The Cooperative International
Neuromuscular Research Group

Ongoing (Closed To New Participants)


  • Expanded Access Protocol for Boys with Duchenne Muscular Dystrophy

    Brief Description: For details, please refer to https://www.clinicaltrials.gov/ct2/show/NCT03863119
    Sponsor: Santhera Pharmaceuticals (Switzerland) Ltd. (Formerly ReveraGen)

  • 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.

  • 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).