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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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ClinicalTrials.gov Identifier: NCT04419649
Recruitment Status : Recruiting
First Posted : June 5, 2020
Last Update Posted : September 29, 2023
Sponsor:
Information provided by (Responsible Party):
Keros Therapeutics, Inc.

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Cytopenia Drug: KER-050 Phase 2

Detailed Description:
KER-050 is a therapeutic protein designed to increase red blood cell and platelet production by inhibiting the signaling of a subset of the transforming growth factor beta (TGF-ß) family of proteins to promote hematopoiesis. It is being developed for the treatment of low blood cell counts, or cytopenias including anemia and thrombocytopenia in patients with Myelodysplastic Syndrome (MDS) and Myelofibrosis (MF).
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 140 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Ascending dose study
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : August 19, 2020
Estimated Primary Completion Date : June 30, 2025
Estimated Study Completion Date : November 30, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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.
 Drug: 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.
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.
 Drug: 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.
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.
 Drug: 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.
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.
 Drug: 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.
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.
 Drug: 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.
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.
 Drug: 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.


Outcome Measures
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Primary Outcome Measures :
  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 Measures :
  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


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Diagnosis of 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.
  2. < 5% blasts in bone marrow.
  3. Peripheral blood white blood cell count <13,000/µL.

  4. Anemia defined as:

    1. In non-transfused participants, having received no red blood cell (RBC) transfusions within 8 weeks Hgb concentration ≤ 10.0 g/dL OR
    2. In LTB participants, having received 1 to 3 units RBCs for Hgb ≤ 9.0 g/dL within 8 weeks OR
    3. In HTB participants, having received ≥ 4 units of RBCs for Hgb ≤ 9.0 g/dL within 8 weeks
  5. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia.
  6. Females of child-bearing potential and sexually active males must agree to use effective methods of contraception.

Key Exclusion Criteria:

  1. 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.
  2. 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).
  3. Vitamin B12 deficiency.
  4. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.

  5. Treatment within 28 days prior to Cycle 1 Day 1 with:

    1. Erythropoiesis stimulating agent (ESA) OR
    2. Granulocyte colony-stimulating factor (G-CSF) OR
    3. Granulocyte-macrophage colony-stimulating factor (GM-CSF)
  6. Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.
  7. Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
  8. Treatment with another investigational drug or device or approved therapy for investigational use < or = 28 days prior to Cycle 1 Day 1, or if the half-life of the previous product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer.
  9. Platelet count > 450 x 10*9/L or < 30 x 10*9/L.
  10. Transferrin saturation < 15%.
  11. Ferritin < 50 µg/L.
  12. Folate < 4.5 nmol/L (< 2.0 ng/mL).
  13. Vitamin B12 < 148 pmol/L (< 200 pg/mL).
  14. Estimated glomerular filtration rate (GFR) < 40 mL/min/1.73 m2 (as determined by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI].
  15. Pregnant or lactating females
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04419649


Contacts
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Contact: Clinical Study Team +1 (617) 314-6297 ker050-md-201@kerostx.com

Locations
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Sponsors and Collaborators
Keros Therapeutics, Inc.
More Information
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Responsible Party: Keros Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04419649    
Other Study ID Numbers: KER050-MD-201
First Posted: June 5, 2020    Key Record Dates
Last Update Posted: September 29, 2023
Last Verified: September 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Keros Therapeutics, Inc.:
transfusion
Additional relevant MeSH terms:
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Preleukemia
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
 Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Neoplasms