Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis

May 22, 2025 updated by: NS Pharma, Inc.

A Phase 2b, Open-label, Multicenter, Randomized, Controlled, 2-Arm Study to Assess the Efficacy and Safety of Orally Administered NS-018 Versus Best Available Therapy in Subjects With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis With Severe Thrombocytopenia (Platelet Count <50,000/μL)

This study will enroll male and female subjects who are 18 years of age or older with Primary Myelofibrosis, post-polycythemia Vera Myelofibrosis, or post-essential Thrombocythemia Myelofibrosis with severe thrombocytopenia (platelet count <50,000/µL) including subjects with intermediate-2 or high-risk MF according to the Dynamic International Prognostic Scoring System (DIPSS).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

NS-018 will be self-administered orally at a dose of 300 mg BID. The BAT will be administered according to manufacturer's instructions and Investigator discretion. Subjects will complete study visits at Screening, Day 1 and Day 15 of Cycle 1, 2, 3, 4, 5, 6 and Day 1 of every cycle thereafter. At these visits, blood/urine sampling, spleen measurements, bone marrow assessments, patient-reported outcome (PRO) assessments, and safety assessments may be performed.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
7

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Germany
      • Augsburg, Germany, 86150
        • Hamatologisch-onkologische Praxis Heinric/Bangerter Ausgsburg GbR
      • Halle, Germany, 6120
        • Universitaetsklinikum Halle (Saale)
      • Jena, Germany, 07747
        • Universitaetsklinikum Jena
      • Rostock, Germany, 18057
        • Universitätsmedizin Rostock
  • Italy
      • Alessandria, Italy, 15121
        • Azienda Ospedaliera SS. Antonio
      • Alessandria, Italy, 15121
        • AO SS Antonio
      • Brescia, Italy, 25123
        • Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
      • Brescia, Italy, 25123
        • ASST Spedali Civili di Brescia
      • Catania, Italy, 95123
        • AOU "Policlinico - San Marco"
      • Milano, Italy, 20122
        • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
      • Milano, Italy, 20121
        • Asst Fatebenefratelli Sacco
      • Naples, Italy, 80131
        • AO di Rilievo Ntl A Cardarelli
      • Naples, Italy, 80131
        • Azienda Ospedaliera di Rilievo Nazionale
      • Napoli, Italy, 80131
        • AO di Rilievo Nazionale
      • Padova, Italy, 35128
        • Azienda Ospedaliera di Padova
      • Palermo, Italy, 90127
        • Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone
      • Roma, Italy, 00144
        • Istituto Nazionale Tumori Regina Elena IRCCS
      • Roma, Italy, 00161
        • Azienda Ospedaliera Universitaria Policlinico Umberto I
      • Rome, Italy, 00161
        • AO Uni Policlinico Umberto I
  • Korea, Republic of
      • Banpo-dong, Korea, Republic of, 164 KR
        • The Catholic University of Korea, Seoul St. Mary's Hospital
      • Incheon, Korea, Republic of, 21565
        • Gachon University Gil Medical Center
      • Jinju-si, Korea, Republic of, 52727
        • Gyeongsang National University Hospital
      • Seongnam, Korea, Republic of, 164 KR
        • CHA Bundang Medical Center, CHA University
      • Seoul, Korea, Republic of, 4401
        • Soon Chun Hyang Central Medical Center
  • Malaysia
      • Ampang, Malaysia, 68000
        • Hospital Ampang
      • Johor Bahru, Malaysia, 80100
        • Hospital Sultanah Aminah
      • Kota Bahru, Malaysia, 15586
        • Hospital Raja Perempuan Zainab II
      • Kota Kinabalu, Malaysia, 88586
        • Hospital Queen Elizabeth
      • Kuala Lumpur, Malaysia, 59100
        • University Malaya Medical Centre
      • Petaling Jaya, Malaysia
        • Sunway Medical Centre
      • Pulau Pinang, Malaysia, 10990
        • Hospital Pulau Pinang
    • Perak
      • Ipoh, Perak, Malaysia, 30450
        • Hospital Raja Permaisuri Bainun
  • Poland
      • Bydgoszcz, Poland, 85-168
        • Szpital Uniwersytecki nr 2 im. dr J. Biziela
      • Katowice, Poland, 40-519
        • Pratia Onkologia Katowice
      • Wrocław, Poland, 53-413 ,
        • Dolnośląskie Centrum Onkologii we Wrocławiu, Oddział Hematologiczny
  • Thailand
      • Khon Kaen, Thailand, 40002
        • Srinagarind Hospital
      • Songkla, Thailand, 90110
        • Songklanagarind Hospital
  • Turkey
      • Tekirdağ, Turkey, 59100
        • Namik Kemal University Medicine School
      • Trabzon, Turkey, 61100
        • Karadeniz Teknik Universitesi Tip Fak,
      • İzmir, Turkey
        • Ege Universitesi Tip Fak,
    • Istanbul
      • Bagcilar, Istanbul, Turkey, 34200
        • Istanbul Medipol University
  • United Kingdom
    • England
      • Bath, England, United Kingdom, BA1 3NG
        • Royal United Hospitals - Bath
      • London, England, United Kingdom, SE1 9RT
        • Guys Hospital
      • London, England, United Kingdom, WC1E 6HX
        • University College London Hospitals
      • Manchester, England, United Kingdom, M20 4BX
        • The Christie NHS Foundation Trust
      • Plymouth, England, United Kingdom, PL6 8DH
        • Derriford Hospital
      • West Bromwich, England, United Kingdom, B71 4HJ
        • Sandwell & West Birmingham Hospital
    • Scotland
      • Edinburgh, Scotland, United Kingdom, EH4 2XU
        • Western General Hospital
      • Edinburgh, Scotland, United Kingdom, EH2XU 4
        • Western General Hospital
  • United States
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Chan Medical School
    • Texas
      • Houston, Texas, United States, 77030
        • MD Anderson Cancer Center
      • Houston, Texas, United States, 77002
        • Houston Methodist Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Primary MF, post-PVMF or post-ETMF according to the DIPSS risk categories of intermediate-2 or high-risk MF
  • Average platelet count of <50,000/µL at Screening based on 2 measurements taken on different days; both measurements must be <50,000/µL.
  • ECOG performance status ≤2.
  • Life expectancy >6 months.
  • Spleen volume of at least 450 cm3 measured by MRI (or by CT for applicable subjects).
  • Total Symptom Score (TSS) ≥10 on the Myelofibrosis Symptom Assessment Form (MFSAF) version 4.0.
  • Peripheral blast count <10%.
  • No MF-directed treatment for at least 2 weeks prior to initiation of NS-018, including JAK inhibitor, erythropoietic, thrombopoietic agent, or any use of corticosteroids for MF symptom or blood count management. Low dose corticosteroids <10 mg/day prednisone or equivalent is allowed for non-MF purposes.

Exclusion Criteria:

  • Active, uncontrolled systemic infection.
  • Any prior treatment with more than two JAK inhibitors.
  • Previous treatment with NS-018.
  • Subjects actively receiving a concurrent investigational agent.
  • Subjects with any unresolved AE greater than Grade 1 other than hematological AEs from previous anticancer therapy.
  • Currently taking medication that is substantially metabolized by cytochrome P450 (CYP) 1A2 or CYP3A4 (see Appendix 5) or taking medication known to be strong inhibitors or inducers of CYP3A4 (see Appendix 5).
  • Radiation therapy for splenomegaly within 6 months prior to study entry (screening).
  • History of splenectomy or planning to undergo splenectomy.
  • Subjects with a serious cardiac condition within the past 6 months such as uncontrolled arrhythmias, myocardial infarction, angina or heart disease
  • Subjects diagnosed with another malignancy within 2 years prior to an enrollment.
  • Subjects who have had surgery (other than placement of vascular access and bone marrow biopsy) within 4 weeks of study entry (screening), or subjects with incomplete recovery from any prior surgical procedures.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: NS-018
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
Drug: NS-018
Experimental
Active Comparator: Best Available Therapy (BAT)
Single agent per Investigator discretion or no therapy
Drug: Best Available Therapy
Active Comparator

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change in Spleen Volume
Time Frame: baseline and week 24
Proportion of subjects who achieve ≥35% reduction in spleen volume from baseline compared to Week 24 as measured by MRI (or by CT for applicable subjects)
baseline and week 24
Change in Total Symptom Score (TSS)
Time Frame: baseline and week 24
Proportion of subjects who achieve ≥50% reduction in total symptom score from baseline compared to Week 24 as measured by the MF-SAF v4.0
baseline and week 24

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change in Spleen Volume
Time Frame: from baseline to anytime before or at week 24
Proportion of subjects in NS-018 vs BAT arm who achieve ≥35% reduction in spleen volume from baseline at any time up to Week 24 as measured by MRI (or by CT for applicable subjects)
from baseline to anytime before or at week 24
Comparison of Treatment-emergent AEs Between NS-018 and BAT
Time Frame: from baseline to week 24
Laboratory events graded by the NCI CTCAE v5.0 will be assessed in both arms, to compare the safety profile of NS-018 versus BAT.
from baseline to week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
NS Pharma, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Nippon Shinyaku Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 31, 2023

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 16, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 16, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 9, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 20, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 22, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 23, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 22, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NS-018-201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Submission to the FDA

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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