• A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

    Brief Description: For details, please refer to
    Sponsor: Santhera Pharmaceuticals (Switzerland) Ltd. (Formerly ReveraGen)
  • Addressing Sleep Health in the Battle Against Duchenne Muscular Dystrophy: A Transdiagnostic Intervention

    Brief Description: This is a Phase 2a/2b intervention study exploring the effectiveness of a caregiver-led pediatric/adolescent sleep intervention for individuals with Duchenne muscular dystrophy (DMD) who are between the ages of 6 and 17 years old. Caregivers and boys will be asked to participate in 10 weekly web-based (Video) intervention sessions with a trained clinician. During these sessions, the clinician works with families to learn and use the essential elements of an evidence-based sleep intervention, including education regarding sleep, setting goals, problem solving, sleep scheduling, and monitoring. Throughout the study, participants are asked to complete a sleep diary, along with knowledge checks about what they are learning, and questionnaires about sleep habits. Boys with DMD will be asked to wear an ActiGraph (at least 10 hours per day and throughout the night) and a heart rate monitor (throughout the night) for up to 14 weeks (baseline, intervention, post-intervention). We are exploring the effects of the intervention to improve sleep health and potentially mitigate the physical and behavioral effects of poor sleep health in these boys. Compensation is being provided. To learn more about study eligibility, visit Sponsor: Medical University of South Carolina, College of Health Professions
  • VBP15-BMD-001: A phase II pilot trial of Vamorolone vs. placebo for the treatment of Becker muscular dystrophy

    Brief Description: VBP15-BMD-001 is a research study of an investigational drug called Vamorolone. Other studies conducted on boys with Duchenne dystrophy have shown that Vamorolone has beneficial effects with lower side effects than traditional glucocorticoid treatment. The goal of this study is to determine the potential treatment for patients with Becker dystrophy (BMD).  To participate in this study, you must be 18 years or older (up to age 65 years) and have a confirmed genetic diagnosis of BMD. You should also be able to walk about 11 yards in less than 30 seconds, have no cardiac symptoms, and have not received any corticosteroid treatment for the past 3 months. The study involves an optional virtual pre-screening visit, and 7 in-person visits over 33 weeks and consists of 3 parts:
    • First, you will have a pre-screening and/or a screening visit. If you are eligible to participate in the study based on the results of your screening visit, you will be randomly assigned to one of the study groups. Group #1 will receive Vamorolone, and group #2 will receive an inactive drug called ‘placebo.’
    • Second, you will receive study medication for 24 weeks. During this treatment period, you will have 4 study visits.
    • When you finish your treatment period, you will safely stop taking study products by progressively reducing your dose. You will complete one last visit.
    Participating in this research study involves different tests, including physical exams, labs, motor function tests, and electrocardiograms. This study will be run at the University of Pittsburgh in Pittsburgh, PA. The sponsor will reimburse you for any reasonable travel expenses (travel, lodging, food, and other direct costs) related to your study visits. To learn more about this research study: (NCT05166109) OR please contact the study coordinator: Gabi Niizawa - - tel:412-383 9775. Si desea participar en este estudio o desea recibir información en español, por favor, comuníquese con la coordinadora del estudio Gabi Niizawa. Correo electrónico: Tel.: 412-383 9775.