Clinical Trial 22333

• Overview

  Study Title:

  A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients with Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)

  Summary:

  The purpose of this study is to evaluate the effects of KER-050 on anemia in patients with very low, low or intermediate risk MDS.

  Objective:

  Primary Objective: Part 1 Dose Escalation: To evaluate the safety and tolerability of ascending doses of KER-050 in participants with very low, low, or intermediate risk MDS in order to determine the dose(s) that will be evaluated in Part 2 of the study. Part 2 Dose Confirmation: To confirm the safety and tolerability of the dose(s) selected in Part 1. Long-Term Extension: To evaluate the long-term safety and tolerability of KER-050 in participants with very low, low, or intermediate risk MDS. Secondary Objectives (All Parts): Safety Objective: To evaluate the progression to higher risk MDS or acute myeloid leukemia (AML). Efficacy Objective: To evaluate the efficacy of KER-050 on anemia in participants with very low, low, or intermediate risk MDS, separately for ring sideroblast (RS)-positive and non-RS populations. Pharmacodynamic Objective: To evaluate the pharmacodynamic (PD) effects of KER-050 on erythropoiesis in participants with very low, low, or intermediate risk MDS, separately for RS-positive and non-RS populations.

• Treatments

  Therapies:

  Chemotherapy (NOS)

  Medications:

  KER-050 ()

• Inclusion Criteria

  Inclusion Criteria:
  • Diagnosis of Myelodysplastic Syndrome (MDS) according to World Health Organization (WHO)/French American British (FAB) classification that meets Revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease.
  • > Peripheral blood white blood cell count > Anemia defined as: In non-transfused participants, having received no red blood cell (RBC) transfusions within 8 weeks Hgb concentration 10.0 g/dL or lower OR; In LTB participants, having received 1 to 3 units RBCs for Hgb 9.0 g/dL or lower within 8 weeks OR; In HTB participants, having received 4 units or more of RBCs for Hgb 9.0 g/dL or lower within 8 weeks
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia).
  • Females of child-bearing potential and sexually active males must agree to use effective methods of contraception.

• Exclusion Criteria

  Exclusion Criteria:
  • Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1.
  • Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
  • Vitamin B12 deficiency.
  • Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
  • Treatment within 28 days prior to Cycle 1 Day 1 with: Erythropoiesis stimulating agent (ESA) OR; Granulocyte colony-stimulating factor (G-CSF) OR; Granulocyte-macrophage colony-stimulating factor (GM-CSF)
  • Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.
  • Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
  • Treatment with another investigational drug or device or approved therapy for investigational use > Platelet count > 450 x 10*9/L or > Transferrin saturation > Ferritin > Folate > Vitamin B12 > Estimated glomerular filtration rate (GFR) > Pregnant or lactating females

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