A single-arm trial of daprodustat for low-risk myelodysplastic syndromes

JRCT ID: jRCTs031240080

Registered date:13/05/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Myelodysplastic syndromes
• Date of first enrollment: 13/05/2024
• Target sample size: 15
• Study type: Interventional
• Intervention(s): Take daprodustat once a day (starting dose; 2mg for patients with hemoglobin 9.0g/dLor over, 4mg for patients with hemoglobin less than 9.0g/dL). Adjust dosage based on fixed rules. Maximum dosage is 24mg per day. Treatment duration is 24 weeks.

Outcome(s)

• Primary Outcome: Percentage of patients who achieved hematological improvement-erythroid (HI-E) within 24 weeks since treatment initiation

Secondary Outcome

(1) Progression free survival
(2) Adverse events after treatment
(3) Correlation between pretreatment serum erythropoietin level and efficacy of daprodustat
(4) Correlation between pretreatment iron-related parameters and efficacy of daprodustat
(5) Correlation between past history and efficacy of daprodustat

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

(1) Patient with low-risk myelodysplastic syndromes 18 years old or over
(2) Patients harboring anemia with serum hemoglobin less than 10g/dL
(3) Patients who can self-administer their own medication
(4) Patients who provided informed consent with written documents

Exclude criteria

(1) Patients who have allergy to daprodustat
(2) Pregnant women
(3) Nursing women
(4) Patients with liver dysfunction (AST 150IU/L or over, or ALT 200IU/L or over)
(5) Patients with renal dysfunction (serum creatinine 2.0mg/dL or over, or eGFR below 30mL/min/1.73m2)
(6) Patients diagnosed with 5q- syndrome
(7) Anemia due to other causes except renal anemia
(8) Patients who were diagnosed with acute myocardial infaction or angina within 4 weeks from informed consent
(9) Patients who were diagnosed with cerebral infaction within 4 weeks from informed consent
(10) Patients who were diagnosed with deep venous thrombosis within 4 weeks from informed consent
(11) Patients who are in active treatment for malignancies at the time of informed consent
(12) Patients who completed their treatment for malignancies but within 1 year since CR achievement at the time of informed consent
(13) Patients with severe hypertension (mean systolic blood pressure 180 mmHg or over, mean diastolic blood pressure 120 mmHg or over)
(14) Patients who were diagnosed with proliferative retinopathy
(15) Patients diagnosed with renal anemia being actively treated with EPO
(16) Patients with both MDS and renal anemia who have been treated with daprodustat but showed no improvement of his anemia
(17) Patients who were judged to be unsuitable for this study by physicians

Related Information

• Primary Sponsor: Kunimoto Hiroyoshi
• Secondary Sponsor: Hideaki Nakajima,Yokohama City University Research Grant for the Promotion of Advanced Medicine