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

NCT ID: NCT04854096

Last Updated: 2025-05-23

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-01-31
• Study Completion Date: 2024-05-16

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).

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

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

Group Type: EXPERIMENTAL

NS-018

Intervention Type: DRUG

Experimental

Best Available Therapy (BAT)

Single agent per Investigator discretion or no therapy

Group Type: ACTIVE_COMPARATOR

Best Available Therapy

Intervention Type: DRUG

Active Comparator

Interventions

NS-018

Experimental

Intervention Type: DRUG

Best Available Therapy

Active Comparator

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Nippon Shinyaku Co., Ltd.

INDUSTRY

Sponsor Role: collaborator

NS Pharma, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University of Massachusetts Chan Medical School

Worcester, Massachusetts, United States

Houston Methodist Hospital

Houston, Texas, United States

MD Anderson Cancer Center

Houston, Texas, United States

Hamatologisch-onkologische Praxis Heinric/Bangerter Ausgsburg GbR

Augsburg, , Germany

Universitaetsklinikum Halle (Saale)

Halle, , Germany

Universitaetsklinikum Jena

Jena, , Germany

Universitätsmedizin Rostock

Rostock, , Germany

AO SS Antonio

Alessandria, , Italy

Azienda Ospedaliera SS. Antonio

Alessandria, , Italy

ASST Spedali Civili di Brescia

Brescia, , Italy

Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia

Brescia, , Italy

AOU "Policlinico - San Marco"

Catania, , Italy

ASST Fatebenefratelli Sacco

Milan, , Italy

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, , Italy

AO di Rilievo Ntl A Cardarelli

Naples, , Italy

Azienda Ospedaliera di Rilievo Nazionale

Naples, , Italy

AO di Rilievo Nazionale

Napoli, , Italy

Azienda Ospedaliera di Padova

Padua, , Italy

Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone

Palermo, , Italy

Istituto Nazionale Tumori Regina Elena IRCCS

Roma, , Italy

Azienda Ospedaliera Universitaria Policlinico Umberto I

Roma, , Italy

AO Uni Policlinico Umberto I

Rome, , Italy

Hospital Raja Permaisuri Bainun

Ipoh, Perak, Malaysia

Hospital Ampang

Ampang, , Malaysia

Hospital Sultanah Aminah

Johor Bahru, , Malaysia

Hospital Raja Perempuan Zainab II

Kota Bharu, , Malaysia

Hospital Queen Elizabeth

Kota Kinabalu, , Malaysia

University Malaya Medical Centre

Kuala Lumpur, , Malaysia

Sunway Medical Centre

Petaling Jaya, , Malaysia

Hospital Pulau Pinang

Pulau Pinang, , Malaysia

Szpital Uniwersytecki nr 2 im. dr J. Biziela

Bydgoszcz, , Poland

Pratia Onkologia Katowice

Katowice, , Poland

Dolnośląskie Centrum Onkologii we Wrocławiu, Oddział Hematologiczny

Wroclaw, , Poland

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

Banpo-dong, , South Korea

Gachon University Gil Medical Center

Incheon, , South Korea

Gyeongsang National University Hospital

Jinju, , South Korea

CHA Bundang Medical Center, CHA University

Seongnam, , South Korea

Soon Chun Hyang Central Medical Center

Seoul, , South Korea

Srinagarind Hospital

Khon Kaen, , Thailand

Songklanagarind Hospital

Songkhla, , Thailand

Istanbul Medipol University

Bağcılar, Istanbul, Turkey (Türkiye)

Ege Universitesi Tip Fak,

Izmir, , Turkey (Türkiye)

Namik Kemal University Medicine School

Tekirdağ, , Turkey (Türkiye)

Karadeniz Teknik Universitesi Tip Fak,

Trabzon, , Turkey (Türkiye)

Royal United Hospitals - Bath

Bath, England, United Kingdom

Guys Hospital

London, England, United Kingdom

University College London Hospitals

London, England, United Kingdom

The Christie NHS Foundation Trust

Manchester, England, United Kingdom

Derriford Hospital

Plymouth, England, United Kingdom

Sandwell & West Birmingham Hospital

West Bromwich, England, United Kingdom

Western General Hospital

Edinburgh, Scotland, United Kingdom

Western General Hospital

Edinburgh, Scotland, United Kingdom

Countries

United States; Germany; Italy; Malaysia; Poland; South Korea; Thailand; Turkey (Türkiye); United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

NS-018-201

Identifier Type: -

Identifier Source: org_study_id