1. Incidence of adverse events (AEs) and serious adverse events (SAEs). (Time Frame - From treatment initiation to end of study, approximately 2 years): Type, frequency, severity of AEs and relationship of AEs to KER-050
Secondary outcome:
1. Maximum concentrations of KER-050 (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): Pharmacokinetics of KER-050
2. Erythroid response in low-transfusion burden (LTB) and high-transfusion burden (HTB) participants. (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): In LTB participants, the proportion of participants who have a hemoglobin increase of ≥ 1.5 g/dL from Baseline for ≥ 14 days (in the absence of RBC transfusions).
In HTB participants, the proportion of participants having a reduction of ≥ 50% or ≥ 4 RBC units transfused compared to pretreatment over an 8-week period.
3. Incidence of progression to higher risk MDS or AML per World Health Organization 2016 criteria (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): Change in baseline laboratory assessments prior to treatment with KER-050 and after treatment with KER-050
4. Incidence of progression to higher risk MDS or AML per World Health Organization 2016 criteria (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): Change in baseline bone marrow assessments prior to treatment with KER-050 and after treatment with KER-050
5. Modified 2006 International Working Group (IWG) Hematologic Improvement Erythroid (HI-E) response (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): In LTB participants, the proportion of participants with an increase of ≥ 1.5 g/dL from pretreatment hemoglobin during any 8-week period on study compared to Baseline.
In HTB participants, the proportion of participants having a reduction by ≥ 4 units of RBCs transfused (for a hemoglobin ≤ 9.0 g/dL) during any 8-week period on study, compared with the 8-week period prior to study Cycle 1 Day 1.
6. Mean change from baseline in hemoglobin (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): Change in baseline hemoglobin prior to treatment with KER-050 and after treatment with KER-050
7. Time to erythroid response and modified 2006 IWG HI-E response (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): Time to erythroid response prior to treatment with KER-050 and after treatment with KER-050
8. Duration of erythroid response and modified 2006 IWG HI-E response (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): Duration of erythroid response prior to treatment with KER-050 and after treatment with KER-050
9. Frequency of red blood cell (RBC) transfusions and rate of RBC transfusion independence ≥ 8 weeks (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): Frequency and rate of RBC transfusions prior to treatment with KER-050 and after treatment with KER-050
10. Change from Baseline in RBC counts and reticulocytes (Time Frame - Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years): Change in baseline RBC counts and reticulocytes prior to treatment with KER-050 and after treatment with KER-050
Experimental: KER-050 Cohort 1 Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 4 cycles. Participants have the option to continue to receive KER-050 once 4 cycles have been completed for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.
Experimental: KER-050 Cohort 2 Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 4 cycles. Participants have the option to continue to receive KER-050 once 4 cycles have been completed for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.
Experimental: KER-050 Cohort 3 Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.
Experimental: KER-050 Cohort 4 Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.
Experimental: KER-050 Cohort 5 Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.
Experimental: KER-050 Dose Confirmation Cohort Participants to receive KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.
KER-050: KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles in a Long-Term Extension.
Quelle: ClinicalTrials.gov
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"A Study of KER-050 to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes"
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