Myopathy clinical trials at UCLA
5 in progress, 1 open to eligible people
Understand How the Study Medicine (PF-06823859) Works in People With Active Idiopathic Inflammatory Myopathies [Dermatomyositis (DM) and Polymyositis (PM)]
open to eligible people ages 18 years and up
The purpose of the study is to understand how the study medicine PF-06823859 works in people with idiopathic inflammatory myopathies (DM and PM). These disorders cause inflammation that weakens the muscles that are important for movement and may also cause skin rash in people with DM. This study is seeking participants who: - Are 18 years of age or older or minimum legal adult age as defined per local regulation, whichever is greater - Have active DM or active PM. - Are receiving a stable dose of 1 corticosteroid taken by mouth and/or 1 traditional immunosuppressant. - Note: Corticosteroids and immunosuppressants are medicines that help reduce inflammation and may signal to the immune system not to attack the body. Dermatomyositis (DM) is a rare disease that causes muscle inflammation that results in muscle weakness and low muscle stamina. Patients with DM have a characteristic skin rash. Polymyositis (PM) is a rare disease that involves mainly muscle inflammation resulting in muscle weakness, that can sometimes be painful. Patients with DM and PM may have trouble going up the steps, walking or getting to a standing position. Some of the participants will receive the study medicine (PF-06823859) and some will receive placebo (which is similar to study medicine but contains no medicine in it). The study medicine or placebo will be given as an intravenous (IV) infusion (directly into the veins), which takes about1 hour; every 4 weeks from Day 1 to Week 48 of the study. Both PF-06823859 and placebo and will be given at the study site. The study will compare the experiences of people receiving study medication to those of the people who do not. This will help to see if PF-06823859 is safe and effective. Participants will take part in this study for about 13 months. During this time, participants will have 15 study visits. These visits will be performed at the study site.
Los Angeles, California and other locations
Nipocalimab in Participants With Active Idiopathic Inflammatory Myopathies
Sorry, in progress, not accepting new patients
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
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 was a multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and efficacy of resamirigene bilparvovec in participants with X-Linked Myotubular Myopathy aged less than 5 years old. Participants received a single dose of resamirigene bilparvovec and followed for safety and efficacy for 10 years
Los Angeles, 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
Our lead scientists for Myopathy research studies include Perry Shieh.
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