Severe aplastic anemia (SAA)
Conditions
Brief summary
Part A: Incidence of adverse events (AEs), incidence of serious adverse events (SAEs), and incidence and severity of treatment-emergent adverse events (TEAEs);, Part B: Incidence of serious adverse events (SAEs), incidence and severity of treatment-emergent adverse events (TEAEs);, Part B: Overall response rate (ORR);
Detailed description
Overall response rate (ORR); Parts A & B, Complete response (CR); Parts A and B, Partial response (PR); Parts A and B, Time to best response; Part A, Time to best response; Part B, Time to first response; Part A, Time to first response; Part B, Any clinical response; Part A, Any clinical response; Part B, Platelet transfusions per month over time; Part A, Platelet transfusions per month over time; Part B, Red blood cell transfusions per month over time; Part A, Red blood cell transfusions per month over time; Part B, Changes in lymphocyte cell counts; Part A, Changes in lymphocyte cell counts; Part B, Changes in neutrophil cell counts; Part A, Changes in neutrophil cell counts; Part B, Changes in hemoglobin cell counts; Part A, Changes in hemoglobin cell counts; Part B, Changes in reticulocyte cell counts; Part A, Changes in reticulocyte cell counts; Part B, Changes in platelet cell counts; Part A, Changes in platelet cell counts; Part B, Changes in the whole blood immune cell subsets (T cells); Part A, Changes in the whole blood immune cell subsets (T cells); Part B, Changes in the whole blood immune cell subsets (B cells); Part A, Changes in the whole blood immune cell subsets (B cells); Part B, Changes in the whole blood immune cell subsets [Natural killer (NK) cells]; Part A, Changes in the whole blood immune cell subsets (NK cells); Part B, Drug concentrations in serum over time; Part A, Drug concentrations in serum over time; Part B, Incidence of treatment-emergent anti-drug antibody (ADA) over time; Part A, Incidence of treatment-emergent ADA over time; Part B
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: Incidence of adverse events (AEs), incidence of serious adverse events (SAEs), and incidence and severity of treatment-emergent adverse events (TEAEs);, Part B: Incidence of serious adverse events (SAEs), incidence and severity of treatment-emergent adverse events (TEAEs);, Part B: Overall response rate (ORR); | — |
Secondary
| Measure | Time frame |
|---|---|
| Overall response rate (ORR); Parts A & B, Complete response (CR); Parts A and B, Partial response (PR); Parts A and B, Time to best response; Part A, Time to best response; Part B, Time to first response; Part A, Time to first response; Part B, Any clinical response; Part A, Any clinical response; Part B, Platelet transfusions per month over time; Part A, Platelet transfusions per month over time; Part B, Red blood cell transfusions per month over time; Part A, Red blood cell transfusions per month over time; Part B, Changes in lymphocyte cell counts; Part A, Changes in lymphocyte cell counts; Part B, Changes in neutrophil cell counts; Part A, Changes in neutrophil cell counts; Part B, Changes in hemoglobin cell counts; Part A, Changes in hemoglobin cell counts; Part B, Changes in reticulocyte cell counts; Part A, Changes in reticulocyte cell counts; Part B, Changes in platelet cell counts; Part A, Changes in platelet cell counts; Part B, Changes in the whole blood | — |
Countries
France