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 IIb Randomized, Double-blind, Parallel Placebo- and Active-controlled Study to Assess the Efficacy and Safety of Vamorolone in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD)

    Brief Description: The study is a Phase IIb randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, and pharmacodynamics of vamorolone in boys with DMD. To learn more about the study eligibility and participating sites, please visit https://clinicaltrials.gov/ct2/show/NCT03439670  or contact us at info@trinds.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.    
  • Use of a Powered Arm Support Device for Upper Limb Function in Non-Ambulatory Men with Duchenne Muscular Dystrophy

    Brief Description: Brief Description:  This study is a longitudinal, randomized comparison trial evaluating the use of two commercially available dynamic arm support devices (Armon Ayura-O540 and JAECO WREX) to promote participation in activities of daily living in non-ambulatory individuals with Duchenne muscular dystrophy (DMD) with upper extremity weakness. Up to 30 individuals will be enrolled to participate in this research study, which includes a two-week baseline data collection period, a four-week device trial and a two-week post device data collection period. Participants will be randomly assigned to trial one of the dynamic arm support devices during the four week in-home trial, with the potential for a cross-over trial with the non-randomized device. The ActiGraph, a wrist worn activity monitoring device, will be worn during the baseline period, the device trial and the post device data collection period to capture UE movement patterns. UE performance will be further quantified with use of a physical motor assessment, the Performance of Upper Limb (PUL) assessment and patient reported outcomes. Data gleaned will provide important knowledge and objective results regarding the potential benefit of dynamic arm supports in individuals with DMD with limited functional use of their upper extremities. To learn more about the study eligibility and participating sites, please visit https://clinicaltrials.gov/ct2/show/NCT03531788  or contact Dr. Roxanna Bendixen (bendixen@pitt.edu) for additional information.
  • Community Based Outcome Measures in Boys with DMD

    Brief Description: Participants are being invited to take part in this parallel research study if they are enrolled in VBP15 2b clinical trial for boys with Duchenne Muscular Dystrophy (DMD). This study requests boys enrolled in VBP 15 wear the ActiGraph GT9x, a wrist-worn portable fitness tracking device, to objectively explore sleep factors and daily physical activity throughout the clinical trial. Patient reported outcomes regarding sleep and daily participation will also be collected. 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 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).