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Duchenne Muscular Dystrophy clinical trials at UC Davis

9 research studies open to eligible people

Showing trials for
  • A Long-Term Safety and Tolerability Study of Experimental Givinostat in Duchenne Muscular Dystrophy

    open to eligible males ages 7 years and up

    This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

    Sacramento, California and other locations

  • A Study of Experimental Ataluren for Nonsense Mutation Dystrophinopathy (nmDBMD)

    open to eligible males

    The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan. The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.

    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

  • An Open-Label Extension Study of Experimental Edasalonexent in Boys With Duchenne Muscular Dystrophy (DMD)

    open to eligible males ages 4-12

    The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-12 years of age (up to 13th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is a 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

  • 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

  • Expanded Access to Experimental Vamorolone for Boys With Duchenne Muscular Dystrophy

    open to eligible males

    The intent of this protocol is to provide continued access to vamorolone for subjects in the United States who Have Completed the VBP15-LTE or VBP15- 004 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review

    Davis, California and other locations

  • Study to Test the Effectiveness and Safety of Experimental Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

    open to eligible males ages 4-7

    The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.

    Sacramento, California and other locations

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