Ongoing (Closed To New Participants)


  • An Open-Label, Expanded Access Treatment Protocol to Provide Access to Vamorolone Treatment for Boys with Duchenne Muscular Dystrophy who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 Studies

    Brief Description: This study is intended to provide continued access to vamorolone for subjects in the United States who have completed the VBP15-LTE or VBP15-004 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review. Participants who have completed the Long Term Extension study or Phase IIb study are eligible to enroll. To learn more about the study eligibility and participating sites please contact Reveragen at laurie.conklin@reveragen.com.
  • 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 visit https://clinicaltrials.gov/ct2/show/NCT03167255 or contacts us at info@trinds.com.    
  • 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 ActiGraph GT9x, a wrist-worn 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 ActiGraph throughout the day to record daily activity. 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 or contact us at info@trinds.com.