University of Miami Hospital and Clinics 33136 Miami United StatesRekrutierend» Google-MapsOcala Oncology 34474 Ocala United StatesRekrutierend» Google-MapsUniversity of Maryland Greenebaum Comprehensive Cancer Center 21201 Baltimore United StatesRekrutierend» Google-MapsMaimonides Medical Center 11219 Brooklyn United StatesRekrutierend» Google-MapsNYU Langone Health 10016 New York United StatesRekrutierend» Google-MapsColumbia University Irving Medical Center 10032 New York United StatesRekrutierend» Google-MapsBaptist Clinical Research Institute 38120 Memphis United StatesRekrutierend» Google-MapsUniversity of British Columbia - St. Paul's Hospital V6Z 2K5 Vancouver CanadaRekrutierend» Google-MapsNice University Hospital - Hôpital de l'Archet 06200 Route De Saint-Antoine FranceRekrutierend» Google-MapsHôpital Avicenne 93000 Bobigny FranceRekrutierend» Google-MapsCHU de Nantes - Hôtel Dieu 44000 Nantes FranceRekrutierend» Google-MapsHopital Saint Louis Paris FranceRekrutierend» Google-Maps
1. The hematologic improvement based on an erythroid response (HI -E) ≥ 8 weeks duration in patients with MDS after 16 weeks of etavopivat treatment (Time Frame - 16 weeks): Measure in number of patient incidence
This endpoint will be based on the combined incidence of:
Non-transfusion dependent (NTD) patients: ≥ 1.5 g/dL increase in hemoglobin (Hb) from baseline maintained ≥ 8 consecutive weeks
Low transfusion burden (LTB) patients: absence of any transfusion for ≥ 8 consecutive weeks
High transfusion burden (HTB) patients: reduction by ≥ 50% of red blood cell (RBC) units for ≥ 8 consecutive weeks
Secondary outcome:
1. The HI-E ≥ 8 weeks duration in this population of patients after 24 and 48 weeks of etavopivat treatment (Time Frame - 24 and 48 weeks): Measure in number of patient incidence
This endpoint will be based on the combined incidence of:
Non-transfusion dependent (NTD) patients: ≥ 1.5 g/dL increase in hemoglobin (Hb) from baseline maintained ≥ 8 consecutive weeks
Low transfusion burden (LTB) patients: absence of any transfusion for ≥ 8 consecutive weeks
High transfusion burden (HTB) patients: reduction by ≥ 50% of red blood cell (RBC) units for ≥ 8 consecutive weeks
2. The HI-E ≥ 16 weeks duration in this population of patients after 24 and 48 weeks of etavopivat treatment (Time Frame - 24 and 48 weeks): Measure in number of patient incidence
This endpoint will be based on the combined incidence of:
Non-transfusion dependent (NTD) patients: ≥ 1.5 g/dL increase in hemoglobin (Hb) from baseline maintained ≥ 16 consecutive weeks
Low transfusion burden (LTB) patients: absence of any transfusion for ≥ 16 consecutive weeks
High transfusion burden (HTB) patients: reduction by ≥ 50% of red blood cell (RBC) units for ≥ 16 consecutive weeks
3. Incidence of AEs, serious adverse events (SAEs), and AEs related to etavopivat (Time Frame - 4(first 6 participants), 16, 24, and 48 weeks): Measure in number of patient incidences
4. Number of premature discontinuations, dose interruptions, and dose reductions (Time Frame - 4(first 6 participants), 16, 24, and 48 weeks): Measure in number of patient incidences
5. Overall response rate for MDS (Time Frame - 16, 24, and 48 weeks.): Measure in number of patient incidence, per Chelson, 2006 International Working Group [IWG] Criteria
6. Duration of response (Time Frame - 16, 24, and 48 weeks.): Measure in number of days, per 2006 IWG Criteria
7. Percentage of participants who achieved RBC transfusion independence in participants with LTB or HTB at study entry (Time Frame - 16, 24, and 48 weeks.): Measure in percentage
8. Change from baseline in RBC units transfused in patients with NTD, LTB or HTB at study entry (Time Frame - 16, 24, and 48 weeks.): Measure in RBC units
9. Participants who achieved a hematologic improvement in neutrophil (Time Frame - 16, 24, and 48 weeks.): Measure in number of patient incidence
10. Participants who achieved a hematologic improvement in platelet response (Time Frame - 16, 24, and 48 weeks.): Measure in number of patient incidence
11. Change from baseline in mean serum ferritin (Time Frame - 16, 24, and 48 weeks.): Measure in ng/mL
12. Change from baseline in mean daily dose of iron chelation therapy (Time Frame - 16, 24, and 48 weeks.): Measure in dose unit
13. Overall survival (Time Frame - 16, 24, and 48 weeks.): Measure in number of patient incidence