Ignite Creation Date:
2025-12-26 @ 11:08 AM
Ignite Modification Date:
2026-02-22 @ 7:22 PM
Study NCT ID:
NCT03692312
Status:
COMPLETED
Last Update Posted:
2025-10-08
First Post:
2018-03-16
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
Sponsor:
AMO Pharma Limited
Organization:
Study Overview
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 (REACH CDM)
Status:
COMPLETED
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
True
If Expanded Access, NCT#:
NCT07119775
Has Expanded Access, NCT# Status:
AVAILABLE
Acronym:
None
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 Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2016-004623-23 | EUDRACT_NUMBER | None | View |