<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/NCT03303066</url>
  </required_header>
  <id_info>
    <org_study_id>FGCL-4592-813</org_study_id>
    <nct_id>NCT03303066</nct_id>
  </id_info>
  <brief_title>Efficacy and Safety of FG-4592 for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome (MDS)</brief_title>
  <official_title>A Phase 2/3 Trial of FG-4592 for Treatment of Anemia in Subjects With Lower Risk Myelodysplastic Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>FibroGen</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>AstraZeneca</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>FibroGen</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      The objective of this study to evaluate the efficacy FG-4592 for the treatment of anemia in&#xD;
      Chinese participants with lower risk MDS.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a Phase 2/3 trial with sequential Phase 2 and Phase 3 components. In each part, there&#xD;
      is an up to 4 weeks screening period followed by a treatment period of 26 weeks and a 4 week&#xD;
      follow-up period.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 6, 2018</start_date>
  <completion_date type="Actual">February 8, 2023</completion_date>
  <primary_completion_date type="Actual">January 11, 2023</primary_completion_date>
  <phase>Phase 2/Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
    <masking_description>Phase 2 part is an open label, single arm, dose-escalation study. Phase 3 component is a randomized, double blind, placebo-controlled study.</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion</measure>
    <time_frame>26 weeks</time_frame>
    <description>Hemoglobin response to FG-4592 is defined as an increase in mean Hb of ≥1.0 grams (g)/deciliter (dL) from baseline within any 8-week period during the study without transfusion.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With Hb Increase ≥1.0 g/dL From Baseline</measure>
    <time_frame>26 weeks</time_frame>
    <description>Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to First Hb Response</measure>
    <time_frame>26 weeks</time_frame>
    <description>Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Mean Hb in Weeks 25 to 27</measure>
    <time_frame>Baseline, Weeks 25 to 27</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of Participants With Mean Hb ≥10.0 g/dL Within Any 8-week Period During the Study Without Transfusion</measure>
    <time_frame>26 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants that Require Transfusions or Hb &lt;6 g/dL up to Week 27</measure>
    <time_frame>Up to Week 27</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Mean Change From Baseline in Functional Assessment of Cancer Therapy - Anemia (FACT-An) and Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) Scores at Week 27</measure>
    <time_frame>30 weeks</time_frame>
    <description>Frame: Baseline, Week 27</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">43</enrollment>
  <condition>Anemia</condition>
  <condition>Myelodysplastic Syndrome</condition>
  <arm_group>
    <arm_group_label>Phase 2 Part: FG-4592</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Fixed starting doses (different doses for lower body weight &amp; higher body weight) of FG-4592 administered orally 3 times a week (TIW) for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Phase 3 Part: FG-4592</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Fixed starting doses (different doses for lower body weight &amp; higher body weight) of FG-4592 administered orally TIW for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Phase 3 Part: Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo (matching to FG-4592) administered orally TIW for up to 26 weeks.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>FG-4592</intervention_name>
    <description>Oral</description>
    <arm_group_label>Phase 2 Part: FG-4592</arm_group_label>
    <arm_group_label>Phase 3 Part: FG-4592</arm_group_label>
    <other_name>Roxadustat</other_name>
    <other_name>AZD9941</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Oral</description>
    <arm_group_label>Phase 3 Part: Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Key Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of primary MDS classified as very low, low or intermediate risk with &lt;5%&#xD;
             blasts (documented within 12 weeks prior to Day 1)&#xD;
&#xD;
          -  Screening Hb &lt;10 g/dL and ≥6g/dL&#xD;
&#xD;
          -  Transfusion independent defined as no red blood cell transfusions within 12 weeks of&#xD;
             Day 1&#xD;
&#xD;
          -  Erythropoiesis-stimulating agent (ESA)-naïve (not within 30 days of Day 1)&#xD;
&#xD;
          -  Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2&#xD;
&#xD;
        Key Exclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of secondary MDS&#xD;
&#xD;
          -  Significant myelofibrosis (&gt;2+fibrosis)&#xD;
&#xD;
          -  Prior therapy with azacitidine, decitabine, antithymocyte globulin, cyclosporine,&#xD;
             thalidomide, or lenalidomide within 12 weeks prior to Day 1&#xD;
&#xD;
          -  Baseline erythropoietin level of &gt;400 units (U)/liter (L)&#xD;
&#xD;
          -  Clinically significant anemia due to non-MDS etiologies&#xD;
&#xD;
        Note: Other inclusion and exclusion criteria may apply.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>The First Affiliated Hospital of Bengbu Medical College</name>
      <address>
        <city>Bengbu</city>
        <state>Anhui</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Anhui Provincial Hospital</name>
      <address>
        <city>Hefei</city>
        <state>Anhui</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The First Affiliated Hospital of Anhui Medical University</name>
      <address>
        <city>Hefei</city>
        <state>Anhui</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>China-Japan Friendship Hospital</name>
      <address>
        <city>Beijing</city>
        <state>Beijing</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Peking Union Medical College Hospital</name>
      <address>
        <city>Beijing</city>
        <state>Beijing</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Peking University First Hospital</name>
      <address>
        <city>Beijing</city>
        <state>Beijing</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Peking University Third Hospital</name>
      <address>
        <city>Beijing</city>
        <state>Beijing</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Xiyuan Hospital, CACMS</name>
      <address>
        <city>Beijing</city>
        <state>Beijing</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fujian Medical University Union Hospital</name>
      <address>
        <city>Fuzhou</city>
        <state>Fujian</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Lanzhou University Second Hospital</name>
      <address>
        <city>Lanzhou</city>
        <state>Gansu</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Guangdong General Hospital</name>
      <address>
        <city>Guangzhou</city>
        <state>Guangdong</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nan Fang Hospital</name>
      <address>
        <city>Guangzhou</city>
        <state>Guangdong</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Zhuzhou Central Hospital</name>
      <address>
        <city>Zhuzhou</city>
        <state>Guangdong</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hainan Central Hospital</name>
      <address>
        <city>Haikou</city>
        <state>Hainan</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Second hospital of Hebei Medical University</name>
      <address>
        <city>Shijiazhuang</city>
        <state>Hebei</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tumor Hospital of Henan province</name>
      <address>
        <city>Zhengzhou</city>
        <state>Henan</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tongji Hospital, Tongji Medical College of HUST</name>
      <address>
        <city>Wuhan</city>
        <state>Hubei</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Union Hospital Affiliated Tongji Medical College Huazhong University of Science and Technolog</name>
      <address>
        <city>Wuhan</city>
        <state>Hubei</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Third Xiangya Hospital of Central South University</name>
      <address>
        <city>Changsha</city>
        <state>Hunan</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Jiangsu Province Hospital</name>
      <address>
        <city>Nanjing</city>
        <state>Jiangsu</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Zhongda Hospital Southeast University</name>
      <address>
        <city>Nanjing</city>
        <state>Jiangsu</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The First Affiliated Hospital Of Soochow University</name>
      <address>
        <city>Suzhou</city>
        <state>Jiangsu</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Northern Jiangsu People's Hospital</name>
      <address>
        <city>Yangzhou</city>
        <state>Jiangsu</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Affiliated Hospital of Shandong University of Traditional Chinese Medicine</name>
      <address>
        <city>Jinan</city>
        <state>Shandong</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Qilu Hospital of Shandong University</name>
      <address>
        <city>Jinan</city>
        <state>Shandong</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Affiliated Hospital of Xuzhou Medical University</name>
      <address>
        <city>Jinan</city>
        <state>Shandong</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Huashan Hospital Affiliated to Fudan University</name>
      <address>
        <city>Shanghai</city>
        <state>Shanghai</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shanghai Sixth People's Hospital</name>
      <address>
        <city>Shanghai</city>
        <state>Shanghai</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shanghai Tongji Hospital</name>
      <address>
        <city>Shanghai</city>
        <state>Shanghai</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shaanxi Provincial People's Hospital</name>
      <address>
        <city>Xian</city>
        <state>Shanxi</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The First Affiliated Hospital of Xi'an Jiao Tong University Medical College</name>
      <address>
        <city>Xian</city>
        <state>Shanxi</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sichuan Provincial People's Hospital</name>
      <address>
        <city>Chengdu</city>
        <state>Sichuan</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>West China Hospital, Sichuan University</name>
      <address>
        <city>Chengdu</city>
        <state>Sichuan</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Blood disease hospital of Chinese Academy of Medical Sciences</name>
      <address>
        <city>Tianjin</city>
        <state>Tianjin</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Second Hospital of Tianjin Medical University</name>
      <address>
        <city>Tianjin</city>
        <state>Tianjin</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tianjin Medical University General Hospital</name>
      <address>
        <city>Tianjin</city>
        <state>Tianjin</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>First Affiliated Hospital of Kunming Medical University</name>
      <address>
        <city>Kunming</city>
        <state>Yunnan</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The First Affiliated Hospital, Zhejiang University Medical College</name>
      <address>
        <city>Hangzhou</city>
        <state>Zhejiang</state>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>April 2023</verification_date>
  <study_first_submitted>October 2, 2017</study_first_submitted>
  <study_first_submitted_qc>October 2, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">October 5, 2017</study_first_posted>
  <last_update_submitted>May 1, 2023</last_update_submitted>
  <last_update_submitted_qc>May 1, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 3, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Myelodysplastic Syndromes</keyword>
  <keyword>Anemia</keyword>
  <keyword>Hemoglobin (Hb)</keyword>
  <keyword>Low Risk Myelodysplastic Syndrome</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Preleukemia</mesh_term>
    <mesh_term>Anemia</mesh_term>
    <mesh_term>Myelodysplastic Syndromes</mesh_term>
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>
  <!-- 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/NCT03303066
org study id: FGCL-4592-813
nct id: NCT03303066
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
is us export: No
The objective of this study to evaluate the efficacy FG-4592 for the treatment of anemia in
      Chinese participants with lower risk MDS.
This is a Phase 2/3 trial with sequential Phase 2 and Phase 3 components. In each part, there
      is an up to 4 weeks screening period followed by a treatment period of 26 weeks and a 4 week
      follow-up period.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
masking description: Phase 2 part is an open label, single arm, dose-escalation study. Phase 3 component is a randomized, double blind, placebo-controlled study.
measure: Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion
time frame: 26 weeks
description: Hemoglobin response to FG-4592 is defined as an increase in mean Hb of ≥1.0 grams (g)/deciliter (dL) from baseline within any 8-week period during the study without transfusion.
measure: Number of Participants With Hb Increase ≥1.0 g/dL From Baseline
time frame: 26 weeks
description: Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion.
measure: Time to First Hb Response
time frame: 26 weeks
description: Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion.
measure: Change From Baseline in Mean Hb in Weeks 25 to 27
time frame: Baseline, Weeks 25 to 27
measure: Percentage of Participants With Mean Hb ≥10.0 g/dL Within Any 8-week Period During the Study Without Transfusion
time frame: 26 weeks
measure: Number of Participants that Require Transfusions or Hb <6 g/dL up to Week 27
time frame: Up to Week 27
measure: Mean Change From Baseline in Functional Assessment of Cancer Therapy - Anemia (FACT-An) and Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) Scores at Week 27
time frame: 30 weeks
description: Frame: Baseline, Week 27
arm group label: Phase 2 Part: FG-4592
arm group type: Experimental
description: Fixed starting doses (different doses for lower body weight & higher body weight) of FG-4592 administered orally 3 times a week (TIW) for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.
arm group label: Phase 3 Part: FG-4592
arm group type: Experimental
description: Fixed starting doses (different doses for lower body weight & higher body weight) of FG-4592 administered orally TIW for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.
arm group label: Phase 3 Part: Placebo
arm group type: Placebo Comparator
description: Placebo (matching to FG-4592) administered orally TIW for up to 26 weeks.
intervention type: Drug
intervention name: FG-4592
description: Oral
arm group label: Phase 2 Part: FG-4592
arm group label: Phase 3 Part: FG-4592
other name: Roxadustat
other name: AZD9941
intervention type: Drug
intervention name: Placebo
description: Oral
arm group label: Phase 3 Part: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: 80 Years
healthy volunteers: No
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country: China
responsible party type: Sponsor
mesh term: Preleukemia
mesh term: Anemia
mesh term: Myelodysplastic Syndromes
mesh term: Syndrome