Myelofibrosis
Conditions
Brief summary
Part 1 Dose Escalation: •Safety and tolerability as determined by the incidence of adverse events (AEs), including dose-limiting toxicities (DLTs), severe AEs, and serious AEs (SAEs), Part 2 Dose Expansion: •Safety and tolerability as determined by the incidence of AEs, including severe AEs and SAEs, Long-Term Extension: •Safety and tolerability as determined by the incidence of AEs, including severe AEs and SAEs over time
Detailed description
Proportion of participants with progression to AML, Proportion of participants with progression to accelerated MF, Subgroup of participants with anemia requiring red blood cell (RBC) transfusions (See Table 3 in Protocol KER050-MF-301 v 6.0)., Subgroup of transfusion-independent participants (and, when appropriate, the subgroup of participants with anemia requiring RBC transfusions who are deemed not evaluable for assessment of transfusion independence) (See Table 3 Protocol KER050-MF-301 v 6.0)., Proportion of participants with improvement in the MF-SAF (v4.0)-TSS of ≥50% from baseline at Week 24, Proportion of participants with decrease in spleen volume of ≥35% from baseline as measured by computed tomography/magnetic resonance imaging (CT/MRI) at Week 24 (excluding participants’ status post splenectomy or splenic irradiation), Plasma concentrations of elritercept, Maximum observed serum concentration (Cmax), time to maximum observed concentration (Tmax), and area under the concentration-time curve from time 0 to the time of last quantifiable concentration (AUC0-last; Cycle 1 only), Minimum observed serum concentration (Cmin) and accumulation rate (Rac), Change from baseline of red cell parameters, including reticulocyte count, Hgb, mean corpuscular volume, mean corpuscular hemoglobin, and reticulocyte cell Hgb by visit
Interventions
Sponsors
Takeda Development Center Americas Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part 1 Dose Escalation: •Safety and tolerability as determined by the incidence of adverse events (AEs), including dose-limiting toxicities (DLTs), severe AEs, and serious AEs (SAEs), Part 2 Dose Expansion: •Safety and tolerability as determined by the incidence of AEs, including severe AEs and SAEs, Long-Term Extension: •Safety and tolerability as determined by the incidence of AEs, including severe AEs and SAEs over time | — |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of participants with progression to AML, Proportion of participants with progression to accelerated MF, Subgroup of participants with anemia requiring red blood cell (RBC) transfusions (See Table 3 in Protocol KER050-MF-301 v 6.0)., Subgroup of transfusion-independent participants (and, when appropriate, the subgroup of participants with anemia requiring RBC transfusions who are deemed not evaluable for assessment of transfusion independence) (See Table 3 Protocol KER050-MF-301 v 6.0)., Proportion of participants with improvement in the MF-SAF (v4.0)-TSS of ≥50% from baseline at Week 24, Proportion of participants with decrease in spleen volume of ≥35% from baseline as measured by computed tomography/magnetic resonance imaging (CT/MRI) at Week 24 (excluding participants’ status post splenectomy or splenic irradiation), Plasma concentrations of elritercept, Maximum observed serum concentration (Cmax), time to maximum observed concentration (Tmax), and area under the concentra | — |
Countries
France, Italy, Spain
Outcome results
None listed