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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05927012
Registration number
NCT05927012
Ethics application status
Date submitted
8/06/2023
Date registered
3/07/2023
Date last updated
24/11/2023
Titles & IDs
Public title
A Study to Evaluate the Safety and Preliminary Efficacy of a Response-guided Dose Titration of KER-047 in the Treatment of Functional IDA (Iron Deficiency Anemia).
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Scientific title
A Phase 2, Intrapatient Dose Titration Study of KER-047 in Participants With Functional Iron Deficiency Anemia Associated With Myelodysplastic Syndromes, Myelofibrosis, and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes
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Secondary ID [1]
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KER047-IR-202
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Iron Deficiency Anemia
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Condition category
Condition code
Blood
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Anaemia
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Blood
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Haematological diseases
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Blood
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Other blood disorders
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Diet and Nutrition
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Other diet and nutrition disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - KER-047
Experimental: Part 1 (Initial Titration Strategy) - KER-047(30 mg, 60mg or 80mg) oral tablet daily (or every other day) for up to 24 weeks.
Experimental: Part 2 (Cohort Expansion or Alternate Titration Strategy) - The starting dose regimen and titration schedule of KER-047 oral tablet will be based on the SRC (Safety Review Committee) recommendation from Part 1.
Treatment: Drugs: KER-047
Oral tablet, daily (or every other day) administration
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage of participants experiencing Treatment-emergent adverse events (TEAEs)
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Assessment method [1]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [1]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [2]
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Dose limiting toxicities (DLTs)
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Assessment method [2]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [2]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [3]
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Percentage of participants experiencing Treatment-related AEs (Adverse events)
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Assessment method [3]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [3]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [4]
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Number of participants discontinuing due to AEs (Adverse events)
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Assessment method [4]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [4]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [5]
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Change from Baseline in clinical laboratory values
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Assessment method [5]
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To determine the safety and tolerability based on changes from baseline in select clinical laboratory parameters including: Alkaline phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase, Gamma Glutamyl Transferase, Glucose, Potassium, Sodium, Total bilirubin, Folate, WBC count, Platelet Count, Reticulocyte Count, Transferrin Saturation percentage.
Note - Select safety parameters will be listed as separate outcomes during results update.
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Timepoint [5]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [6]
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Systolic Blood Pressure
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Assessment method [6]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [6]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [7]
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Diastolic Blood Pressure
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Assessment method [7]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [7]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [8]
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Respiratory rate
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Assessment method [8]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [8]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [9]
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Heart rate
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Assessment method [9]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [9]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [10]
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Body temperature
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Assessment method [10]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [10]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [11]
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Fridericia corrected QT interval via 12-lead Electrocardiogram (ECG)
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Assessment method [11]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [11]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [12]
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QT interval via 12-lead Electrocardiogram (ECG)
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Assessment method [12]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [12]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [13]
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QRS interval via 12-lead Electrocardiogram (ECG)
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Assessment method [13]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [13]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [14]
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PR interval via 12-lead Electrocardiogram (ECG)
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Assessment method [14]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [14]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Primary outcome [15]
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Body weight (in kg)
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Assessment method [15]
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To determine safety and tolerability of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [15]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Secondary outcome [1]
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Change from baseline in reticulocyte hemoglobin content (RET-He)
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Assessment method [1]
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To evaluate the Pharmacodynamic (PD) effects of KER-047 on iron metabolism in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [1]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Secondary outcome [2]
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Change from baseline in hepcidin concentration
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Assessment method [2]
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To evaluate the Pharmacodynamic (PD) effects of KER-047 on iron metabolism in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [2]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Secondary outcome [3]
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Change from baseline in hemoglobin (Hgb)
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Assessment method [3]
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To evaluate the effect of KER-047 on anemia in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [3]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Secondary outcome [4]
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Proportion of participants who have Hgb increase of =1.0 g/dL (0.6 mmol/L)
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Assessment method [4]
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To evaluate the effect of KER-047 on anemia in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [4]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Secondary outcome [5]
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Proportion of participants who have Hgb increase of =1.5 g/dL (0.9 mmol/L)
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Assessment method [5]
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To evaluate the effect of KER-047 on anemia in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [5]
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Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
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Secondary outcome [6]
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Proportion of RBC-transfused participants who achieve =8 weeks of transfusion independence during any consecutive period up to End of Treatment
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Assessment method [6]
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To evaluate the effect of KER-047 on anemia in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [6]
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Up to 29 weeks or up to 101 weeks if in the treatment extension
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Secondary outcome [7]
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Plasma KER-047 and any metabolites concentration, summarized by time point
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Assessment method [7]
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To evaluate the Pharmacokinetic (PK) of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [7]
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Week 1 and Week 13 in Part 1 and 2
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Secondary outcome [8]
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Estimated peak plasma concentration (Cmax)
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Assessment method [8]
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To evaluate the Pharmacokinetic (PK) of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [8]
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Week 1 and Week 13 in Part 1 and 2
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Secondary outcome [9]
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Time to peak plasma concentration (Tmax)
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Assessment method [9]
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To evaluate the Pharmacokinetic (PK) of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [9]
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Week 1 and Week 13 in Part 1 and 2
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Secondary outcome [10]
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Area under the plasma KER-047 concentration curve (AUClast)
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Assessment method [10]
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To evaluate the Pharmacokinetic (PK) of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [10]
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Week 1 and Week 13 in Part 1 and 2
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Secondary outcome [11]
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Mean trough (Ctrough) plasma KER-047 and metabolites of interest concentration
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Assessment method [11]
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To evaluate the Pharmacokinetic (PK) of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [11]
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Up to 25 weeks
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Secondary outcome [12]
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Plasma KER-047 and metabolites of interest accumulation (Rac)
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Assessment method [12]
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To evaluate the Pharmacokinetic (PK) of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [12]
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Up to 25 weeks
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Secondary outcome [13]
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Determination of steady-state (as appropriate)
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Assessment method [13]
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To evaluate the Pharmacokinetic (PK) of KER-047 in participants with functional IDA associated with MDS, MF, and MDS/MPN overlap syndromes
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Timepoint [13]
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Up to 25 weeks
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Eligibility
Key inclusion criteria
- Male or female =18 years of age, at the time of signing informed consent.
- One of the following:
1. Diagnosis of MDS according to the 2016 World Health Organization (WHO)
classification that meets Revised International Prognostic Scoring System
(IPSS-R) classification of very low, low, or intermediate risk disease with bone
marrow blast percentage <5% within 6 months prior to Day 1 (D1).
2. Diagnosis of primary myelofibrosis, post polycythemia vera MF, or post-essential
thrombocytopenia MF according to the 2017 WHO criteria with bone marrow and
peripheral blood blast percentage <2%, or stable between 2% to 5% over 6 months.
3. Diagnosis of MDS/MPN overlap syndromes according to the 2016 WHO classification,
with bone marrow blast percentage <5% within 6 months prior to D1.
- Anemia with iron-restricted erythropoiesis as assessed by laboratory criteria during
screening.
- Ability to understand the purpose and risks of the study and provide signed and dated
informed consent and authorization to use protected health information in accordance
with national and local study participant privacy regulations.
- Females of childbearing potential and sexually active males must meet the
contraception requirements as outlined in the protocol.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Active infection within 14 days of D1.
- IPSS-R score indicating high or very high risk MDS, accelerated myelofibrosis (defined
as >10% blasts), or diagnosis of acute leukemia.
- Diagnosis of hemolytic anemia.
- Diagnosis of porphyria.
- Anemia due to blood loss 28 days prior to D1.
- Diagnosis of thalassemia, thalassemia trait, or other hemoglobinopathy.
- History of drug or alcohol abuse, as defined by the Investigator, within the past 2
years.
- History of stroke, arterial embolism, or deep venous thrombosis within 6 months prior
to D1.
- Known positive for human immunodeficiency virus, active infectious hepatitis B virus
or active infectious hepatitis C virus.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Withdrawn
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
30/11/2023
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Actual
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
4/01/2026
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Actual
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Sample size
Target
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
SA
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Recruitment hospital [1]
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Royal Adelaide Hospital - Adelaide
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Recruitment postcode(s) [1]
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5000 - Adelaide
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Recruitment outside Australia
Country [1]
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Israel
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State/province [1]
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Jerusalem
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Country [2]
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Israel
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State/province [2]
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Nahariya
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Country [3]
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Israel
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State/province [3]
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Netanya
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Country [4]
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Israel
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State/province [4]
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Zrifin
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Keros Therapeutics, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This study aims to explore the safety and preliminary efficacy of a response-guided dose
titration of KER-047 in the treatment of functional IDA (Iron deficiency anemia) in MDS
(Myelodysplastic syndrome), MF(Myelofibrosis), and MDS/MPN (Myeloproliferative neoplasm)
overlap syndromes.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05927012
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05927012
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