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.|
|ClinicalTrials.gov Identifier: NCT03692312|
Recruitment Status : Not yet recruiting
First Posted : October 2, 2018
Last Update Posted : February 18, 2020
|Condition or disease||Intervention/treatment||Phase|
|Congenital Myotonic Dystrophy||Drug: Tideglusib Drug: Placebo||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||56 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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 :||April 2020|
|Estimated Primary Completion Date :||July 2021|
|Estimated Study Completion Date :||August 2021|
Weight adjusted tideglusib, orally, once daily
Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily
Matching placebo formulation
Placebo Comparator: Placebo
Matching placebo, orally, once daily
Matching placebo formulation
- 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.
- 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.
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
|United States, California|
|Palo Alto, California, United States, 94304|
|Contact: Tia Nguyen 650-498-8771 email@example.com|
|Principal Investigator: John Day, MD|
|United States, Iowa|
|University of Iowa Hospitals|
|Iowa City, Iowa, United States, 52242|
|Contact: Diane Recker, BSN, RN 319-335-6073 firstname.lastname@example.org|
|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 james_hilbert@URMC.Rochester.edu|
|Contact: Jeanne Dekdebrun 585-276-4611 jeanne_Dekdebrun@URMC.Rochester.edu|
|Principal Investigator: Johanna Hamel, MD|
|Sub-Investigator: Emma Ciafaloni, 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|
|Children's Hospital London Health Sciences Centre (LHSC)|
|London, Ontario, Canada, N6A4G5|
|Contact: Rhiannon Hicks 519-685-8441 email@example.com|
|Principal Investigator: Craig Campbell, MD, MSc, FRCPC|
|Study Director:||Joseph P Horrigan, MD||AMO Pharma|