Myelodysplastic syndrome (MDS)
Conditions
Interventions
Experimental group:To evaluate the safety and tolerability of three starting doses of zematostat (350 mg, 300 mg and 250 mg, administered orally twice daily, on days 1–28) in combination with a fixed
Sponsors
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1. Age 18 - 80, gender not restricted; 2. Diagnosed with higher-risk myelodysplastic syndromes (IPSS-R > 3.5); 3. Patients who have no response, relapse, or experience disease progression after receiving first-line standard treatment; 4. Must have at least 1 evaluable lesion/measurable lesion meeting the response criteria of the International Working Group (IWG) 2023 edition; 5. ECOG PS 0 - 2; 6. Normal function of major organs, with laboratory test results meeting the following criteria: ? Alanine aminotransferase (ALT) = 50%; 7. Expected survival of at least 3 months; 8. Male and female subjects of childbearing potential are willing to use effective contraceptive measures throughout the study period and for 6 months after the last dose; 9. Voluntarily participate in the clinical study, sign the informed consent form, and be willing and able to follow and complete all trial procedures.
Exclusion criteria
Exclusion criteria: 1. Have received treatment with EZH2 inhibitors before enrollment; 2. History of other primary invasive malignancies that have not achieved remission or have relapsed within 3 years; 3. Patients with involvement of the Central Nervous System (meninges or brain parenchyma); 4. Patients who, after medical and comprehensive evaluation, meet the criteria for transplantation treatment; 5. Known allergy to any of the drugs in the study; 6. Participation in other drug clinical trials within 4 weeks before the start of the study; 7. Pregnant or lactating women; 8. Patients with active infections, excluding tumor-related B symptoms of fever; 9. Comorbidities or medical histories that severely affect organ function; 10. History of immunodeficiency, including positive HIV test, or other acquired or congenital immunodeficiency diseases, or history of organ transplantation; 11. Presence of IDH1 mutation and availability of an applicable IDH1 inhibitor as a treatment option; 12. Other situations judged by the investigator as unsuitable for inclusion in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Objective response rate (ORR) evaluated according to the IWG 2023 criteria;Phase II recommended dose (RP2D) for treatment with the combination of Zemeitos and azacitidine; | — |
Secondary
| Measure | Time frame |
|---|---|
| Complete remission (CR) rate assessed according to the IWG 2023 criteria;Morphological complete response (mCR) rate assessed according to the IWG 2023 criteria;Haematological Improvement (HI) rate assessed according to the IWG 2023 criteria;Disease Control Rate (DCR) assessed according to the IWG 2023 criteria;Duration of Remission (DOR) assessed in accordance with the IWG 2023 criteria;Progression-free survival (PFS) assessed according to the IWG 2023 criteria;Overall survival (OS);Safety indicators; | — |
Countries
China
Contacts
Public ContactZhang Sujiang
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed