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Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

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ClinicalTrials.gov Identifier: NCT03692312
Recruitment Status : Not yet recruiting
First Posted : October 2, 2018
Last Update Posted : October 2, 2018
Sponsor:
Information provided by (Responsible Party):
AMO Pharma Limited

Tracking Information
First Submitted Date  ICMJE March 16, 2018
First Posted Date  ICMJE October 2, 2018
Last Update Posted Date October 2, 2018
Estimated Study Start Date  ICMJE October 2018
Estimated Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 26, 2018)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: September 26, 2018)
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
Official Title  ICMJE 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
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).
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Congenital Myotonic Dystrophy
Intervention  ICMJE
  • Drug: Tideglusib
    Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily
  • Drug: Placebo
    Matching placebo formulation
Study Arms  ICMJE
  • Experimental: Tideglusib
    Weight adjusted tideglusib, orally, once daily
    Interventions:
    • Drug: Tideglusib
    • Drug: Placebo
  • Placebo Comparator: Placebo
    Matching placebo, orally, once daily
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: September 26, 2018)
56
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2019
Estimated Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 16 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03692312
Other Study ID Numbers  ICMJE AMO-02-MD-2-003
2016-004623-23 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party AMO Pharma Limited
Study Sponsor  ICMJE AMO Pharma Limited
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Joseph P Horrigan, MD AMO Pharma
PRS Account AMO Pharma Limited
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP