Recruiting


  • A Phase II, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)

    Brief Description: This study is evaluating the safety and tolerability of a low and high dose exon skipping intravenous intervention in patients with Duchenne muscular dystrophy (DMD). The exon skipping intervention, NS-065/NCNP-01, is targeted for the skipping of exon 53. The study is seeking DMD boys aged 4 to less than 10 years old with a mutation amenable to exon 53 skipping. Approximately 16 patients will be enrolled in sites in the United States. To learn more about the study eligibility and participating sites please visit: https://clinicaltrials.gov/ct2/show/NCT02740972 or contact us at info@trinds.com.
    Sponsor: NS Pharma
  • A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)

    Brief Description: This study is evaluating the safety and tolerability of a new steroid-like intervention called Vamorolone in boys with DMD ages 4 to less than 7 years old. Approximately 48 patients will be enrolled in participating sites in the United States, Canada, Australia, Israel, Sweden, and the United Kindgom. To learn more about the study eligibility and participating sites please visit: https://clinicaltrials.gov/ct2/show/NCT02760264 or contact us at info@trinds.com.
    Sponsor: ReveraGen
  • 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).
  • A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)

    Brief Description: This study is evaluating the long-term safety and tolerability of a new steroid-like intervention called vamorolone in boys with DMD ages 4 to less than 7 years oldParticipants who have completed the Phase IIa study (described above) are eligible to enroll. To learn more about the study eligibility and participating sites please visit:https://clinicaltrials.gov/ct2/show/NCT02760277?term=vamorolone&rank=2 or contact us at info@trinds.com.
    Sponsor: ReveraGen