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Glycogen Synthase Kinase 3 Beta Inhibitor

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Phase 2 & 3
Waitlist Available
Research Sponsored by AMO Pharma Limited
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 22 to 28 weeks

Summary

This trial is testing tideglusib, a medication, to see if it can help children with a genetic muscle disorder. The study involves children aged 6 to 16 who have Congenital Myotonic Dystrophy. Tideglusib might work by blocking a substance that causes muscle issues.

Eligible Conditions
  • Myotonic Dystrophy, Congenital

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~22 to 28 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 22 to 28 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
Secondary study objectives
10-meter walk-run test
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
Change in Clinical Global Impression- Improvement Scale (CGI-I) scores
+4 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: TideglusibExperimental Treatment2 Interventions
Weight adjusted tideglusib, orally, once daily
Group II: PlaceboPlacebo Group1 Intervention
Matching placebo, orally, once daily
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670
Tideglusib
2016
Completed Phase 3
~160

Find a Location

Who is running the clinical trial?

AMO Pharma LimitedLead Sponsor
2 Previous Clinical Trials
92 Total Patients Enrolled
2 Trials studying Myotonic Dystrophy
92 Patients Enrolled for Myotonic Dystrophy
Joseph P Horrigan, MDStudy DirectorAMO Pharma
1 Previous Clinical Trials
76 Total Patients Enrolled
1 Trials studying Myotonic Dystrophy
76 Patients Enrolled for Myotonic Dystrophy
~12 spots leftby Dec 2025
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