Myopathy clinical trials at UCLA

4 in progress, 1 open to eligible people

Showing trials for

  • Nipocalimab in Participants With Active Idiopathic Inflammatory Myopathies

    open to eligible people ages 18 years and up

    The purpose of this study is to evaluate the efficacy and safety of Nipocalimab versus placebo in participants with active idiopathic inflammatory myopathies (IIM).

    Torrance, California and other locations

  • ManNAc for GNE Myopathy

    Sorry, in progress, not accepting new patients

    GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation. Currently, there is no therapy available for this disease. N-Acetylmannosamine (ManNAc), an orphan drug in development for GNE myopathy, is an uncharged monosaccharide and the first committed precursor in Neu5Ac biosynthesis. In this randomized, double-blind, placebo-controlled trial the efficacy and long-term safety of ManNAc will be evaluated in subjects with GNE myopathy.

    Los Angeles, California and other locations

  • 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

    Los Angeles, California and other locations

  • Gene Transfer Clinical Study in X-Linked Myotubular Myopathy

    Sorry, in progress, not accepting new patients

    This is a multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and efficacy of AT132 in subjects with X-Linked Myotubular Myopathy aged less than 5 years old. Subjects will receive a single dose of AT132 and will be followed for safety and efficacy for 10 years

    Los Angeles, California and other locations

Our lead scientists for Myopathy research studies include .

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