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

NCT04854096 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: NS-018-201
• Study Type: interventional
• Target: 7
• Locations: 9 countries
• Sites: 52

Brief 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 the Dynamic International Prognostic Scoring System (DIPSS).

Conditions

Primary Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis; Post-polycythemia Vera Myelofibrosis

Outcome Measures

Primary Outcomes (2)

• 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)

  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

  baseline and week 24

Secondary Outcomes (2)

• Change in Spleen Volume

  from baseline to anytime before or at week 24

• Comparison of Treatment-emergent AEs Between NS-018 and BAT

  from baseline to week 24

Study Arms (2)

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

Drug: NS-018

Best Available Therapy (BAT)

ACTIVE COMPARATOR

Single agent per Investigator discretion or no therapy

Drug: Best Available Therapy

Interventions

NS-018 DRUG

Experimental

NS-018

Best Available Therapy DRUG

Active Comparator

Best Available Therapy (BAT)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• NS Pharma, Inc.: lead
• Nippon Shinyaku Co., Ltd.: collaborator

Study Sites (52)

University of Massachusetts Chan Medical School

Worcester, Massachusetts, 01655, United States

Location

Houston Methodist Hospital

Houston, Texas, 77002, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Hamatologisch-onkologische Praxis Heinric/Bangerter Ausgsburg GbR

Augsburg, 86150, Germany

Location

Universitaetsklinikum Halle (Saale)

Halle, 6120, Germany

Location

Universitaetsklinikum Jena

Jena, 07747, Germany

Location

Universitätsmedizin Rostock

Rostock, 18057, Germany

Location

AO SS Antonio

Alessandria, 15121, Italy

Location

Azienda Ospedaliera SS. Antonio

Alessandria, 15121, Italy

Location

ASST Spedali Civili di Brescia

Brescia, 25123, Italy

Location

Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia

Brescia, 25123, Italy

Location

AOU "Policlinico - San Marco"

Catania, 95123, Italy

Location

ASST Fatebenefratelli Sacco

Milan, 20121, Italy

Location

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

AO di Rilievo Ntl A Cardarelli

Naples, 80131, Italy

Location

Azienda Ospedaliera di Rilievo Nazionale

Naples, 80131, Italy

Location

AO di Rilievo Nazionale

Napoli, 80131, Italy

Location

Azienda Ospedaliera di Padova

Padua, 35128, Italy

Location

Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone

Palermo, 90127, Italy

Location

Istituto Nazionale Tumori Regina Elena IRCCS

Roma, 00144, Italy

Location

Azienda Ospedaliera Universitaria Policlinico Umberto I

Roma, 00161, Italy

Location

AO Uni Policlinico Umberto I

Rome, 00161, Italy

Location

Hospital Raja Permaisuri Bainun

Ipoh, Perak, 30450, Malaysia

Location

Hospital Ampang

Ampang, 68000, Malaysia

Location

Hospital Sultanah Aminah

Johor Bahru, 80100, Malaysia

Location

Hospital Raja Perempuan Zainab II

Kota Bharu, 15586, Malaysia

Location

Hospital Queen Elizabeth

Kota Kinabalu, 88586, Malaysia

Location

University Malaya Medical Centre

Kuala Lumpur, 59100, Malaysia

Location

Sunway Medical Centre

Petaling Jaya, Malaysia

Location

Hospital Pulau Pinang

Pulau Pinang, 10990, Malaysia

Location

Szpital Uniwersytecki nr 2 im. dr J. Biziela

Bydgoszcz, 85-168, Poland

Location

Pratia Onkologia Katowice

Katowice, 40-519, Poland

Location

Dolnośląskie Centrum Onkologii we Wrocławiu, Oddział Hematologiczny

Wroclaw, 53-413 ,, Poland

Location

The Catholic University of Korea, Seoul St. Mary's Hospital

Banpo-dong, 164 KR, South Korea

Location

Gachon University Gil Medical Center

Incheon, 21565, South Korea

Location

Gyeongsang National University Hospital

Jinju, 52727, South Korea

Location

CHA Bundang Medical Center, CHA University

Seongnam, 164 KR, South Korea

Location

Soon Chun Hyang Central Medical Center

Seoul, 4401, South Korea

Location

Srinagarind Hospital

Khon Kaen, 40002, Thailand

Location

Songklanagarind Hospital

Songkhla, 90110, Thailand

Location

Istanbul Medipol University

Bağcılar, Istanbul, 34200, Turkey (Türkiye)

Location

Ege Universitesi Tip Fak,

Izmir, Turkey (Türkiye)

Location

Namik Kemal University Medicine School

Tekirdağ, 59100, Turkey (Türkiye)

Location

Karadeniz Teknik Universitesi Tip Fak,

Trabzon, 61100, Turkey (Türkiye)

Location

Royal United Hospitals - Bath

Bath, England, BA1 3NG, United Kingdom

Location

Guys Hospital

London, England, SE1 9RT, United Kingdom

Location

University College London Hospitals

London, England, WC1E 6HX, United Kingdom

Location

The Christie NHS Foundation Trust

Manchester, England, M20 4BX, United Kingdom

Location

Derriford Hospital

Plymouth, England, PL6 8DH, United Kingdom

Location

Sandwell & West Birmingham Hospital

West Bromwich, England, B71 4HJ, United Kingdom

Location

Western General Hospital

Edinburgh, Scotland, EH2XU 4, United Kingdom

Location

Western General Hospital

Edinburgh, Scotland, EH4 2XU, United Kingdom

Location

MeSH Terms

Conditions

Primary Myelofibrosis

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Limitations and Caveats

The study was stopped early for business reasons. Therefore the efficacy outcomes were evaluated only in the limited number of patients enrolled.

Results Point of Contact

• Title: Medical Affairs
• Organization: NS Pharma, Inc.

trialinfo@nspharma.com

+1-201-986-3860

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 9, 2021
• First Posted: April 22, 2021
• Study Start: January 31, 2023
• Primary Completion: May 16, 2024
• Study Completion: May 16, 2024
• Last Updated: May 23, 2025
• Results First Posted: May 23, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

DE (4); GB (8); US (3); TH (2); IT (15); MY (8); KR (5); PL (3); TU (4)