Ongoing (Closed To New Participants)


  • 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
  • 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