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Myotonic Dystrophy clinical trials at University of California Health

5 in progress, 4 open to eligible people

Showing trials for
  • Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

    open to eligible people ages 6-16

    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).

    at UCLA

  • Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

    open to eligible people ages 18-70

    Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward.

    at UCLA

  • Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

    open to eligible people ages 18-65

    The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years. The secondary objectives of this study are to assess the impact of pitolisant on fatigue, cognitive function and the burden of disease along with assessing the long-term safety and effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.

    at UC Irvine

  • Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

    open to eligible people ages 18-65

    AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA). Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period. Part B is a multiple-ascending dose design with 3 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.

    at UCLA

  • Safety and Efficacy of Tideglusib in Congenital Myotonic Dystrophy

    Sorry, accepting new patients by invitation only

    This is an open-label extension phase 2/3 study for children and adolescents with Congenital Myotonic Dystrophy (Congenital DM1) who participated in and completed the preceding AMO-02-MD-2-003 study.

    at UCLA

Our lead scientists for Myotonic Dystrophy research studies include .

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