MD, MSc, FRCPC         
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Contact: Rosa Ramos   
Principal Investigator: Hugh McMillan, MD, MSc         
United Kingdom
Newcastle University
Newcastle Upon Tyne, United Kingdom, NE2 4HH
Contact: Angela Green    0191 208 3105   
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 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: September 21, 2020
Last Verified: September 2020

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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
Myotonic Disorders
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn