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Duchenne Muscular Dystrophy clinical trials at UC Health
26 in progress, 10 open to new patients

  • A Study of Experimental Cell Therapy (CAP-1002) in Patients With Duchenne Muscular Dystrophy (DMD)

    “Would the study drug (CAP-1002) help improve weakened skeletal and heart muscle caused by DMD?”

    open to eligible males ages 10 years and up

    HOPE-2 is a double-blind 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 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

    at UCLA UC Davis

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

    open to eligible males ages 5-12

    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

  • A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)

    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.

    at UCLA UC Davis

  • An Extension Study To Test the Safety Of Experimental "PF-06252616" In Boys With Duchenne Muscular Dystrophy

    “This study will assess the long-term safety of the study drug in Boys with Duchenne Muscular Dystrophy”

    open to eligible males ages 6-18

    This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose based on their tolerability profile/data from B5161002. This study will not contain a placebo comparator. Subjects will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional capacity evaluations (4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing, six minute walk test and pulmonary function tests) and pharmacokinetic testing.

    at UCLA UC Davis

  • 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.

    at UCLA UC Davis UCSD

  • 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).

    at UCLA 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 UCSF UC Davis

  • Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

    open to eligible males ages 8-17

    This single center open-label pilot study will enroll 15 non-ambulatory children with Duchenne muscular dystrophy at least 8 years of age and who demonstrate pre-clinical cardiomyopathy (defined as a cardiac ejection fraction >55% with abnormal LV strain by cardiac MRI). They will receive (+)-epicatechin at one of three doses during an 8-week dose-ranging study with assessments at baseline, 2 Weeks, 4weeks, and 8 weeks. The study will determine optimal dosing for future cardiac efficacy studies based on serum / plasma biomarker response using follistatin: myostatin ratio, nitrite/nitrate ratio, cardiac troponins and cardiac BNP. Secondary endpoints will include additional biomarker assessments by SOMAscanTM, cardiac functional evaluations by cardiac MRI (LV strain), and echocardiogram (LV strain by speckle tracking) and measures of strength, range of motion and mobility, and clinical safety assessments. Results of secondary endpoint analysis will be used to refine design of subsequent clinical trials powered to detect changes in clinical outcomes.

    at UC Davis

  • 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 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular Dystrophy. Three intravenous (IV) dose levels will be investigated in a within subject dose escalating fashion. Subjects will be randomly assigned to 1 of 3 sequence groups for approximately 96 weeks (2 periods of 48 weeks each). In period 1, two of the sequence groups will receive PF-06252616 and one sequence group will receive placebo. In period 2, the placebo group will switch to PF-06252616 and the two remaining sequence groups will either receive placebo or PF-06252616. Efficacy will be based on an observed mean change from baseline on function (4 stair climb) of PF-06252616 as compared to the placebo at the end of period 1. Period 2 provides an opportunity to evaluate PK. Subjects will receive monthly IV infused doses of either PF-06252616 or placebo and will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional evaluations (pulmonary function testing, 4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing and the six minute walk test), pharmacokinetic testing and pharmacodynamic testing to evaluate changes in muscle volume (MRI).

    at UCLA UC Davis

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

    Sorry, not yet accepting 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 UCLA UC Davis

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

    “Learning more about DMD to improve clinical management”

    Sorry, in progress, not accepting new patients

    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.

    at UC Davis

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

    “This is an optional Extension Study for the NS Pharma exon skipping study: NS-065/NCNP-01-201 for boys with Duchenne muscular dystrophy”

    Sorry, accepting new patients by invitation only

    This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 72 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

    at UC Davis

  • 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

  • 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 UCLA UC Davis

  • Finding the Optimum Regimen for Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.

    at UCLA UC Davis

  • Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

    Sorry, accepting new patients by invitation only

    This long-term extension study is an open-label, multiple‑dose study to evaluate the long‑term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15‑002 Phase IIa and VBP15-003 Phase IIa extension core studies.

    at UC Davis

  • Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from ≥4 to <8 years of age will be enrolled. Edasalonexent is an orally administered small molecule targeted to inhibit activated NF-κB, a molecule that is activated from infancy in DMD and which is central to causing muscle damage and preventing muscle regeneration. Data on magnetic resonance imaging of the lower and upper leg muscles, physical function (including timed function tests) and muscle strength will be studied.

    at UCLA

  • Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

    Sorry, in progress, not accepting new patients

    The main goal of this Phase 3 extension study is to obtain long term safety of ataluren in boys with nonsense mutation dystrophinopathy as determined by adverse events and laboratory abnormalities. The study will also assess changes in physical function, pulmonary function and other important clinical and laboratory measures.

    at UCLA UC Davis

  • PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMD

    Sorry, in progress, not accepting new patients

    A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 (ezutromid) in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (DMD)

    at UCLA

  • Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

    at UCLA UC Davis

  • Study of Ataluren in Previously Treated Participants With 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 UCLA UC Davis

  • Study of Eteplirsen in DMD Patients

    Sorry, in progress, not accepting new patients

    The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).

    at UCLA UC Davis UCSD

  • 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 UCLA UC Davis UCSD

  • 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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