xml: <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/NCT05970172</url>
    </required_header>
    <id_info>
      <org_study_id>1517-CL-1003</org_study_id>
      <nct_id>NCT05970172</nct_id>
    </id_info>
    <brief_title>A Study of Roxadustat to Treat Anemia in Children and Teenagers With Chronic Kidney Disease</brief_title>
    <official_title>A Phase 3, Open-label, Uncontrolled Study to Evaluate the Activity, Safety, Pharmacokinetics and Pharmacodynamics of Roxadustat for the Treatment of Anemia in Pediatric Participants With Chronic Kidney Disease</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Astellas Pharma Global Development, Inc.</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Astellas Pharma Inc</source>
    <oversight_info>
      <has_dmc>Yes</has_dmc>
      <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
      <is_us_export>Yes</is_us_export>
    </oversight_info>
    <brief_summary>
      <textblock>
        Roxadustat is a licensed medicine to treat anemia in adults with chronic kidney disease&#xD;
        (CKD). Anemia is a low level of red blood cells. Current treatment for anemia is to have&#xD;
        injections of medicines called erythropoietin stimulating agents (also known as ESAs) to help&#xD;
        the bone marrow make more red blood cells. These are often given together with iron. This&#xD;
        treatment is also available to children and teenagers with CKD. However, there are some&#xD;
        safety concerns with ESAs. Also, as roxadustat is taken orally, this may be another option&#xD;
        for treating anemia in children and teenagers with CKD. In this study, children and teenagers&#xD;
        with CKD and anemia will take roxadustat for up to 52 weeks to treat their anemia.&#xD;
  &#xD;
        The main aim of the study is to learn how roxadustat affects anemia in children and teenagers&#xD;
        with CKD.&#xD;
  &#xD;
        This is an open-label study which means the children and teenagers in the study and the&#xD;
        clinic staff know they will be taking roxadustat. In this study, the children and teenagers&#xD;
        with CKD who need treatment for anemia can take part. Those currently being treated with an&#xD;
        ESA will be switched to roxadustat. Those who have not been treated with an ESA can start on&#xD;
        roxadustat straight away. All children and teenagers in the study will take roxadustat 3&#xD;
        times a week for up to 52 weeks (1 year). They will start on a fixed dose of roxadustat for 4&#xD;
        weeks. Blood samples will be taken regularly to check hemoglobin levels. The roxadustat dose&#xD;
        may be changed if the blood levels of hemoglobin are too high, too low, or change too&#xD;
        quickly. After 4 weeks the dose may be changed, if needed, to keep blood levels of hemoglobin&#xD;
        in the blood to just below the normal range.&#xD;
  &#xD;
        Firstly, teenagers will take roxadustat. 10 teenagers will take their fixed dose of&#xD;
        roxadustat for 4 weeks. They will give blood samples to help the researchers work out the&#xD;
        most suitable dose for the rest of the teenagers in the study. When the rest of the teenagers&#xD;
        start taking roxadustat at the most suitable dose for teenagers, 10 children will take&#xD;
        roxadustat for 4 weeks. These 10 children will give blood samples to help the researchers&#xD;
        work out the most suitable dose for the rest of the children in the study. Then, the rest of&#xD;
        the children will take roxadustat at the most suitable dose for children.&#xD;
  &#xD;
        There will be many clinic visits during the study. Overnight hospital stays are not expected.&#xD;
        There will be 1 visit every 2 weeks for the first 4 weeks of taking roxadustat, then every 4&#xD;
        weeks until the end of treatment. Finally there is 1 visit 4 weeks after treatment has&#xD;
        finished.&#xD;
  &#xD;
        During most visits, the children and teenagers will have their vital signs checked (blood&#xD;
        pressure, body temperature and heart rate). Fluid status (how much water is in the body) will&#xD;
        also be checked for those who need dialysis. The children and teenagers will also have blood&#xD;
        tests and the study doctors will check for any medical problems. The children and teenagers&#xD;
        will have a medical examination before their first dose of roxadustat and again at about&#xD;
        24-week (6-month) and 52-week (13-month) visits. They will have an electrocardiogram (ECG)&#xD;
        before their first dose of roxadustat and again at the 12-week, 24-week, 36-week, and 52-week&#xD;
        visit. They will also have urine tests at the 4-week, 24-week and 52-week visits. At the&#xD;
        52-week visit, the children and teenagers will also have blood tests for hemoglobin and iron&#xD;
        levels. The study doctors will also check for any medical problems.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">November 30, 2023</start_date>
    <completion_date type="Anticipated">June 30, 2026</completion_date>
    <primary_completion_date type="Anticipated">November 30, 2025</primary_completion_date>
    <phase>Phase 3</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>N/A</allocation>
      <intervention_model>Single Group Assignment</intervention_model>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Change from baseline in Hb level to the average Hb level over treatment weeks 20 to 24</measure>
      <time_frame>Baseline and weeks 20 to 24</time_frame>
      <description>Mean change in Hb level between baseline and average Hb level over treatment weeks 20 to 24.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Pharmacokinetics (PK) of roxadustat in plasma: Maximum concentration (Cmax)</measure>
      <time_frame>Up to week 8</time_frame>
      <description>Cmax will be recorded from the PK plasma samples collected.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>PK of roxadustat in plasma: Area under the plasma-concentration time curve (AUC)</measure>
      <time_frame>Up to week 8</time_frame>
      <description>AUC will be recorded from the PK plasma samples collected.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>PK of roxadustat in plasma: Apparent total clearance (CL/F)</measure>
      <time_frame>Up to week 8</time_frame>
      <description>CL/F will be recorded from the PK plasma samples collected.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>PK of roxadustat in plasma: Time of the maximum concentration (Tmax)</measure>
      <time_frame>Up to week 8</time_frame>
      <description>Tmax will be recorded from the PK plasma samples collected.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Hb levels at all timepoints</measure>
      <time_frame>Up to week 56</time_frame>
      <description>Hb levels at all timepoints will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Dose titration history at all timepoints</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Dose titration history at all timepoints will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of participants with treatment-emergent adverse events (TEAEs)</measure>
      <time_frame>From first dose of study drug and up to week 56</time_frame>
      <description>An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of roxadustat, whether or not considered related to roxadustat. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of roxadustat. This includes events related to the comparator and events related to the (study) procedures. A TEAE is defined as an AE observed after starting administration of roxadustat through 28 days after the last dose of roxadustat.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of participants with treatment-emergent cardiovascular AEs</measure>
      <time_frame>From first dose of study drug and up to week 56</time_frame>
      <description>Treatment-emergent cardiovascular AEs will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of participants with treatment-emergent thrombotic AEs</measure>
      <time_frame>From first dose of study drug and up to week 56</time_frame>
      <description>Treatment-emergent thrombotic AEs will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of participants with laboratory value abnormalities and/or AEs</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Number of participants with potentially clinically significant laboratory values.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of participants with vital sign abnormalities and/or AEs</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Number of participants with potentially clinically significant vital sign values.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change from baseline in growth parameter value: Height</measure>
      <time_frame>Baseline and up to week 52</time_frame>
      <description>Height will be recorded at all timepoints.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change from baseline in growth parameter value: Weight</measure>
      <time_frame>Baseline and up to week 52</time_frame>
      <description>Weight will be recorded at all timepoints.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of participants with physical examinations abnormalities and/or AEs</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Number of participants with potentially clinically significant physical examination abnormalities.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of participants with Hb response</measure>
      <time_frame>Up to Week 24</time_frame>
      <description>For ESA-treated participants, response is defined as a participant achieving 2 consecutive Hb values within 1 g/dL of baseline or the target range of 10.0 to 12.0 g/dL at the end of the 24-week treatment evaluation period (i.e., weeks 20 to 24) without having received rescue therapy within 6 weeks prior to or during this 4-week evaluation period. For ESA-naïve participants, response is defined as a participant achieving 2 consecutive Hb values within the target range of 10.0 to 12.0 g/dL at the end of the 24-week treatment evaluation period (i.e., weeks 20 to 24) without having received rescue therapy within 6 weeks prior to or during this 4-week evaluation period</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Mean monthly intravenous (IV) iron use</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Summaries of monthly IV iron usage (number of administrations) will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Mean Monthly total IV iron dosage</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Summaries of monthly IV iron usage (total dosage) will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of participants using IV iron</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Percentage of participants using IV iron by study period will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of participants using oral iron</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Percentage of participants using oral iron by study period will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Percentage of participants using rescue therapy</measure>
      <time_frame>Up to week 52</time_frame>
      <description>Percentage of participants using rescue therapy by study period will be recorded.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Palatability questionnaire</measure>
      <time_frame>Up to week 52</time_frame>
      <description>The palatability questionnaire will assess taste and ability to swallow the medication using a 5 point Likert scale: for taste from 0 (Really bad) to 4 (Really good); for ability to swallow from 0 (Really Difficult) to 4 (Really Easy). The number of participants recorded for each response category will be provided.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change from baseline in Pediatric Quality of Life (PedsQL) Inventory Score</measure>
      <time_frame>Baseline and up to week 52</time_frame>
      <description>PedsQL Inventory Scale comprises of 23 items (except for ages 2-4 which has only 21 items, 3 of which are only answered if the child attends school or daycare) in 4 domains: physical functioning, emotional functioning, social functioning and school functioning. A Likert scale from 0 (Never) to 4 (Almost always) will be used to record response. Higher scores indicate better quality of life.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change from baseline in PedsQL Multidimensional Fatigue Scale questionnaire QoL Score</measure>
      <time_frame>Baseline and up to week 52</time_frame>
      <description>PedsQL Multidimensional Fatigue Scale comprises of 18 items in 3 domains: general fatigue, sleep/rest fatigue and cognitive fatigue. A Likert scale from 0 (Never) to 4 (Almost always) will be used to record response. Higher scores indicate better quality of life.</description>
    </secondary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">100</enrollment>
    <condition>Chronic Kidney Disease</condition>
    <condition>Renal Anemia</condition>
    <arm_group>
      <arm_group_label>Roxadustat</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Participants will receive roxadustat orally (or via gastric tube as an aqueous dispersion, if necessary) 3 times per week. The 24-week treatment period is defined as 4 weeks of fixed dose treatment followed by 20 weeks of dose titration(s). Dose titrations will be based on hemoglobin (Hb) monitoring. Participants in the study may receive roxadustat treatment for up to 52 weeks.</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Roxadustat</intervention_name>
      <description>Oral</description>
      <arm_group_label>Roxadustat</arm_group_label>
      <other_name>ASP1517</other_name>
      <other_name>FG-4592</other_name>
      <other_name>Evrenzo</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Participant has a diagnosis of anemia in CKD Kidney Disease Outcomes Quality&#xD;
               Initiative stages 3 or 4 or 5. This can include participants not on dialysis or&#xD;
               dialysis dependent (DD) participants (including hemodialysis, peritoneal dialysis and&#xD;
               hemodiafiltration participants).&#xD;
  &#xD;
            -  Participants not on dialysis must have an estimated glomerular filtration rate&#xD;
               (Schwartz formula) of &lt; 60 mL/min per 1.73 m^2.&#xD;
  &#xD;
            -  ESA-treated participants should have a screening Hb level, assessed via HemoCue,&#xD;
               between 10.0 and 12.0 g/dL; ESA-naïve participants can have a Hb level ≤ 11 g/dL.&#xD;
  &#xD;
            -  Participant has a ferritin level &gt; 100 ng/mL or a transferrin saturation (TSAT) value&#xD;
               &gt; 20%.&#xD;
  &#xD;
            -  Participant has an alanine aminotransferase (ALT) and aspartate aminotransferase (AST)&#xD;
               ≤ 2 x upper limit of normal (ULN) and total bilirubin (TBL) ≤ 1.5 x ULN at enrollment&#xD;
               visit.&#xD;
  &#xD;
            -  Participant is treated with an ESA or is ESA-naïve, where ESA status is defined as:&#xD;
  &#xD;
                 -  ESA-treated: Participant is taking a stable dose of an ESA for at least 4 weeks&#xD;
                    prior to screening.&#xD;
  &#xD;
                 -  ESA-naïve: Participant has no prior ESA exposure OR participant's total prior ESA&#xD;
                    exposure ≤ 3 weeks within the preceding 4 weeks from screening OR participant was&#xD;
                    previously treated with and discontinued an ESA ≥ 8 weeks prior to screening.&#xD;
  &#xD;
            -  Female participant is not pregnant and at least 1 of the following conditions apply:&#xD;
  &#xD;
                 -  Not a woman of childbearing potential (WOCBP)&#xD;
  &#xD;
                 -  WOCBP who agrees to follow the contraceptive guidance from the time of informed&#xD;
                    consent through at least 4 weeks after final study intervention administration.&#xD;
  &#xD;
            -  Female participant must agree not to breastfeed starting at screening and throughout&#xD;
               the study and for 4 weeks post-last roxadustat dose.&#xD;
  &#xD;
            -  Female participant must not donate ova starting at first administration of roxadustat&#xD;
               and throughout the study period and for 4 weeks post-last roxadustat dose.&#xD;
  &#xD;
            -  Male participants with female partner(s) of childbearing potential (including&#xD;
               breastfeeding partner) must agree to use contraception throughout the treatment period&#xD;
               and for 4 weeks post-last roxadustat dose.&#xD;
  &#xD;
            -  Male participants must not donate sperm during the treatment period and for 4 weeks&#xD;
               post-last roxadustat dose.&#xD;
  &#xD;
            -  Male participants with pregnant partner(s) must agree to remain abstinent or use a&#xD;
               condom for the duration of the pregnancy throughout the study period and for 4 weeks&#xD;
               post-last roxadustat dose.&#xD;
  &#xD;
            -  Participant and/or participant's parent or legal guardian agrees for the participant&#xD;
               not to participate in another interventional study while participating in the present&#xD;
               study.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Participant has received any investigational therapy within 28 days or 5 half-lives,&#xD;
               whichever is longer, prior to screening.&#xD;
  &#xD;
            -  Participant has any medical condition, including active, systemic or clinically&#xD;
               significant infection which may pose a safety risk to a participant in this study,&#xD;
               which may confound the safety or activity assessment or may interfere with study&#xD;
               participation making the participant unsuitable for study.&#xD;
  &#xD;
            -  Participant has a known or suspected hypersensitivity to roxadustat, related&#xD;
               hypoxia-inducible factor-prolyl hydroxylase inhibitors (HIF-PHI), or any components of&#xD;
               the formulation used.&#xD;
  &#xD;
            -  Participant has uncontrolled hypertension in the 2 weeks prior to screening.&#xD;
  &#xD;
            -  Participant has a known hematologic disease other than anemia secondary to renal&#xD;
               disease,(e.g., history of sickle cell disease, sickle cell anemia, hemoglobin sickle&#xD;
               cell disease, or hemoglobin sickle cell beta thalassemia).&#xD;
  &#xD;
            -  Participant has untreated hypothyroidism.&#xD;
  &#xD;
            -  Participant has severe hyperparathyroidism defined as serum parathyroid hormone (PTH)&#xD;
               levels above 1000 pg/mL intact PTH within 4 weeks of screening.&#xD;
  &#xD;
            -  Participant has a functioning kidney allograft.&#xD;
  &#xD;
            -  Participant has a folate or B12 or carnitine deficiency. Acceptable if treated to&#xD;
               normal values within 4 weeks of screening.&#xD;
  &#xD;
            -  Participant has a known active malignancy or malignancy within 18 months before the&#xD;
               screening visit. Radiation or chemotherapy must be completed at least 12 months before&#xD;
               the screening visit.&#xD;
  &#xD;
            -  Participant has a scheduled living donor organ transplantation date within 12 weeks of&#xD;
               screening.&#xD;
  &#xD;
            -  Participant has a whole blood or packed red blood cells (pRBC) transfusion during the&#xD;
               8 weeks prior to screening.&#xD;
  &#xD;
            -  Participant has any current condition leading to active significant blood loss in the&#xD;
               past 4 weeks.&#xD;
  &#xD;
            -  Participant has a diagnosis of hemolytic uremic syndrome within 12 weeks prior to&#xD;
               screening.&#xD;
  &#xD;
                 -  Participant who has a previous diagnosis of atypical hemolytic syndrome must be&#xD;
                    relapse-free (stable hemoglobin (Hb), normal platelet count, normal serum lactate&#xD;
                    dehydrogenase, and normal haptoglobin level) for more than 12 weeks prior to&#xD;
                    screening.&#xD;
  &#xD;
            -  Participant has a history of chronic liver disease, including comorbidity with&#xD;
               autosomal recessive polycystic kidney disease, cystinosis, and primary hyperoxaluria.&#xD;
  &#xD;
            -  Participant had an episode of peritonitis within 30 days of screening.&#xD;
  &#xD;
            -  Participant has active inflammation such as glomerulonephritis flare (i.e., lupus&#xD;
               nephritis, immunoglobulin A (IgA) nephritis, rapidly progressive glomerulonephritis,&#xD;
               membranoproliferative glomerulonephritis, antineutrophil cytoplasmic antibodies&#xD;
               vasculitis) requiring pulse corticosteroid treatment or induction treatment with an&#xD;
               immunosuppressive agent (i.e., cyclophosphamide, rituximab, or another monoclonal&#xD;
               antibody) within 6 weeks of screening visit. Receipt of monoclonal antibody or&#xD;
               biologic for maintenance treatment of underlying condition is acceptable.&#xD;
  &#xD;
            -  Participant has a known history of human immunodeficiency virus infection.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>2 Years</minimum_age>
      <maximum_age>17 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Medical Monitor</last_name>
      <role>Study Director</role>
      <affiliation>Astellas Pharma Global Development, Inc.</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Astellas Pharma Global Development, Inc.</last_name>
      <phone>800-888-7704</phone>
      <email>Astellas.registration@astellas.com</email>
    </overall_contact>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>July 25, 2023</study_first_submitted>
    <study_first_submitted_qc>July 25, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 1, 2023</study_first_posted>
    <last_update_submitted>July 25, 2023</last_update_submitted>
    <last_update_submitted_qc>July 25, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 1, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Roxadustat</keyword>
    <keyword>ASP1517</keyword>
    <keyword>Pediatric</keyword>
    <keyword>Renal Anemia</keyword>
    <keyword>Chronic renal failure</keyword>
    <keyword>Chronic Kidney Disease</keyword>
    <keyword>Renal Insufficiency, Chronic</keyword>
    <keyword>Anemia</keyword>
    <keyword>Open-label</keyword>
    <keyword>Uncontrolled</keyword>
    <keyword>Safety</keyword>
    <keyword>Pharmacokinetics</keyword>
    <keyword>Pharmacodynamics</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Kidney Diseases</mesh_term>
      <mesh_term>Renal Insufficiency, Chronic</mesh_term>
      <mesh_term>Anemia</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>Yes</sharing_ipd>
      <ipd_description>Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as compounds terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com.</ipd_description>
      <ipd_info_type>Study Protocol</ipd_info_type>
      <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
      <ipd_info_type>Clinical Study Report (CSR)</ipd_info_type>
      <ipd_time_frame>Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.</ipd_time_frame>
      <ipd_access_criteria>Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.</ipd_access_criteria>
      <ipd_url>https://www.clinicalstudydatarequest.com</ipd_url>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT05970172
  org study id: 1517-CL-1003
  nct id: NCT05970172
  lead sponsor: 
  has dmc: Yes
  is fda regulated drug: Yes
  is fda regulated device: No
  is us export: Yes
  Roxadustat is a licensed medicine to treat anemia in adults with chronic kidney disease
        (CKD). Anemia is a low level of red blood cells. Current treatment for anemia is to have
        injections of medicines called erythropoietin stimulating agents (also known as ESAs) to help
        the bone marrow make more red blood cells. These are often given together with iron. This
        treatment is also available to children and teenagers with CKD. However, there are some
        safety concerns with ESAs. Also, as roxadustat is taken orally, this may be another option
        for treating anemia in children and teenagers with CKD. In this study, children and teenagers
        with CKD and anemia will take roxadustat for up to 52 weeks to treat their anemia.
  
        The main aim of the study is to learn how roxadustat affects anemia in children and teenagers
        with CKD.
  
        This is an open-label study which means the children and teenagers in the study and the
        clinic staff know they will be taking roxadustat. In this study, the children and teenagers
        with CKD who need treatment for anemia can take part. Those currently being treated with an
        ESA will be switched to roxadustat. Those who have not been treated with an ESA can start on
        roxadustat straight away. All children and teenagers in the study will take roxadustat 3
        times a week for up to 52 weeks (1 year). They will start on a fixed dose of roxadustat for 4
        weeks. Blood samples will be taken regularly to check hemoglobin levels. The roxadustat dose
        may be changed if the blood levels of hemoglobin are too high, too low, or change too
        quickly. After 4 weeks the dose may be changed, if needed, to keep blood levels of hemoglobin
        in the blood to just below the normal range.
  
        Firstly, teenagers will take roxadustat. 10 teenagers will take their fixed dose of
        roxadustat for 4 weeks. They will give blood samples to help the researchers work out the
        most suitable dose for the rest of the teenagers in the study. When the rest of the teenagers
        start taking roxadustat at the most suitable dose for teenagers, 10 children will take
        roxadustat for 4 weeks. These 10 children will give blood samples to help the researchers
        work out the most suitable dose for the rest of the children in the study. Then, the rest of
        the children will take roxadustat at the most suitable dose for children.
  
        There will be many clinic visits during the study. Overnight hospital stays are not expected.
        There will be 1 visit every 2 weeks for the first 4 weeks of taking roxadustat, then every 4
        weeks until the end of treatment. Finally there is 1 visit 4 weeks after treatment has
        finished.
  
        During most visits, the children and teenagers will have their vital signs checked (blood
        pressure, body temperature and heart rate). Fluid status (how much water is in the body) will
        also be checked for those who need dialysis. The children and teenagers will also have blood
        tests and the study doctors will check for any medical problems. The children and teenagers
        will have a medical examination before their first dose of roxadustat and again at about
        24-week (6-month) and 52-week (13-month) visits. They will have an electrocardiogram (ECG)
        before their first dose of roxadustat and again at the 12-week, 24-week, 36-week, and 52-week
        visit. They will also have urine tests at the 4-week, 24-week and 52-week visits. At the
        52-week visit, the children and teenagers will also have blood tests for hemoglobin and iron
        levels. The study doctors will also check for any medical problems.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Change from baseline in Hb level to the average Hb level over treatment weeks 20 to 24
  time frame: Baseline and weeks 20 to 24
  description: Mean change in Hb level between baseline and average Hb level over treatment weeks 20 to 24.
  measure: Pharmacokinetics (PK) of roxadustat in plasma: Maximum concentration (Cmax)
  time frame: Up to week 8
  description: Cmax will be recorded from the PK plasma samples collected.
  measure: PK of roxadustat in plasma: Area under the plasma-concentration time curve (AUC)
  time frame: Up to week 8
  description: AUC will be recorded from the PK plasma samples collected.
  measure: PK of roxadustat in plasma: Apparent total clearance (CL/F)
  time frame: Up to week 8
  description: CL/F will be recorded from the PK plasma samples collected.
  measure: PK of roxadustat in plasma: Time of the maximum concentration (Tmax)
  time frame: Up to week 8
  description: Tmax will be recorded from the PK plasma samples collected.
  measure: Hb levels at all timepoints
  time frame: Up to week 56
  description: Hb levels at all timepoints will be recorded.
  measure: Dose titration history at all timepoints
  time frame: Up to week 52
  description: Dose titration history at all timepoints will be recorded.
  measure: Number of participants with treatment-emergent adverse events (TEAEs)
  time frame: From first dose of study drug and up to week 56
  description: An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of roxadustat, whether or not considered related to roxadustat. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of roxadustat. This includes events related to the comparator and events related to the (study) procedures. A TEAE is defined as an AE observed after starting administration of roxadustat through 28 days after the last dose of roxadustat.
  measure: Number of participants with treatment-emergent cardiovascular AEs
  time frame: From first dose of study drug and up to week 56
  description: Treatment-emergent cardiovascular AEs will be recorded.
  measure: Number of participants with treatment-emergent thrombotic AEs
  time frame: From first dose of study drug and up to week 56
  description: Treatment-emergent thrombotic AEs will be recorded.
  measure: Number of participants with laboratory value abnormalities and/or AEs
  time frame: Up to week 52
  description: Number of participants with potentially clinically significant laboratory values.
  measure: Number of participants with vital sign abnormalities and/or AEs
  time frame: Up to week 52
  description: Number of participants with potentially clinically significant vital sign values.
  measure: Change from baseline in growth parameter value: Height
  time frame: Baseline and up to week 52
  description: Height will be recorded at all timepoints.
  measure: Change from baseline in growth parameter value: Weight
  time frame: Baseline and up to week 52
  description: Weight will be recorded at all timepoints.
  measure: Number of participants with physical examinations abnormalities and/or AEs
  time frame: Up to week 52
  description: Number of participants with potentially clinically significant physical examination abnormalities.
  measure: Percentage of participants with Hb response
  time frame: Up to Week 24
  description: For ESA-treated participants, response is defined as a participant achieving 2 consecutive Hb values within 1 g/dL of baseline or the target range of 10.0 to 12.0 g/dL at the end of the 24-week treatment evaluation period (i.e., weeks 20 to 24) without having received rescue therapy within 6 weeks prior to or during this 4-week evaluation period. For ESA-naïve participants, response is defined as a participant achieving 2 consecutive Hb values within the target range of 10.0 to 12.0 g/dL at the end of the 24-week treatment evaluation period (i.e., weeks 20 to 24) without having received rescue therapy within 6 weeks prior to or during this 4-week evaluation period
  measure: Mean monthly intravenous (IV) iron use
  time frame: Up to week 52
  description: Summaries of monthly IV iron usage (number of administrations) will be recorded.
  measure: Mean Monthly total IV iron dosage
  time frame: Up to week 52
  description: Summaries of monthly IV iron usage (total dosage) will be recorded.
  measure: Percentage of participants using IV iron
  time frame: Up to week 52
  description: Percentage of participants using IV iron by study period will be recorded.
  measure: Percentage of participants using oral iron
  time frame: Up to week 52
  description: Percentage of participants using oral iron by study period will be recorded.
  measure: Percentage of participants using rescue therapy
  time frame: Up to week 52
  description: Percentage of participants using rescue therapy by study period will be recorded.
  measure: Palatability questionnaire
  time frame: Up to week 52
  description: The palatability questionnaire will assess taste and ability to swallow the medication using a 5 point Likert scale: for taste from 0 (Really bad) to 4 (Really good); for ability to swallow from 0 (Really Difficult) to 4 (Really Easy). The number of participants recorded for each response category will be provided.
  measure: Change from baseline in Pediatric Quality of Life (PedsQL) Inventory Score
  time frame: Baseline and up to week 52
  description: PedsQL Inventory Scale comprises of 23 items (except for ages 2-4 which has only 21 items, 3 of which are only answered if the child attends school or daycare) in 4 domains: physical functioning, emotional functioning, social functioning and school functioning. A Likert scale from 0 (Never) to 4 (Almost always) will be used to record response. Higher scores indicate better quality of life.
  measure: Change from baseline in PedsQL Multidimensional Fatigue Scale questionnaire QoL Score
  time frame: Baseline and up to week 52
  description: PedsQL Multidimensional Fatigue Scale comprises of 18 items in 3 domains: general fatigue, sleep/rest fatigue and cognitive fatigue. A Likert scale from 0 (Never) to 4 (Almost always) will be used to record response. Higher scores indicate better quality of life.
  arm group label: Roxadustat
  arm group type: Experimental
  description: Participants will receive roxadustat orally (or via gastric tube as an aqueous dispersion, if necessary) 3 times per week. The 24-week treatment period is defined as 4 weeks of fixed dose treatment followed by 20 weeks of dose titration(s). Dose titrations will be based on hemoglobin (Hb) monitoring. Participants in the study may receive roxadustat treatment for up to 52 weeks.
  intervention type: Drug
  intervention name: Roxadustat
  description: Oral
  arm group label: Roxadustat
  other name: ASP1517
  other name: FG-4592
  other name: Evrenzo
  criteria: 
  gender: All
  minimum age: 2 Years
  maximum age: 17 Years
  healthy volunteers: No
  last name: Medical Monitor
  role: Study Director
  affiliation: Astellas Pharma Global Development, Inc.
  last name: Astellas Pharma Global Development, Inc.
  phone: 800-888-7704
  email: Astellas.registration@astellas.com
  responsible party type: Sponsor
  mesh term: Kidney Diseases
  mesh term: Renal Insufficiency, Chronic
  mesh term: Anemia
  sharing ipd: Yes
  ipd description: Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as compounds terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com.
  ipd info type: Study Protocol
  ipd info type: Statistical Analysis Plan (SAP)
  ipd info type: Clinical Study Report (CSR)
  ipd time frame: Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.
  ipd access criteria: Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.
  ipd url: https://www.clinicalstudydatarequest.com