Ongoing (Closed To New Participants)


  • 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
  • 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
  • Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

    Brief Description: The purpose of the research study is to test an angiotensin converting enzyme (ACE) inhibitor, lisinopril, and an antioxidant, coenzyme Q10 (CoQ10), to ameliorate the decline in cardiac muscle function that occurs in muscular dystrophies. To learn more about the study eligibility and participating sites please visit: https://clinicaltrials.gov/ct2/show/NCT01126697.
  • Becker Muscular Dystrophy- A Natural History Study to Predict Efficacy of Exon Skipping

    Brief Description: The purpose of this study is to understand the disease progression and quality of life in patients with BMD. The information collected will help us understand the clinical progression and quality of life that would result from treating individuals with DMD with exon skipping therapies. To learn more about the study eligibility and participating sites please visit: https://clinicaltrials.gov/ct2/show/NCT01539772.