Muscular Dystrophies clinical trials at UC Irvine

4 in progress, 3 open to eligible people

Showing trials for

  • LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

    open to eligible people ages 18-65

    The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of LION-101 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)

    Irvine, California and other locations

  • Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

    open to eligible people ages 4-65

    Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

    Irvine, California and other locations

  • Defining Endpoints in Becker Muscular Dystrophy

    open to eligible males ages 6 years and up

    This is a 24-month, observational study of 80 participants with Becker muscular dystrophy (BMD)

    Orange, California and other locations

  • AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)

    Sorry, not currently recruiting here

    A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

    Irvine, California and other locations

Our lead scientists for Muscular Dystrophies research studies include .

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