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Glycogen Synthase Kinase 3 Beta Inhibitor

Tideglusib for 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

Awards & highlights

Study Summary

This trial is testing if a drug called "Dystrophin-2" can help people with a rare disease called "Congenital Myotonic Dystrophy."

Eligible Conditions

Myotonic Dystrophy, Congenital

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 22 to 28 weeks

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)

Secondary outcome measures

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

Do I qualify?Apply below to find out

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Do we have a history of research with Tideglusib?

"Since the initial study of tideglusib in 2021 at Children's Hospital of Eastern Ontario, there have been 18,268 completed trials. There are 3 active studies at this moment, a significant number of which are based in Los Angeles, California."

Answered by AI

Are you currently looking for test subjects who are above the age of 20?

"This particular clinical trial is only taking applications from patients aged 6-16. In total, there are 5 clinical trials for patients under 18 and 10 for patients over 65."

Answered by AI

Are there any unfilled positions for trial participants?

"Yes, this clinical trial is seeking participants. It was first posted on March 3rd, 2021 and was updated most recently on August 11th, 2022."

Answered by AI

How many medical facilities are testing this treatment?

"There are 11 enrolling patients for this clinical trial such as University of California, Los Angeles (UCLA) in Los Angeles, University of Rochester Medical Center in Rochester, and Stanford University in Palo Alto,as well as 11 other locations."

Answered by AI

Does this research build on prior knowledge in this area?

"Presently, Tideglusib is being trialed in 13 cities and 6 countries. The first trial began in 2021 and has completed its Phase 2 & 3. That study, sponsored by AMO Pharma Limited, had a total of 56 participants. Since then, a total of 18268 studies have been completed."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

2021. "Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03692312.