Recruiting


  • A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long‑Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

    Brief Description: 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.
    Sponsor: ReveraGen
  • A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)

    Brief Description: This study is an extension of the Phase II Dose Finding study evaluating the exon skipping intervention, NS-065/NCNP-01, targeted for the skipping of exon 53. The study will evaluate the tolerability and the effect of low and high IV doses of NS-065/NCNP-01 after an additional 24-week of weekly infusions. Patients who completed the NS-065/NCCNP-01 trial are eligible to enroll. To learn more about the study eligibility and participating sites please contact us at info@trinds.com.
    Sponsor: NS Pharma
  • Use of Microsoft Bands as an Outcome Measure in Boys with DMD

    Brief Description: This study is using the Microsoft (MS) Band, a portable fitness tracking device that is worn on the wrist like a watch, and a special app, as a secondary outcome measure for boys with Duchenne Muscular Dystrophy (DMD) who are enrolled in the Vamorolone Phase 2a clinical trial. To learn more about the study eligibility please contact Dr. Roxanna Bendixen (bendixen@pitt.edu).
  • Sleep Efficiency in Boys with DMD and their Caregivers

    Brief Description:
    This study requests boys with Duchenne Muscular Dystrophy and their caregivers/parents to wear the Microsoft (MS) Band, a portable fitness tracking device, to objectively explore sleep factors, such as amount of time to achieve sleep, time in various levels of sleep, overall awake times, and movement during the night. In addition, boys with DMD will wear the MS Band throughout the day to record daily activity. To learn more about the study eligibility please contact Dr. Roxanna Bendixen (bendixen@pitt.edu).
  • Use of Microsoft Bands as an Outcome Measure in Boys with DMD

    Brief Description: This study is exploring the use of the Microsoft (MS) Band, a portable fitness tracking device that is worn on the wrist like a watch, and a special app, as an outcome measure for boys with Duchenne Muscular Dystrophy in comparison to unaffected children and youth. The goal of this study is to demonstrate that community-based activity monitoring through the use of the MS Band is feasible and reliable and to establish its potential use in clinical trials in patients with DMD. To learn more about the study eligibility please contact Dr. Roxanna Bendixen (bendixen@pitt.edu).