<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT04419649</url>
  </required_header>
  <id_info>
    <org_study_id>KER050-MD-201</org_study_id>
    <nct_id>NCT04419649</nct_id>
  </id_info>
  <brief_title>A Study of KER-050 to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes</brief_title>
  <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)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Keros Therapeutics, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Keros Therapeutics, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to evaluate the effects of KER-050 on anemia in patients with&#xD;
      very low, low or intermediate risk MDS.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      KER-050 is a therapeutic protein designed to increase red blood cell and platelet production&#xD;
      by inhibiting the signaling of a subset of the transforming growth factor beta (TGF-ß) family&#xD;
      of proteins to promote hematopoiesis. It is being developed for the treatment of low blood&#xD;
      cell counts, or cytopenias including anemia and thrombocytopenia in patients with&#xD;
      Myelodysplastic Syndrome (MDS) and Myelofibrosis (MF).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">August 19, 2020</start_date>
  <completion_date type="Anticipated">November 30, 2025</completion_date>
  <primary_completion_date type="Anticipated">June 30, 2025</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Sequential Assignment</intervention_model>
    <intervention_model_description>Ascending dose study</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Incidence of adverse events (AEs) and serious adverse events (SAEs).</measure>
    <time_frame>From treatment initiation to end of study, approximately 2 years</time_frame>
    <description>Type, frequency, severity of AEs and relationship of AEs to KER-050</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Maximum concentrations of KER-050</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>Pharmacokinetics of KER-050</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Erythroid response in low-transfusion burden (LTB) and high-transfusion burden (HTB) participants.</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>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).&#xD;
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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of progression to higher risk MDS or AML per World Health Organization 2016 criteria</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>Change in baseline laboratory assessments prior to treatment with KER-050 and after treatment with KER-050</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of progression to higher risk MDS or AML per World Health Organization 2016 criteria</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>Change in baseline bone marrow assessments prior to treatment with KER-050 and after treatment with KER-050</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Modified 2006 International Working Group (IWG) Hematologic Improvement Erythroid (HI-E) response</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>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.&#xD;
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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Mean change from baseline in hemoglobin</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>Change in baseline hemoglobin prior to treatment with KER-050 and after treatment with KER-050</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to erythroid response and modified 2006 IWG HI-E response</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>Time to erythroid response prior to treatment with KER-050 and after treatment with KER-050</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Duration of erythroid response and modified 2006 IWG HI-E response</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>Duration of erythroid response prior to treatment with KER-050 and after treatment with KER-050</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Frequency of red blood cell (RBC) transfusions and rate of RBC transfusion independence ≥ 8 weeks</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>Frequency and rate of RBC transfusions prior to treatment with KER-050 and after treatment with KER-050</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from Baseline in RBC counts and reticulocytes</measure>
    <time_frame>Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years</time_frame>
    <description>Change in baseline RBC counts and reticulocytes prior to treatment with KER-050 and after treatment with KER-050</description>
  </secondary_outcome>
  <number_of_arms>6</number_of_arms>
  <enrollment type="Anticipated">140</enrollment>
  <condition>Myelodysplastic Syndromes</condition>
  <condition>Cytopenia</condition>
  <arm_group>
    <arm_group_label>KER-050 Cohort 1</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>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.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>KER-050 Cohort 2</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>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.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>KER-050 Cohort 3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>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.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>KER-050 Cohort 4</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>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.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>KER-050 Cohort 5</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>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.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>KER-050 Dose Confirmation Cohort</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>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.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>KER-050</intervention_name>
    <description>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.</description>
    <arm_group_label>KER-050 Cohort 1</arm_group_label>
    <arm_group_label>KER-050 Cohort 2</arm_group_label>
    <arm_group_label>KER-050 Cohort 3</arm_group_label>
    <arm_group_label>KER-050 Cohort 4</arm_group_label>
    <arm_group_label>KER-050 Cohort 5</arm_group_label>
    <arm_group_label>KER-050 Dose Confirmation Cohort</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Key Inclusion Criteria:&#xD;
&#xD;
          1. Diagnosis of MDS according to World Health Organization (WHO)/French American British&#xD;
             (FAB) classification that meets Revised International Prognostic Scoring System&#xD;
             (IPSS-R) classification of very low, low, or intermediate risk disease.&#xD;
&#xD;
          2. &lt; 5% blasts in bone marrow.&#xD;
&#xD;
          3. Peripheral blood white blood cell count &lt;13,000/µL.&#xD;
&#xD;
          4. Anemia defined as:&#xD;
&#xD;
               1. In non-transfused participants, having received no red blood cell (RBC)&#xD;
                  transfusions within 8 weeks Hgb concentration ≤ 10.0 g/dL OR&#xD;
&#xD;
               2. In LTB participants, having received 1 to 3 units RBCs for Hgb ≤ 9.0 g/dL within&#xD;
                  8 weeks OR&#xD;
&#xD;
               3. In HTB participants, having received ≥ 4 units of RBCs for Hgb ≤ 9.0 g/dL within&#xD;
                  8 weeks&#xD;
&#xD;
          5. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related&#xD;
             to anemia.&#xD;
&#xD;
          6. Females of child-bearing potential and sexually active males must agree to use&#xD;
             effective methods of contraception.&#xD;
&#xD;
        Key Exclusion Criteria:&#xD;
&#xD;
          1. Any active infection requiring parenteral antibiotic therapy within 28 days prior to&#xD;
             Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1.&#xD;
&#xD;
          2. Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical&#xD;
             injury or treatment with chemotherapy and/or radiation for other diseases).&#xD;
&#xD;
          3. Vitamin B12 deficiency.&#xD;
&#xD;
          4. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or&#xD;
             sotatercept.&#xD;
&#xD;
          5. Treatment within 28 days prior to Cycle 1 Day 1 with:&#xD;
&#xD;
               1. Erythropoiesis stimulating agent (ESA) OR&#xD;
&#xD;
               2. Granulocyte colony-stimulating factor (G-CSF) OR&#xD;
&#xD;
               3. Granulocyte-macrophage colony-stimulating factor (GM-CSF)&#xD;
&#xD;
          6. Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.&#xD;
&#xD;
          7. Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.&#xD;
&#xD;
          8. Treatment with another investigational drug or device or approved therapy for&#xD;
             investigational use &lt; or = 28 days prior to Cycle 1 Day 1, or if the half-life of the&#xD;
             previous product is known, within 5 times the half-life prior to Cycle 1 Day 1,&#xD;
             whichever is longer.&#xD;
&#xD;
          9. Platelet count &gt; 450 x 10*9/L or &lt; 30 x 10*9/L.&#xD;
&#xD;
         10. Transferrin saturation &lt; 15%.&#xD;
&#xD;
         11. Ferritin &lt; 50 µg/L.&#xD;
&#xD;
         12. Folate &lt; 4.5 nmol/L (&lt; 2.0 ng/mL).&#xD;
&#xD;
         13. Vitamin B12 &lt; 148 pmol/L (&lt; 200 pg/mL).&#xD;
&#xD;
         14. Estimated glomerular filtration rate (GFR) &lt; 40 mL/min/1.73 m2 (as determined by the&#xD;
             Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI].&#xD;
&#xD;
         15. Pregnant or lactating females&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_contact>
    <last_name>Clinical Study Team</last_name>
    <phone>+1 (617) 314-6297</phone>
    <email>ker050-md-201@kerostx.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>University of Miami, Sylvester Comprehensive Cancer Center</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33136</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <contact>
      <last_name>Mikkael A Sekeres, MD</last_name>
    </contact>
    <investigator>
      <last_name>Mikkael A Sekeres, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>H. Lee Moffitt Cancer Center and Research Center</name>
      <address>
        <city>Tampa</city>
        <state>Florida</state>
        <zip>33612</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <contact>
      <last_name>Rami Komrokji</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>University of Pittsburgh Medical Health Center</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15213</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <contact>
      <last_name>Robert Redner</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Border Medical Oncology Research Unit</name>
      <address>
        <city>Albury</city>
        <state>New South Wales</state>
        <zip>2640</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Anish Puliyayil</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>The Tweed Hospital</name>
      <address>
        <city>Tweed Heads</city>
        <state>New South Wales</state>
        <zip>2485</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Alejandro Arbelaez</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Westmead Hospital</name>
      <address>
        <city>Westmead</city>
        <state>New South Wales</state>
        <zip>2145</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>John Kwan</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Townsville University Hospital</name>
      <address>
        <city>Douglas</city>
        <state>Queensland</state>
        <zip>4814</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Joel Wight</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Royal Adelaide Hospital</name>
      <address>
        <city>Adelaide</city>
        <state>South Australia</state>
        <zip>5000</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Devendra Hiwase</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Flinders Medical Centre</name>
      <address>
        <city>Bedford Park</city>
        <state>South Australia</state>
        <zip>5042</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>David Ross, MD</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Box Hill Hospital</name>
      <address>
        <city>Box Hill</city>
        <state>Victoria</state>
        <zip>3128</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Tse-Chieh Teh</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>University Hospital Geelong</name>
      <address>
        <city>Geelong</city>
        <state>Victoria</state>
        <zip>3220</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Hannah Rose</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Austin Health</name>
      <address>
        <city>Heidelberg</city>
        <state>Victoria</state>
        <zip>3084</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Chun Fong</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Royal Melbourne Hospital</name>
      <address>
        <city>Melbourne</city>
        <state>Victoria</state>
        <zip>3050</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Lynette Chee</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>St Vincent's Hospital Melbourne</name>
      <address>
        <city>Melbourne</city>
        <state>Victoria</state>
        <zip>3065</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Shuh Ying Tan</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Ballarat Oncology and Haematology Service</name>
      <address>
        <city>Wendouree</city>
        <state>Victoria</state>
        <zip>3355</zip>
        <country>Australia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>George Kannourakis, MD</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Fakultni Nemocnice Brno</name>
      <address>
        <city>Brno</city>
        <country>Czechia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jiri Mayer</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Fakultni Nemocnice Kralovske Vinohrady</name>
      <address>
        <city>Praha</city>
        <country>Czechia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Olga Cerna</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Vseobecna Fakultni Nemocnice Praha</name>
      <address>
        <city>Praha</city>
        <country>Czechia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Anna Jonasova</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Angers - Hôpital Hôtel Dieu</name>
      <address>
        <city>Angers</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sylvain Thepot</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU de Nantes - Hotel Dieu</name>
      <address>
        <city>Nantes</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Alice Garnier</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Nice - Hôpital de l'Archet</name>
      <address>
        <city>Nice</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Thomas Cluzeau</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Saint-Louis</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Lionel Ades</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CH René-Dubos</name>
      <address>
        <city>Pontoise</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Riad Benramdane</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU de Bordeaux - Hôpital Haut-Lévêque</name>
      <address>
        <city>Talence</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sophie Dimicoli Salazar</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Centre Hospitalier de la Région d'Annecy</name>
      <address>
        <city>Épagny</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Natacha Mauz</last_name>
    </contact>
    <contact_backup>
      <last_name>Charlotte Doublet</last_name>
    </contact_backup>
  </location>
  <location>
    <facility>
      <name>Klinikum Bayreuth GmbH</name>
      <address>
        <city>Bayreuth</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Alexander Kiani</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Marien Hospital Dusseldorf GMBH</name>
      <address>
        <city>Düsseldorf</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Aristoteles Giagounidis</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Universitaetsklinikum Duesseldorf AoeR</name>
      <address>
        <city>Düsseldorf</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Ulrich Germing</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Klinikum Esslingen GmbH</name>
      <address>
        <city>Esslingen</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Swen Wessendorf</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Universitaetsklinikum Leipzig AoeR</name>
      <address>
        <city>Leipzig</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Anne Kubasch</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz</name>
      <address>
        <city>Mainz</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <contact>
      <last_name>Daniel Sasca</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Universitaetsmedizin Rostock</name>
      <address>
        <city>Rostock</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Christoph Wittke</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Sheba Medical Center</name>
      <address>
        <city>Ramat Gan</city>
        <zip>52621</zip>
        <country>Israel</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Drorit Merkel</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Tel-Aviv Sourasky Medical Center</name>
      <address>
        <city>Tel Aviv</city>
        <zip>6423906</zip>
        <country>Israel</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Yakir Moshe</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Middlemore Hospital</name>
      <address>
        <city>Auckland</city>
        <zip>2025</zip>
        <country>New Zealand</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>James Liang</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Central de Asturias</name>
      <address>
        <city>Barcelona</city>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Teresa Bernal del Castillo</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Vall d'Hebron</name>
      <address>
        <city>Barcelona</city>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>David V Ferreiras</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>ICO l'Hospitalet - Hospital Duran i Reynals</name>
      <address>
        <city>Barcelona</city>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Montserrat Sangerman</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario de Salamanca</name>
      <address>
        <city>Salamanca</city>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Maria Campelo</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Virgen del Rocio</name>
      <address>
        <city>Sevilla</city>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jose Gonzalez</last_name>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hospital Universitari i Politecnic La Fe</name>
      <address>
        <city>Valencia</city>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Guillermo Santillana</last_name>
    </contact>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Czechia</country>
    <country>France</country>
    <country>Germany</country>
    <country>Israel</country>
    <country>New Zealand</country>
    <country>Spain</country>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>June 3, 2020</study_first_submitted>
  <study_first_submitted_qc>June 3, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 5, 2020</study_first_posted>
  <last_update_submitted>June 13, 2023</last_update_submitted>
  <last_update_submitted_qc>June 13, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 18, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>transfusion</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Preleukemia</mesh_term>
    <mesh_term>Myelodysplastic Syndromes</mesh_term>
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04419649
org study id: KER050-MD-201
nct id: NCT04419649
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
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.
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).
allocation: Non-Randomized
intervention model: Sequential Assignment
intervention model description: Ascending dose study
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence of adverse events (AEs) and serious adverse events (SAEs).
time frame: From treatment initiation to end of study, approximately 2 years
description: Type, frequency, severity of AEs and relationship of AEs to KER-050
measure: 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
description: Pharmacokinetics of KER-050
measure: 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
description: 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.
measure: 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
description: Change in baseline laboratory assessments prior to treatment with KER-050 and after treatment with KER-050
measure: 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
description: Change in baseline bone marrow assessments prior to treatment with KER-050 and after treatment with KER-050
measure: 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
description: 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.
measure: 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
description: Change in baseline hemoglobin prior to treatment with KER-050 and after treatment with KER-050
measure: 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
description: Time to erythroid response prior to treatment with KER-050 and after treatment with KER-050
measure: 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
description: Duration of erythroid response prior to treatment with KER-050 and after treatment with KER-050
measure: 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
description: Frequency and rate of RBC transfusions prior to treatment with KER-050 and after treatment with KER-050
measure: 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
description: Change in baseline RBC counts and reticulocytes prior to treatment with KER-050 and after treatment with KER-050
arm group label: KER-050 Cohort 1
arm group type: Experimental
description: 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.
arm group label: KER-050 Cohort 2
arm group type: Experimental
description: 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.
arm group label: KER-050 Cohort 3
arm group type: Experimental
description: 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.
arm group label: KER-050 Cohort 4
arm group type: Experimental
description: 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.
arm group label: KER-050 Cohort 5
arm group type: Experimental
description: 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.
arm group label: KER-050 Dose Confirmation Cohort
arm group type: Experimental
description: 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.
intervention type: Drug
intervention name: KER-050
description: 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.
arm group label: KER-050 Cohort 1
arm group label: KER-050 Cohort 2
arm group label: KER-050 Cohort 3
arm group label: KER-050 Cohort 4
arm group label: KER-050 Cohort 5
arm group label: KER-050 Dose Confirmation Cohort
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Clinical Study Team
phone: +1 (617) 314-6297
email: ker050-md-201@kerostx.com
facility: 
status: Not yet recruiting
contact: 
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country: Australia
country: Czechia
country: France
country: Germany
country: Israel
country: New Zealand
country: Spain
country: United States
responsible party type: Sponsor
mesh term: Preleukemia
mesh term: Myelodysplastic Syndromes
mesh term: Syndrome
sharing ipd: No