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Duchenne Muscular Dystrophy clinical trials at UC Health

24 in progress, 6 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.

    at UC Davis

  • 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.

    at UC Davis

  • Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

    open to eligible males ages 5 years and up

    This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

    at UC Davis UCSF

  • Open-label Extension of the HOPE-2 Trial

    open to eligible people ages 10 years and up

    This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of four intravenous administrations of CAP-1002, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of CAP-1002. Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9 and 12. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6 and 9 trial visits, unless otherwise indicated. All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6 and 9. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day if medically cleared by the site Investigator.

    at UC Davis

  • Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy

    open to eligible males ages 16-59

    This is a Phase 1, open-label, dose escalation study aimed at evaluating the safety, early efficacy and potential biomarkers of (+)-epicatechin in patients with Becker or Becker-like Muscular Dystrophy (BMD).

    at UC Davis UCLA

  • Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD

    open to eligible males ages 7-21

    This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in heart health over time in boys with DMD. Currently, there is a lack of sufficiently well characterized cardiac MRI biomarkers that can serve as endpoints for detecting on-target and/or off-target cardiac effects during clinical drug trials for boys with DMD. Consequently, the first objective is to identify and characterize several cardiac MRI biomarkers for boys with DMD.

    at UCLA

  • A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)

    Sorry, not currently recruiting here

    To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).

    at UCLA

  • A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).

    at UCLA

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

    Sorry, accepting new patients by invitation only

    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.

    at UC Davis UCLA

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

    Sorry, in progress, not accepting new patients

    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

    at UC Davis UCLA

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

    Sorry, in progress, not accepting new patients

    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.

    at UC Davis UCLA

  • A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

    Sorry, in progress, not accepting new patients

    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.

    at UC Davis UCLA

  • A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

    Sorry, accepting new patients by invitation only

    This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function. Two dose cohorts are planned with up to 6 subjects for each. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between the two cohorts and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.

    at UCLA

  • An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

    Sorry, accepting new patients by invitation only

    The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).

    at UCLA

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

    Sorry, accepting new patients by invitation only

    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.

    at UC Davis

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

    Sorry, in progress, not accepting new patients

    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.

    at UC Davis UCLA UCSD

  • Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

    Sorry, not accepting new patients

    The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.

    at UC Davis UCLA

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

    Sorry, not accepting new patients

    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

    at UC Davis

  • Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

    Sorry, in progress, not accepting new patients

    This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 168 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

    at UC Davis

  • Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy

    Sorry, not currently recruiting here

    Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cases each year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause of death. As there are yet no specific cardiac treatments to extend life, the current study aims to address this unmet medical need using a new therapeutic strategy for patients with DMD. Funding Source - FDA OOPD

    at UCLA

  • Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    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.

    at UC Davis

  • Study of SRP-4045 and SRP-4053 in DMD Patients

    Sorry, not currently recruiting here

    The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

    at UC Davis UCLA UCSD

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

    Sorry, not currently recruiting here

    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.

    at UC Davis

  • Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

    Sorry, in progress, not accepting new patients

    This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory subjects with DMD.

    at UCLA UCSF

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