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A Randomized, Controlled Phase 3 Study of Pacritinib Versus Physician's Choice in Patients with Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post Essential Thrombocythemia Myelofibrosis with Severe Thrombocytopenia (Platelet Counts <50,000/microL)

Phase 3 Study of SB1518 in Patients with Myelofibrosis

Ito Masayuki

Swedish Orphan Biovitrum Japan Co., Ltd.

2-6-1, Toranomon, Minato-ku, Tokyo

SobiJapanContact@sobi.com

Ito Masayuki

Swedish Orphan Biovitrum Japan Co., Ltd

2-6-1, Toranomon, Minato-ku, Tokyo

+81-3-4400-6872

SobiJapanContact@sobi.com

Not Recruiting

Feb. 07, 2025

Interventional

randomized controlled trial

open(masking not used)

active control

parallel assignment

treatment purpose

1. Primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF
2. Platelet count of <50,000/microL at Screening (Day -35 to Day -3)
3. Dynamic International Prognostic Scoring System Intermediate-1, Intermediate 2, or High-Risk
4. Palpable splenomegaly >-5 cm below the lower costal margin in the midclavicular line as assessed by physical examination
5. TSS of >-10 on the MPN-SAF TSS 2.0 or a single symptom score of >-5 or two symptoms of >-3, including only the symptoms of left upper quadrant pain, bone pain, itching, or night sweats. The TSS criteria need only to be met on a single day.
6. Age >-18 years
7. Eastern Cooperative Oncology Group performance status 0 to 2
8. Provision of signed informed consent

1. Life expectancy <6 months
2. Completed allogeneic stem cell transplant, or are eligible for and willing to complete other approved available therapy including allogeneic stem cell transplant
3. History of splenectomy or planning to undergo splenectomy
4. Splenic irradiation within the last 6 months
5. Previously treated with pacritinib
6. Treatment with any MF-directed therapy within 14 days prior to treatment Day 1
7. Prior treatment with more than one JAK2 inhibitor

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Both

Myelofibrosis (MF)

Pacritinib 200 mg (2 capsules) BID orally, at the same time of day, with or without food

1. To compare the efficacy of pacritinib with that of P/C therapy, as assessed by the proportion of participants achieving a >-35% SVR from baseline at Week 24 as measured by MRI (preferred) or CT scans.
2. To compare the efficacy of pacritinib with P/C therapy, as assessed by the proportion of participants achieving a >-50% reduction in TSS from baseline at Week 24

1. To compare the percentage of participants who self-assess as ''very much improved'' or ''much improved'' as measured by the Patient Global Impression of Change (PGIC) in participants treated with pacritinib versus those treated with P/C
2. To compare the OS of participants treated with pacritinib versus those treated with P/C
3. To compare the safety of pacritinib versus P/C therapy

Jan. 30, 2025

No

United States/Australia/Belarus/Bosnia and Herzegovina/Bulgaria/Federal Republic of Brazil/Canada/Czechia/France/Georgia/Hungary/India/Israel/Italy/Korea/Poland/Romania/Russian Federation/Serbia/Spain/Ukraine/United Kingdom/Kazakhstan