Relapsed or Refractory High-Risk Myelodysplastic Syndrome, Acute Myeloid Leukemia with or without NPM1 Mutation
Conditions
Brief summary
Rate of CR
Detailed description
Overall response rate (ORR) including clinical benefit beyond CR • Duration of response (DoR) • Transfusion independence and/or hematological improvement • 1-year progression-free survival (PFS) • 1-year relapse-free survival (RFS) • 1-year overall survival (OS) • Percentage of participants bridged to hematopoietic stem cell transplantation (HSCT), Incidence and severity of AE, PK of RVU120 including, data permitting, Cmax, tmax, AUCtau, AUCinf ax, AUC, and t½, Part 2 only: Changes in patient-reported outcomes as assessed by changes in summary scores of the HM-PRO and QOL-E, Rate of measurable residual disease (MRD) negativity
Interventions
Sponsors
Ryvu Therapeutics S.A.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Rate of CR | — |
Secondary
| Measure | Time frame |
|---|---|
| Overall response rate (ORR) including clinical benefit beyond CR • Duration of response (DoR) • Transfusion independence and/or hematological improvement • 1-year progression-free survival (PFS) • 1-year relapse-free survival (RFS) • 1-year overall survival (OS) • Percentage of participants bridged to hematopoietic stem cell transplantation (HSCT), Incidence and severity of AE, PK of RVU120 including, data permitting, Cmax, tmax, AUCtau, AUCinf ax, AUC, and t½, Part 2 only: Changes in patient-reported outcomes as assessed by changes in summary scores of the HM-PRO and QOL-E, Rate of measurable residual disease (MRD) negativity | — |
Countries
France, Italy, Poland, Spain
Outcome results
None listed