Treatment Trials

3 Clinical Trials for Various Conditions

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      RECRUITING
      Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
      Description

      This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.

      Conditions
      Congenital Myotonic DystrophyChildhood Myotonic DystrophyMyotonic Dystrophy
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      RECRUITING
      DMCRN-02-001: Assessing Pediatric Endpoints in DM1
      Description

      The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and childhood myotonic dystrophy type 1, and develop biomarkers for the condition.

      Conditions
      Congenital Myotonic DystrophyChildhood Myotonic DystrophyCDMCHDM
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      RECRUITING
      Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
      Description

      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
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