Primary MyelofibrosisPhase 2NCT04854096
Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis
A Phase 2 study testing NS-018 and Best Available Therapy in people with Primary Myelofibrosis and Post-essential Thrombocythemia Myelofibrosis, run by NS Pharma, Inc. at 52 sites in 9 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis
- What is being tested?
- NS-018 and Best Available Therapy
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 52 sitesin 9 countries
- Study dates
- 31 Jan 2023to 16 May 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
52 sites in 9 countries.
52 sites in 9 countries
Italy15 sites
AO SS Antonio
Alessandria, 15121
Azienda Ospedaliera SS. Antonio
Alessandria, 15121
ASST Spedali Civili di Brescia
Brescia, 25123
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Brescia, 25123
AOU "Policlinico - San Marco"
Catania, 95123
ASST Fatebenefratelli Sacco
Milan, 20121
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, 20122
AO di Rilievo Nazionale
Naples, 80131
AO di Rilievo Ntl A Cardarelli
Naples, 80131
Azienda Ospedaliera di Rilievo Nazionale
Naples, 80131
Azienda Ospedaliera di Padova
Padova, 35128
Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone
Palermo, 90127
Istituto Nazionale Tumori Regina Elena IRCCS
Roma, 00144
Azienda Ospedaliera Universitaria Policlinico Umberto I
Roma, 00161
AO Uni Policlinico Umberto I
Rome, 00161
Malaysia8 sites
Hospital Ampang
Ampang, 68000
Hospital Raja Permaisuri Bainun
Ipoh, Perak, 30450
Hospital Sultanah Aminah
Johor Bahru, 80100
Hospital Raja Perempuan Zainab II
Kota Bharu, 15586
Hospital Queen Elizabeth
Kota Kinabalu, 88586
University Malaya Medical Centre
Kuala Lumpur, 59100
Sunway Medical Centre
Petaling Jaya
Hospital Pulau Pinang
Pulau Pinang, 10990
United Kingdom8 sites
Royal United Hospitals - Bath
Bath, England, BA1 3NG
Western General Hospital
Edinburgh, Scotland, EH2XU 4
Western General Hospital
Edinburgh, Scotland, EH4 2XU
Guys Hospital
London, England, SE1 9RT
University College London Hospitals
London, England, WC1E 6HX
The Christie NHS Foundation Trust
Manchester, England, M20 4BX
Derriford Hospital
Plymouth, England, PL6 8DH
Sandwell & West Birmingham Hospital
West Bromwich, England, B71 4HJ
South Korea5 sites
The Catholic University of Korea, Seoul St. Mary's Hospital
Banpo-dong, 164 KR
Gachon University Gil Medical Center
Incheon, 21565
Gyeongsang National University Hospital
Jinju, 52727
CHA Bundang Medical Center, CHA University
Seongnam, 164 KR
Soon Chun Hyang Central Medical Center
Seoul, 4401
Germany4 sites
Hamatologisch-onkologische Praxis Heinric/Bangerter Ausgsburg GbR
Augsburg, 86150
Universitaetsklinikum Halle (Saale)
Halle, 6120
Universitaetsklinikum Jena
Jena, 07747
Universitätsmedizin Rostock
Rostock, 18057
Turkey (Türkiye)4 sites
Istanbul Medipol University
Bağcılar, Istanbul, 34200
Ege Universitesi Tip Fak,
Izmir
Namik Kemal University Medicine School
Tekirdağ, 59100
Karadeniz Teknik Universitesi Tip Fak,
Trabzon, 61100
United States3 sites
Houston Methodist Hospital
Houston, Texas, 77002
MD Anderson Cancer Center
Houston, Texas, 77030
University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655
Poland3 sites
Szpital Uniwersytecki nr 2 im. dr J. Biziela
Bydgoszcz, 85-168
Pratia Onkologia Katowice
Katowice, 40-519
Dolnośląskie Centrum Onkologii we Wrocławiu, Oddział Hematologiczny
Wroclaw, 53-413 ,
Thailand2 sites
Srinagarind Hospital
Khon Kaen, 40002
Songklanagarind Hospital
Songkhla, 90110
About this study
Official title: 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).
Read the 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.
- Conditions
- Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis
- Being tested
- NS-018, Best Available Therapy
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NS-018Drug
Experimental
Best Available TherapyDrug
Active Comparator
| Study group | Role | What they receive |
|---|---|---|
| NS-018 | Experimental | Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles Receives: Drug: NS-018 |
| Best Available Therapy (BAT) | Active comparator | Single agent per Investigator discretion or no therapy Receives: Drug: Best Available Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Spleen Volume
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)
Measured: baseline and week 24
Change in Total Symptom Score (TSS)
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
Measured: baseline and week 24
Secondary outcomes (2)
Change in Spleen Volume
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)
Measured: from baseline to anytime before or at week 24
Comparison of Treatment-emergent AEs Between NS-018 and BAT
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.
Measured: from baseline to week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2021
- Study start31 Jan 2023
- Primary completion16 May 2024
- Study completion16 May 2024
Why it stopped: The study was stopped due to a business decision.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary MyelofibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- NS Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04854096
- Sponsor's protocol ID
- NS-018-201
- Status last verified
- May 2025
- First submitted
- 9 Apr 2021
- Last updated
- 23 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Submission to the FDA
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT04854096
- Acronym / sponsor ID
- NS-018-201
- Conditions
- Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was stopped due to a business decision.
- Enrolment
- 7 participants (actual)
- Lead sponsor
- NS Pharma, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Study start
- 31 Jan 2023 (actual)
- Primary completion
- 16 May 2024 (actual)
- Study completion
- 16 May 2024 (actual)
- First posted
- 22 Apr 2021 (actual)
- Results
- Posted 23 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NS-018 | Experimental | Drug: NS-018Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles |
| Best Available Therapy (BAT) | Active comparator | Drug: Best Available TherapySingle agent per Investigator discretion or no therapy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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).
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NS-018Experimental | Drug | NS-018 |
| Best Available TherapyActive Comparator | Drug | Best Available Therapy (BAT) |
Primary outcome
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)
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
2 secondary outcomes
Change in Spleen Volume
Time frame: 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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. Submission to the FDA
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
52 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NS-018 and Best Available Therapy in people with Primary Myelofibrosis and Post-essential Thrombocythemia Myelofibrosis, run by NS Pharma, Inc. at 52 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 52 sites in Italy, Malaysia, United Kingdom, South Korea, Germany and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Spleen Volume (time frame: baseline and week 24). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 May 2025.