Myotonic Dystrophy clinical trials at University of California Health

6 in progress, 3 open to eligible people

Showing trials for

  • PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

    open to eligible people ages 18-60

    The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

    at UC Irvine

  • DMCRN-02-001: Assessing Pediatric Endpoints in DM1

    open to eligible people ages up to 59 months

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

    at UCLA

  • Estab 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. Funding Source- FDA OOPD

    at UCLA UCSD

  • Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

    Sorry, in progress, not accepting new patients

    AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients

    at UCLA

  • Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

    Sorry, accepting new patients by invitation only

    A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

    at UCLA

  • Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

    Sorry, not currently recruiting here

    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.

    at UCLA

Our lead scientists for Myotonic Dystrophy research studies include .

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