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

Recruiting Phase 2 Phase 3 Interventional Study
Congenital Myotonic Dystrophy
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
6 – 45
Sex
Any
Trial phase
Phase 2/3
Study type
Interventional
Purpose
Treatment
Participants needed
76 (estimated)
Sponsor
AMO Pharma Limited · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be conside…
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