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Duchenne Muscular Dystrophy clinical trials at UC Davis
10 research studies open to eligible people

  • A Study of the Experimental Medicine Edasalonexent in Boys With Duchenne Muscular Dystrophy

    open to eligible males ages 4-7

    The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years of age (up to 8th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD.

    Sacramento, California and other locations

  • A Study of the Experimental Medicine Idebenone For Duchenne Muscular Dystrophy

    open to eligible males ages 10 years and up

    The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids

    Sacramento, California and other locations

  • A Study of the Experimental Medicine Idebenone for Duchenne Muscular Dystrophy (DMD)

    open to eligible males ages 11 years and up

    The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.

    Sacramento, California and other locations

  • A Study of the Safety and Effectiveness of Experimental Medicine Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

    open to eligible males ages 4-7

    Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years with DMD.

    Davis, California and other locations

  • A Study of the Safety, Tolerability, and Pharmacokinetics (drug activity) of Experimental SRP-5051 For Duchenne Muscular Dystrophy

    open to eligible males ages 12 years and up

    The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.

    Sacramento, California and other locations

  • A Study to Improve Treatment and Quality of Life in Patients With Duchenne Muscular Dystrophy

    “Learning more about DMD to improve clinical management”

    open to eligible males ages 2-30

    The purpose of this study is to establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD). In this study, the investigators associated with the Cooperative International Neuromuscular Research Group CINRG) will take a detailed look (for a minimum of eight years) at DMD participant's physical abilities, the medical problems they experience, and how they use health care services. Physical abilities will be compared to a group of healthy controls. The second purpose of this study is to find out whether small, normal differences in the genetic makeup of people with DMD (called "single nucleotide polymorphisms" or "SNPs") affect how their disease progresses and relates to muscle strength/size and steroid response. The third purpose of this study is to study genetic variations associated with DMD. The final purpose of this study is to determine whether certain biomarkers are present in people with DMD and not in healthy controls.

    Sacramento, California and other locations

  • A Study to Test Dystrophin (muscle protein) Levels in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

    open to eligible males ages 2-7

    This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD. The study will evaluate the levels of dystrophin before and after 40 weeks of ataluren therapy using muscle biopsies and two validated assay methods, electrochemiluminescence (ECL) and immunohistochemistry.

    Sacramento, California and other locations

  • An Extension of Study SRP-5051 for Duchenne Muscular Dystrophy

    open to eligible males ages 7 years and up

    The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.

    Kansas City, Kansas and other locations

  • Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

    open to eligible males ages 6-17

    it is a randomised, double blind, parallel group, placebo controlled study. A total of 213 male ambulant subjects will be randomised 2:1 (givinostat:placebo). Subjects will be stratified for their concomitant use of steroids in 4 strata: 1. Deflazacort daily regimen 2. Deflazacort intermittent regimen 3. Other steroids daily regimen 4. Other steroids intermittent regimen. The study duration is planned for 19 months.

    Davis, California and other locations

  • Clinical Trial with Experimental Medicine (BMS-986089) in Ambulatory Boys With Duchenne Muscular Dystrophy

    “Will the new medicine be safe and effective in ambulatory boys with Duchenne Muscular Dystrophy?”

    open to eligible males ages 6-11

    This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

    Sacramento, California and other locations

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