Can a new pill tame myelofibrosis?

NCT ID NCT04676529

Completed Disease control Sponsor: Syntara Source: ClinicalTrials.gov ↗

First seen Aug 04, 2026 · Last updated Aug 05, 2026 · Updated 1 time

Summary

This trial is testing an experimental oral drug called PXS-5505 (amsulostat) in people with myelofibrosis, a type of bone marrow cancer. The study focuses on patients who are not eligible for a stem cell transplant. The main goal is to see if the drug is safe and tolerable, and to learn how the body processes it. The trial is open-label, meaning everyone knows they are receiving the drug, and it involves dose escalation and expansion to find the best dose.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: PXS-5505 (amsulostat), an experimental oral drug
What this could lead to: If PXS-5505 proves safe and shows promise, it could become a new treatment option for myelofibrosis, potentially helping to control the disease and improve symptoms.
What could go wrong: This is an early-phase trial with a small number of participants, so the drug may not work as hoped or may cause unexpected side effects. Success in this trial does not guarantee approval or widespread use.

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Conditions

The condition(s) this trial relates to.

myelofibrosis primary myelofibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MYELOFIBROSIS POST POLYCYTHEMIA VERA MYELOFIBROSIS POST-ESSENTIAL THROMBOCYTHEMIA MYELOFIBROSIS PRIMARY MYELOFIBROSIS (PMF)

Contacts and locations

Locations

Asan Medical Centre

Seoul, 05505, South Korea
Ashford Cancer Centre Research

Adelaide, South Australia, 5037, Australia
Chang Gung Medical Foundation - ChiaYi Chang Gung Memorial Hospital - Hematology and Oncology

Chiayi City, Chiayi, 613, Taiwan
China Medical University Hospital - Internal Medicine - Taichung

Taichung, 40447, Taiwan
Comprehensive Cancer Center (UAB CCC)

Birmingham, Alabama, 98374, United States
Gachon University Gil Hospital

Incheon, Incheon Gwang'yeogsi [Inch'n-K, 21565, South Korea
Inje University Busan Paik Hospital - Internal Medicine

Busan, Busan Gwang'yeogsi [Pusan-Kwan, 47392, South Korea
Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, 807, Taiwan
Keimyung University Dongsan Hospital

Daegu, Daegu Gwang'yeogsi [Taegu-Kwangyokshi], 42601, South Korea
Liverpool Hospital

Liverpool, New South Wales, 2170, Australia
National Cancer Center (Seoul Metro; northern)

Gyeonggi-do, 10408, South Korea
National Cheng Kung University Hospital

Tainan, 70403, Taiwan
National Taiwan University Hospital - Hematology And Oncology

Taipei, 100, Taiwan
Novant Health Cancer Institute

Winston-Salem, North Carolina, 27103, United States
One Clinical Research

Perth, Western Australia, 6009, Australia
Samsung Medical Center

Seoul, 06351, South Korea
Seoul National University Hospital

Seoul, 03080, South Korea
Seoul National University Hospital - Bundang

Gyeonggi-do, 13620, South Korea
Severance Hospital, Yonsei University Health System- Haemat

Seoul, 03711, South Korea
St Vincent's Hospital Melbourne

Fitzroy, Victoria, 3065, Australia
The Catholic University of Korea, Seoul St. Mary's Hospital

Seoul, 06591, South Korea
The Perth Blood Institute

West Perth, Western Australia, 6005, Australia
The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

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