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Trial record 1 of 1 for:    NS018-201
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Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis

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ClinicalTrials.gov Identifier: NCT04854096
Recruitment Status : Recruiting
First Posted : April 22, 2021
Last Update Posted : November 1, 2022
Sponsor:
Collaborator:
Nippon Shinyaku Co., Ltd.
Information provided by (Responsible Party):
NS Pharma, Inc.

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).

Condition or disease Intervention/treatment Phase
Primary Myelofibrosis Post-essential Thrombocythemia Myelofibrosis Post-polycythemia Vera Myelofibrosis Drug: NS-018 Drug: Best Available Therapy Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
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)
Estimated Study Start Date : December 1, 2022
Estimated Primary Completion Date : March 24, 2024
Estimated Study Completion Date : April 24, 2024


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Change in spleen volume [ Time Frame: from baseline to week 24 ]
    Proportion of subjects who achieve ≥35% change in spleen volume from baseline to Week 24 as measured by MRI (or by CT for applicable subjects)

  2. Change in Total Symptom Score (TSS) [ Time Frame: from baseline to week 24 ]
    Proportion of subjects who achieve ≥50% change in total symptom score from baseline to Week 24 as measured by the MFSAF v4.0


Secondary Outcome Measures :
  1. Change in spleen volume [ Time Frame: from baseline at anytime up to week 24 ]
    Proportion of subjects in NS-018 vs BAT arm who achieve ≥35% change in spleen volume from baseline at any time up to Week 24 as measured by MRI (or by CT for applicable subjects)

  2. Comparison of treatment-emergent AEs [ Time Frame: from baseline to week 24 ]
    Laboratory events graded by the NCI CTCAE v5.0 will be assessed in both arms, NS-018 vs BAT.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 one JAK inhibitor.
  • Subject has received prior JAK inhibitor treatment and meets one of the following criteria:

    1. A duration of the treatment is 180 days or more.
    2. Loss of spleen response (response defined as at least 50% decrease in spleen length by palpation; loss of response defined as >50% increase in spleen length from the best response).
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04854096


Contacts
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Contact: NS Pharma, Inc. trialinfo@nspharma.com

Locations
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Sponsors and Collaborators
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.
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Responsible Party: NS Pharma, Inc.
ClinicalTrials.gov Identifier: NCT04854096    
Other Study ID Numbers: NS-018-201
First Posted: April 22, 2021    Key Record Dates
Last Update Posted: November 1, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Submission to the FDA

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Polycythemia Vera
Primary Myelofibrosis
Polycythemia
Thrombocytosis
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Blood Platelet Disorders
Blood Coagulation Disorders
Hemorrhagic Disorders