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Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

NCT05004129 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 76

Last updated 2025-05-28

No results posted yet for this study

Summary

This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

Conditions

  • Congenital Myotonic Dystrophy

Interventions

DRUG

Tideglusib

Tideglusib dosing will be weight-adjusted at 400 mg, 600 mg, or 1000 mg dose levels, or weight banded fixed doses of 400 mg, 600 mg, 800 mg or 1000 mg, with each subject starting at a weight-adjusted 400 mg dose level for 2 weeks, then up titrating to a weight-adjusted 600 mg dose level for the next 2 weeks.

Sponsors & Collaborators

  • AMO Pharma Limited

    lead INDUSTRY

Principal Investigators

  • Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM · AMO Pharma

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-08-23
Primary Completion
2026-12-31
Completion
2026-12-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • New Zealand

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05004129 on ClinicalTrials.gov