NS-018 versus Best Available Therapy in Subjects with Primary Myelofibrosis
Research type
Research Study
Full 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)
IRAS ID
1004875
Contact name
Kavita Philips
Contact email
Sponsor organisation
NS Pharma, Inc.
Eudract number
2021-000369-34
Clinicaltrials.gov Identifier
Research summary
Study will look at an investigational medication, NS-018, in people with a blood condition that affects the bone marrow, called primary myelofibrosis, post-polycythaemia vera myelofibrosis, or post-essential thrombocythaemia myelofibrosis.
Myelofibrosis is a rare bone marrow cancer in which bone marrow (spongy tissue found inside some of the bones that help make red blood cells, which help carry oxygen through the body), white blood cells (which help fight infections), and platelets (which help blood to clot) are replaced by fibrous scar tissue. This results in the bone marrow not being able to make normal blood cells. Myelofibrosis can occur either on its own, which is known as primary myelofibrosis or as a progression of other bone marrow diseases.
Polycythaemia vera is a condition that causes the bone marrow to make too many red blood cells in the blood, which over a period of time can lead to myelofibrosis (also known as post-polycythaemia vera myelofibrosis).
Essential thrombocythaemia is a condition in which the bone marrow makes too many platelets in the blood, which over a period of time can lead to myelofibrosis (also known as post-essential thrombocytopenia myelofibrosis).
The use of the study drug in this study is investigational. This means that the study drug is not approved by any health regulatory agencies and is not yet available by prescription to treat primary myelofibrosis, post-polycythaemia vera myelofibrosis, or post-essential thrombocythaemia myelofibrosis.
The main purpose of this study is to look at whether the study drug works in participants with these blood conditions. The study will also look at: How safe the study drug is and what the body does to the study drug (pharmacokinetics)
The study drug is designed to work by blocking the action of a protein called Janus Kinase 2 (JAK 2). By doing so, it is hoped that the study drug will improve participants symptoms. The study drug will be compared with best available therapy.REC name
London - Fulham Research Ethics Committee
REC reference
22/LO/0220
Date of REC Opinion
25 May 2022
REC opinion
Further Information Unfavourable Opinion