Duchenne Muscular Dystrophy Clinical Trials in Philadelphia, Pennsylvania

4 studies are recruiting for duchenne muscular dystrophy in Philadelphia, Pennsylvania right now. Each one is listed on ClinicalTrials.gov, the public registry the research teams themselves file with. TrialBoost is not a doctor, a research site, or a study sponsor: the research team decides who takes part.

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Studies now recruiting

  1. Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

    The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years an…

    Stage
    Not phased
    Ages
    2 and older
    Site
    Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania
    Locations
    33 in total
    Run by
    Catalyst Pharmaceuticals, Inc.

    See if you can join NCT06564974 on ClinicalTrials.gov

  2. A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

    This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to < 7 years o…

    Stage
    Phase 1
    Ages
    0 to 17
    Site
    Children's Hospital of Philadelphia, Philadelphia, Pennsylvania
    Locations
    15 in total
    Run by
    Solid Biosciences Inc.

    See if you can join NCT06138639 on ClinicalTrials.gov

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

    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.

    Stage
    Phase 2
    Ages
    7 and older
    Site
    The Children's Hospital of Philadelphia Colket Translational Research Building, Philadelphia, Pennsylvania
    Locations
    39 in total
    Run by
    Italfarmaco

    See if you can join NCT03373968 on ClinicalTrials.gov

  4. Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

    The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as an objective outcome measure for clinical trials in Muscular Dystrophy (MD). The investigators will compare the muscles…

    Stage
    Not phased
    Ages
    5 to 62
    Site
    Children's Hospital of Philadelphia, Philadelphia, Pennsylvania
    Locations
    3 in total
    Run by
    University of Florida

    See if you can join NCT01484678 on ClinicalTrials.gov

How to read this page. Every study here comes from ClinicalTrials.gov, the public registry that research teams are required to file with. We have summarised each one in plain language and shown where it is running. We have not changed what any study says it is doing.

What TrialBoost is. We help people find research that might suit them, and, if they ask us to, we pass their details to the research team running it. We are not a doctor, a research site, or a study sponsor, and we do not decide who takes part in anything. Joining costs nothing and you can change your mind at any point.

Paid placement. Some studies pay us when someone asks to be connected to them. Those are labelled Sponsored wherever they appear, and they only appear when they match what you were actually looking for.