Safety and Tolerability Study of Oral NS-018 in Patients With Primary Myelofibrosis (MF), Post-polycythemia Vera MF or Post-essential Thrombocythemia MF

This study is currently recruiting participants.
Verified February 2014 by NS Pharma, Inc.
Information provided by (Responsible Party):
NS Pharma, Inc. Identifier:
First received: August 15, 2011
Last updated: February 19, 2014
Last verified: February 2014

The purpose of this study is 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)

Condition Intervention Phase
Primary Myelofibrosis
Post-Polycythemic Vera Myelofibrosis
Post-Essential Thrombocythemia Myelofibrosis
Drug: NS-018
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 1/2, 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

Resource links provided by NLM:

Further study details as provided by NS Pharma, Inc.:

Primary Outcome Measures:
  • To evaluate safety and tolerability of NS-018 assessed by the type, frequency, seriousness and intensity of adverse events [ Time Frame: Throughout the study until 30 days after the last dose of study drug (patients with disease progression or no clinical benefit after 6 cycles [168 days] will be discontinued from the study) ] [ Designated as safety issue: Yes ]
    Phase 1 and Phase 2

  • To establish maximum tolerated dose of NS-018 [ Time Frame: Cycle 1 (28 days) ] [ Designated as safety issue: Yes ]
    Phase 1

  • To evaluate response to NS-018 treatment using the International Working Group (IWG) consensus criteria, change in spleen size and bone marrow assessment [ Time Frame: Day 29, 57, 85, and every 84 days thereafter ] [ Designated as safety issue: No ]
    Phase 2

Secondary Outcome Measures:
  • To determine the pharmacokinetic parameters of NS-018 (Cmax, Tmax, AUC, t1/2 and accumulation ratio) [ Time Frame: Up to 24 hours post-dose on Day 1, 8, 15, 29 ] [ Designated as safety issue: No ]
    Phase 1 and Phase 2

  • To evaluate pharmacodynamic correlates of NS-018 [ Time Frame: Day 1, 8, 15, 29, 57, 85, and every 84 days thereafter ] [ Designated as safety issue: No ]
    Phase 1 and Phase 2

  • To evaluate quality of life assessments using Myelofibrosis Symptom Assessment Form (MF-SAF) [ Time Frame: Day 29, 85, and every 84 days thereafter ] [ Designated as safety issue: No ]
    Phase 1 and Phase 2

Estimated Enrollment: 44
Study Start Date: June 2011
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intervention: Drug: NS-018 Drug: NS-018
Treatment will be administered continuously as oral daily therapy in cycles of 4 weeks in duration (28 day treatment cycles).


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Primary myelofibrosis, post-PV MF, or post-ET MF that requires therapy
  • ≥18 years old
  • ECOG Performance Status of ≤2
  • Estimated life expectancy of ≥12 weeks
  • Male or non-pregnant, non-lactating female patients
  • Serum creatinine of ≤1.5 × the upper limit of normal (ULN)OR estimated creatinine clearance (CrCl) ≥60 ml/min/1.73 m2
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 × the upper limit of normal (ULN) and total bilirubin ≤1.5 × ULN
  • Absolute neutrophil count (ANC) >1000/μL and Platelet count >50,000/μL
  • QTcB ≤480 msec
  • No MF-directed treatment for at least 2 weeks prior to initiation of NS-018

Exclusion Criteria:

  • Active, uncontrolled systemic infection
  • Patients with any unresolved toxicity greater than Grade 1 from previous anticancer therapy
  • Potentially curative therapy is available
  • Currently taking medication that is substantially metabolized by cytochrome P450 (CYP) 1A2 or CYP3A4 or taking medication known to be strong inhibitors or inducers of CYP3A4
  • Patients with a serious cardiac condition within the past 6 months
  • Pregnant or lactating
  • Radiation therapy for splenomegaly within 6 months prior to study entry
  • Splenectomy (Phase 2 portion of the study only)
  • Known HIV positive status
  • Known active hepatitis, a history of viral hepatitis B or hepatitis C, or known positive hepatitis B serologies without a history of immunization
  Contacts and Locations
Please refer to this study by its identifier: NCT01423851

United States, Arizona
Mayo Clinic Scottsdale Recruiting Recruiting
Scottsdale, Arizona, United States, 85259-5499
Contact: Clinical Trials Office - All Mayo Clinic Locations    507-538-7623      
Principal Investigator: Ruben A. Mesa, M.D.         
United States, California
UC San Diego Moores Cancer Center Recruiting
San Diego, California, United States, 92093-0698
Contact: Carla Ichban   
Principal Investigator: Catriona Jamieson, MD, PhD         
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
Contact: Hala Simm    312-695-7559      
Principal Investigator: Brady Stein, MD         
University of Chicago Recruiting
Chicago, Illinois, United States, 60637
Contact: Lisa Pape    773-702-6206   
Contact: Peggy Green    773-7702-0267   
Principal Investigator: Olatoyosi Odenike, MD         
United States, Massachusetts
Dana Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02115
Contact: Sarah Patches   
Principal Investigator: Martha Wadleigh, MD         
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Nabeela Iqbal   
Principal Investigator: Moshe Talpaz, MD         
United States, New York
Weill Cornell Medical College Recruiting
New York, New York, United States, 10021
Contact: Ruth Bauman   
Principal Investigator: Ellen Ritchie, MD         
United States, Texas
MD Anderson Cancer Center, Department of Leukemia Recruiting
Houston, Texas, United States, 77030
Contact: Srdan Verstovsek, M.D., Ph.D.    713-745-3429   
Principal Investigator: Srdan Verstovsek, M.D., Ph.D.         
Sponsors and Collaborators
NS Pharma, Inc.
Principal Investigator: Srdan Verstovsek, M.D., Ph.D. MD Anderson Cancer Center, Houston, TX, 77030
  More Information

No publications provided

Responsible Party: NS Pharma, Inc. Identifier: NCT01423851     History of Changes
Other Study ID Numbers: NS-018-101
Study First Received: August 15, 2011
Last Updated: February 19, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by NS Pharma, Inc.:
post-Essential Thrombocythemia Myelofibrosis
Keywords provided by NS Pharma, Inc.:
JAK2 kinase inhibitor
Myeloproliferative Neoplasms
Primary Myelofibrosis
post-Polycythemia Vera Myelofibrosis
Additional relevant MeSH terms:
Bone Marrow Diseases
Hematologic Diseases
Polycythemia Vera
Thrombocythemia, Essential
post-PV MF
post-ET MF

Additional relevant MeSH terms:
Primary Myelofibrosis
Polycythemia Vera
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Hemorrhagic Disorders processed this record on April 23, 2014