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Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT03692312 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2025-10-08

Study results available
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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).

Conditions

  • Congenital Myotonic Dystrophy

Interventions

DRUG

Tideglusib

Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily

DRUG

Placebo

Matching placebo formulation

Sponsors & Collaborators

  • AMO Pharma Limited

    lead INDUSTRY

Principal Investigators

  • Joseph P Horrigan, MD · AMO Pharma

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2021-03-03
Primary Completion
2023-04-04
Completion
2023-04-04
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • New Zealand
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

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 NCT03692312 on ClinicalTrials.gov