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Duchenne Muscular Dystrophy clinical trials at University of California Health

21 in progress, 5 open to eligible people

Showing trials for
  • A Gene Transfer Therapy Study in Participants With Duchenne Muscular Dystrophy

    “Volunteer for research and contribute to discoveries that may improve health care for you, your family, and your community!”

    open to eligible males ages 4-7

    The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

    at UC Davis UCLA

  • A Study of Experimental ASP0367 in Pediatric Male Patients with Duchenne Muscular Dystrophy

    open to eligible males ages 8-16

    The primary purpose of this study is to evaluate the safety and tolerability of ASP0367. This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.

    at UC Davis

  • Does your child have Duchenne Muscular Dystrophy? You may be interested in this investigational drug study!

    “We are looking for kids age 6 to less than 12 years old with DMD to test a new treatment given with corticosteroids”

    open to eligible males ages 6-11

    To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every two weeks in ambulatory subjects with Duchenne muscular dystrophy (age 6 to <12 years).

    at UC Davis

  • Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy

    open to eligible males ages 7 years and up

    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

  • 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.

    at UC Davis

  • A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD)

    Sorry, accepting new patients by invitation only

    This is an open-label gene transfer therapy study evaluating the safety of and expression from SRP-9001 in participants with DMD over 260 weeks.

    at UC Davis

  • 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 three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Patients who are randomized to placebo in Part 1 will have the opportunity for treatment with SRP-9001 in Part 2.

    at UCLA

  • A Research Study of Pamrevlumab or Placebo with Steroids in Duchenne Muscular Dystrophy (DMD)

    Sorry, in progress, not accepting new patients

    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 UC Davis UCLA

  • A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)

    Sorry, not yet accepting patients

    HOPE-3 is a multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.

    at UC Davis

  • 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, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). 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 UCSF

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

    Sorry, in progress, not accepting new patients

    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 and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function. A total of approximately 35 subjects will receive PF-06939926, and these will include both ambulatory and non-ambulatory subjects. Up to 13 subjects may be included in a cohort that includes the concomitant medication, sirolimus. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between two planned dose-levels 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

  • 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 179 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 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, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial ofvamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.

    at UC Davis

  • Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

    Sorry, accepting new patients by invitation only

    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 UCSD

  • Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

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

    at UC Davis UCSF

  • Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

    Sorry, in progress, not accepting new patients

    This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Patients will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 subjects were dosed. Subjects currently enrolling are assigned to active treatment. Control subjects enrolled under original protocol will continue through the study per the original protocol.

    at UCLA

  • Open-label Extension of the HOPE-2 Trial

    Sorry, in progress, not accepting new patients

    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 eight 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, 12, 15, 18, 21 and 24. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18 and 21 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, 9, 12, 15, 18 and 21. 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

  • Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD)

    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 participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

    at UC Davis UCLA UCSD

  • Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants 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 participants with DMD.

    at UCLA UCSF

Our lead scientists for Duchenne Muscular Dystrophy medical studies include .

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