NS-018 : Phase I/II study

Official Title A Phase I/II, Open-label, Dose-Escalation Multi-center Study to Assess the Safety, Tolerability, PK and PD of Orally Administered NS-018 in Patients With Primary Myelofibrosis (MF), Post-polycythemia Vera MF, or Post-essential Thrombocythemia MF
Purpose To determine the safety and tolerability of orally administered NS-018 in patients with Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (post-PV MF), or Post-essential Thrombocythemia Myelofibrosis (post-ET MF)
click here
Location 9 sites in the US.
Houston (TX), Scottsdale (AZ), Chicago (IL), Boston (MA), San Diego (CA), Ann Arbor (MI), Jacksonville (FL), New York (NY)
Publications click here

For more information, please visit ClinicalTrials.gov (Identifier# : NCT01423851)

NS-065/NCNP-01 : Phase II study

Official Title 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)
Purpose The main objective of this study is to evaluate the safety of high and low dose NS-065/NCNP-01 injection delivered as an intravenous infusion in patients with Duchenne muscular dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.
Approximately 16 patients (4 to <10 years of age) with DMD amenable to exon 53 skipping.
Study design
Low Dose GroupHigh Dose Group
  • All patients will receive NS-065/NCNP-01
  • Muscle biopsy is conducted twice during the study
  • 80 mg/kg cohort will be initiated after safety of 40mg/kg is confirmed (at week 5)
Summary of
inclusion criteria
  • Patient has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53
  • Patient is ≥4 years and <10 years of age
  • Patient is able to walk independently without assistive devices
  • Patient is able to complete strength and function tests
  • Patient must be on a stable dose of glucocorticoid for at least 3 months prior to study entry
Summary of
exclusion criteria
  • Patient has evidence of symptomatic cardiomyopathy.
  • Patient has an allergy or hypersensitivity to the study medication
  • Patient is taking any other investigational drug currently or within 3 months prior to the start of the study treatment
  • Patient has had surgery within the 3 months prior to the first anticipated administration of the study medication
  • Patient has positive test results for HB antigen, HCV antibody or HIV antibody
Location 7 sites in North America.
St. Louis (MO), Sacramento (CA), Gainesville (FL), Richmond (VA), Chicago (IL), Durham (NC), Calgary (Canada)
Publications click here

For more information, please contact Lauren Morgenroth at 412-224-2030 or trialinfo@nspharma.com or visit ClinicalTrials.gov (NCT02740972,NCT03167255)