Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis
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| ClinicalTrials.gov Identifier: NCT04854096 |
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Recruitment Status :
Recruiting
First Posted : April 22, 2021
Last Update Posted : April 18, 2024
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Sponsor:
NS Pharma, Inc.
Collaborator:
Nippon Shinyaku Co., Ltd.
Information provided by (Responsible Party):
NS Pharma, Inc.
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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 |
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 : | 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) |
| Actual Study Start Date : | January 31, 2023 |
| Estimated Primary Completion Date : | May 24, 2024 |
| Estimated Study Completion Date : | May 24, 2024 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Essential thrombocythemia
Primary myelofibrosis
Polycythemia vera
| Arm | Intervention/treatment |
|---|---|
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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
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Drug: NS-018
Experimental |
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Active Comparator: Best Available Therapy (BAT)
Single agent per Investigator discretion or no therapy
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Drug: Best Available Therapy
Active Comparator |
Primary Outcome Measures :
- 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)
- 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 :
- 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)
- 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.
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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 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.
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
| Contact: NS Pharma, Inc. | trialinfo@nspharma.com |
Locations
Show 52 study locations
Sponsors and Collaborators
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.
| 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: | April 18, 2024 |
| Last Verified: | April 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Plan Description: | Submission to the FDA |
| 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 |