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Study on Roxadustat for Treating Anemia in Children with Chronic Kidney Disease

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the treatment of anemia in children and adolescents who have Chronic Kidney Disease (CKD). Anemia is a condition where the body does not have enough healthy red blood cells to carry oxygen, which can make a person feel tired and weak. CKD is a long-term condition where the kidneys do not work as well as they should. The study will use a medication called roxadustat, which is taken as a tablet. Roxadustat is being tested to see how well it works and how safe it is for treating anemia in young patients with CKD.

In this study, participants will take roxadustat tablets for a period of time, and their health will be monitored to see how the medication affects their anemia. The study will also compare roxadustat to other treatments that are commonly used for anemia, such as methoxy polyethylene glycol-epoetin beta, epoetin alfa, and darbepoetin alfa. These are medications that help the body produce more red blood cells. Some participants may receive a placebo, which looks like the real medication but does not contain any active ingredients.

The purpose of the study is to evaluate the effectiveness and safety of roxadustat in treating anemia in children and adolescents with CKD. Participants will be closely monitored by healthcare professionals throughout the study to ensure their safety and to gather information on how the medication affects their condition. The study aims to provide valuable information that could help improve the treatment of anemia in young patients with CKD in the future.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant will begin the treatment phase. This involves taking the medication roxadustat, which is used to treat anemia associated with chronic kidney disease.

    The participant will receive roxadustat in the form of film-coated tablets. The available dosages are 5 mg, 20 mg, and 50 mg. The medication is taken orally.

  2. Step 2

    Treatment period

    The treatment period lasts for 24 weeks. It begins with 4 weeks of a fixed dose of roxadustat, followed by 20 weeks where the dose may be adjusted based on the participant's response.

    During this time, the participant's hemoglobin (Hb) levels will be monitored to assess the effectiveness of the treatment.

  3. Step 3

    Monitoring and assessments

    Throughout the study, various assessments will be conducted. These include monitoring Hb levels, evaluating the participant's response to the medication, and adjusting the dosage as needed.

    Additional assessments include checking for any side effects, such as cardiovascular or thrombotic events, and conducting safety evaluations through laboratory tests and physical examinations.

  4. Step 4

    End of treatment evaluation

    At the end of the 24-week treatment period, the participant's Hb levels will be compared to baseline levels to determine the change and effectiveness of the treatment.

    The study will also evaluate the participant's quality of life and any changes in fatigue levels using specific questionnaires designed for pediatric participants.

  5. Step 5

    Extended observation

    After the initial 24-week treatment period, the study may continue to observe the participant for up to 52 weeks to gather additional data on the long-term effects and safety of roxadustat.

    During this time, the participant may continue to receive the medication and undergo regular assessments as outlined in the study protocol.

Who can join the trial?

15 criteria

  • Written permission and privacy agreement must be obtained from the participant or their parent/legal guardian before any study-related activities. This includes stopping any medications that are not allowed in the study.
  • Female participants must agree not to breastfeed from the start of the study until 4 weeks after the last dose of the study medication, roxadustat.
  • Female participants must not donate eggs from the start of the study until 4 weeks after the last dose of roxadustat.
  • Male participants with female partners who can become pregnant must use birth control during the study and for 4 weeks after the last dose of roxadustat.
  • Male participants must not donate sperm during the study and for 4 weeks after the last dose of roxadustat.
  • Male participants with pregnant partners must agree to not have sexual intercourse or use a condom during the partner's pregnancy and for 4 weeks after the last dose of roxadustat.
  • The participant and/or their parent or legal guardian must agree not to join another study while participating in this one.
  • The participant must be between 2 and 18 years old at the first study visit.
  • The participant must have been diagnosed with **anemia** (a condition where you don't have enough healthy red blood cells) related to **Chronic Kidney Disease (CKD)**, which is a long-term condition where the kidneys do not work as well as they should. This includes those not on dialysis or those on different types of dialysis.
  • Participants not on dialysis must have a kidney function test result of less than 60 mL/min per 1.73 m², which is a measure of how well the kidneys are working.
  • Participants who are already being treated with **ESA** (a medication that helps the body make more red blood cells) should have a hemoglobin level (a measure of red blood cells) between 10.0 and 12.0 g/dL. Those who have not been treated with ESA can have a hemoglobin level of 11 g/dL or less.
  • The participant must have a **ferritin** level (a measure of stored iron in the body) greater than 100 ng/mL or a **TSAT** (a measure of how much iron is available in the blood) value greater than 20%.
  • The participant must have **ALT** and **AST** (liver enzymes) levels that are no more than twice the normal limit and **TBL** (total bilirubin, a substance made by the liver) levels that are no more than 1.5 times the normal limit at the enrollment visit.
  • The participant must either be currently treated with ESA or have not been treated with ESA before. If treated, they must have been on a stable dose for at least 4 weeks before the study. If not treated, they must have had no ESA exposure or limited exposure in the 4 weeks before the study, or they must have stopped ESA treatment at least 8 weeks before the study.
  • Female participants must not be pregnant and must agree to use birth control if they can become pregnant, from the time they give consent to join the study until at least 4 weeks after the last dose of the study medication.

Who cannot join the trial?

10 criteria

  • Patients who have any other serious health condition that could interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent blood transfusion.
  • Patients who have a known allergy to the study medication or its ingredients.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who have a mental health condition that could affect their ability to participate in the study.
  • Patients who have a history of non-compliance with medical treatments.
  • Patients who have a condition that requires them to take medications that could interfere with the study medication.
  • Patients who have a history of certain types of cancer.
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Investigated drugs

Roxadustat is a medication being studied for its ability to treat anemia in children and adolescents who have chronic kidney disease. Anemia is a condition where the body does not have enough healthy red blood cells to carry oxygen to the tissues, which can cause fatigue and weakness. Roxadustat works by helping the body produce more red blood cells, which can improve energy levels and overall health in patients with chronic kidney disease. This study aims to understand how well roxadustat works, how safe it is, and how it behaves in the body of pediatric patients.

What is already known about the treatment

Roxadustat – Roxadustat is administered orally and is currently being studied in clinical trials for its effectiveness in treating anemia associated with chronic kidney disease, particularly in pediatric patients. It is not yet fully established in medical practice but shows promise in medical literature as a potential treatment option. The main therapeutic indication for roxadustat is anemia related to chronic kidney disease. At the molecular level, it works by inhibiting an enzyme called prolyl hydroxylase, which leads to increased production of erythropoietin, a hormone that stimulates red blood cell production. Roxadustat is classified pharmacologically as a hypoxia-inducible factor prolyl hydroxylase inhibitor.

Investigated diseases

Anemia associated with Chronic Kidney Disease – This condition occurs when the kidneys, which are responsible for filtering waste from the blood, are damaged and cannot produce enough of a hormone called erythropoietin. This hormone is crucial for the production of red blood cells. As a result, the body has fewer red blood cells, leading to reduced oxygen delivery to tissues and organs. Symptoms may include fatigue, weakness, and shortness of breath. The progression of this anemia is often gradual and correlates with the decline in kidney function. It is commonly seen in individuals with chronic kidney disease, especially in the later stages.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2022-501980-42-00Protocol code1517-CL-1003Estimated enrolment104 patientsSponsorAstellas Pharma Global Development Inc.

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