Chronic Kidney DiseasePhase 3NCT05970172
A Study of Roxadustat to Treat Anemia in Children and Teenagers With Chronic Kidney Disease
A Phase 3 study testing Roxadustat in people with Chronic Kidney Disease and Renal Anemia, run by Astellas Pharma Global Development, Inc. at 47 sites in 19 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Chronic Kidney Disease, Renal Anemia
- What is being tested?
- Roxadustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 98 participants
- Where
- 47 sitesin 19 countries
- Study dates
- 16 Jan 2024to 30 Oct 2027
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participant has a diagnosis of anemia in CKD Kidney Disease Outcomes Quality Initiative stages 3 or 4 or 5. This can include participants not on dialysis or dialysis dependent (DD) participants (including hemodialysis, peritoneal dialysis and hemodiafiltration participants).
- Participants not on dialysis must have an estimated glomerular filtration rate (Schwartz formula) of < 60 mL/min per 1.73 m^2.
- ESA-treated participants should have a screening Hb level, assessed via HemoCue, between 10.0 and 12.0 g/dL; ESA-naïve participants can have a Hb level ≤ 11 g/dL.
- Participant has a ferritin level > 100 ng/mL or a transferrin saturation (TSAT) value > 20%.
- Participant has an alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2 x upper limit of normal (ULN) and total bilirubin (TBL) ≤ 1.5 x ULN at enrollment visit.
- Participant is treated with an ESA or is ESA-naïve, where ESA status is defined as:
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- ESA-treated: Participant is taking a stable dose of an ESA for at least 4 weeks prior to screening.
- ESA-naïve: Participant has no prior ESA exposure OR participant's total prior ESA exposure ≤ 3 weeks within the preceding 4 weeks from screening OR participant was previously treated with and discontinued an ESA ≥ 8 weeks prior to screening.
- Female participant is not pregnant and at least 1 of the following conditions apply:
- Not a woman of childbearing potential (WOCBP)
- WOCBP who agrees to follow the contraceptive guidance from the time of informed consent through at least 4 weeks after final study intervention administration.
- Female participant must agree not to breastfeed starting at screening and throughout the study and for 4 weeks post-last roxadustat dose.
- Female participant must not donate ova starting at first administration of roxadustat and throughout the study period and for 4 weeks post-last roxadustat dose.
- Male participants with female partner(s) of childbearing potential (including breastfeeding partner) must agree to use contraception throughout the treatment period and for 4 weeks post-last roxadustat dose.
- Male participants must not donate sperm during the treatment period and for 4 weeks post-last roxadustat dose.
- Male participants with pregnant partner(s) must agree to remain abstinent or use a condom for the duration of the pregnancy throughout the study period and for 4 weeks post-last roxadustat dose.
- Participant and/or participant's parent or legal guardian agrees for the participant not to participate in another interventional study while participating in the present study.
Who cannot join
- Participant has received any investigational therapy within 28 days or 5 half-lives, whichever is longer, prior to screening.
- Participant has any medical condition, including active, systemic or clinically significant infection which may pose a safety risk to a participant in this study, which may confound the safety or activity assessment or may interfere with study participation making the participant unsuitable for study.
- Participant has a known or suspected hypersensitivity to roxadustat, related hypoxia-inducible factor-prolyl hydroxylase inhibitors (HIF-PHI), or any components of the formulation used.
- Participant has uncontrolled hypertension (defined as ≥ 95th percentile + 12 mm Hg or ≥ 140/90 mm Hg [whichever is lower] for participants < 13 years of age and ≥ 140/90 mm Hg for participants ≥ 13 years of age measured 3 times at the same visit) in the 2 weeks prior to screening.
- Participant has a known hematologic disease other than anemia secondary to renal disease,(e.g., history of sickle cell disease, sickle cell anemia, hemoglobin sickle cell disease, or hemoglobin sickle cell beta thalassemia).
- Participant has untreated hypothyroidism.
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- Participant has severe hyperparathyroidism defined as serum parathyroid hormone (PTH) levels above 1000 pg/mL intact PTH within 4 weeks of screening.
- Participant has a functioning kidney allograft.
- Participant has a folate or B12 or carnitine deficiency. Acceptable if treated to normal values within 4 weeks of screening.
- Participant has a known active malignancy or malignancy within 18 months before the screening visit. Radiation or chemotherapy must be completed at least 12 months before the screening visit.
- Participant has a scheduled living donor organ transplantation date within 12 weeks of screening. If participant becomes eligible for a kidney transplant during study conduct, the participant should be discontinued.
- Participant has a whole blood or packed red blood cells (pRBC) transfusion during the 8 weeks prior to screening.
- Participant has any current condition leading to active significant blood loss in the past 4 weeks.
- Participant has a diagnosis of hemolytic uremic syndrome within 12 weeks prior to screening.
- Participant who has a previous diagnosis of atypical hemolytic syndrome must be relapse-free (stable hemoglobin (Hb), normal platelet count, normal serum lactate dehydrogenase, and normal haptoglobin level) for more than 12 weeks prior to screening.
- Participant has a history of chronic liver disease, including comorbidity with autosomal recessive polycystic kidney disease, cystinosis, and primary hyperoxaluria.
- Participant had an episode of peritonitis within 30 days of screening.
- Participant has active inflammation such as glomerulonephritis flare (i.e., lupus nephritis, immunoglobulin A (IgA) nephritis, rapidly progressive glomerulonephritis, membranoproliferative glomerulonephritis, antineutrophil cytoplasmic antibodies vasculitis) requiring pulse corticosteroid treatment or induction treatment with an immunosuppressive agent (i.e., cyclophosphamide, rituximab, or another monoclonal antibody) within 6 weeks of screening visit. Receipt of monoclonal antibody or biologic for maintenance treatment of underlying condition is acceptable.
- Participant has a known history of human immunodeficiency virus infection.
- Participant has rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption or is allergic to peanut or soya.
Trial locations and contacts
47 sites in 19 countries.
47 sites in 19 countries
Turkey (Türkiye)10 sites
Site TR90001
Ankara
Site TR90007
Ankara
Site TR90010
Ankara
Site TR90003
Istanbul
Site TR90004
Istanbul
Site TR90008
Istanbul
Site TR90009
Istanbul
Site TR90005
İzmit
Site TR90006
Kayseri
Site TR90002
Manisa
United Kingdom7 sites
Site GB44005
Cardiff
Site GB44006
Glasgow
Site GB44008
Liverpool
Site GB44007
London
Site GB44003
Newcastle upon Tyne
Site GB44001
Nottingham
Site GB44004
Southampton
Belgium5 sites
Site BE32002
Brussels
Site BE32005
Brussels
Site BE32001
Edegem
Site BE32004
Ghent
Site BE32003
Leuven
About this study
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
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.
Read more
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.
- Conditions
- Chronic Kidney Disease, Renal Anemia
- Being tested
- Roxadustat
- Keywords
- Roxadustat, ASP1517, Pediatric, Renal Anemia, Chronic renal failure, Chronic Kidney Disease, Renal Insufficiency, Chronic, Anemia, Open-label, Uncontrolled, Safety, Pharmacokinetics, Pharmacodynamics
- Medical subject headings
- Renal Insufficiency, Chronic, Kidney Failure, Chronic, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RoxadustatDrug
Also called ASP1517, FG-4592, Evrenzo
Oral
| Study group | Role | What they receive |
|---|---|---|
| Roxadustat | Experimental | 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. Receives: Drug: Roxadustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Hb level to the average Hb level over treatment weeks 20 to 24
Mean change in Hb level between baseline and average Hb level over treatment weeks 20 to 24.
Measured: Baseline and weeks 20 to 24
Secondary outcomes (23)
Pharmacokinetics (PK) of roxadustat in plasma: Maximum concentration (Cmax)
Cmax will be recorded from the PK plasma samples collected.
Measured: Up to week 8
PK of roxadustat in plasma: Area under the plasma-concentration time curve (AUC)
AUC will be recorded from the PK plasma samples collected.
Measured: Up to week 8
PK of roxadustat in plasma: Apparent total clearance (CL/F)
CL/F will be recorded from the PK plasma samples collected.
Measured: Up to week 8
PK of roxadustat in plasma: Time of the maximum concentration (Tmax)
Tmax will be recorded from the PK plasma samples collected.
Measured: Up to week 8
Hb levels at all timepoints
Hb levels at all timepoints will be recorded.
Measured: Up to week 56
Dose titration history at all timepoints
Dose titration history at all timepoints will be recorded.
Measured: Up to week 52
Number of participants with treatment-emergent adverse events (TEAEs)
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.
Measured: From first dose of study drug and up to week 56
Number of participants with treatment-emergent cardiovascular AEs
Treatment-emergent cardiovascular AEs will be recorded.
Measured: From first dose of study drug and up to week 56
Number of participants with treatment-emergent thrombotic AEs
Treatment-emergent thrombotic AEs will be recorded.
Measured: From first dose of study drug and up to week 56
Number of participants with laboratory value abnormalities and/or AEs
Number of participants with potentially clinically significant laboratory values.
Measured: Up to week 52
Number of participants with vital sign abnormalities and/or AEs
Number of participants with potentially clinically significant vital sign values.
Measured: Up to week 52
Change from baseline in growth parameter value: Height
Height will be recorded at all timepoints.
Measured: Baseline and up to week 52
Change from baseline in growth parameter value: Weight
Weight will be recorded at all timepoints.
Measured: Baseline and up to week 52
Number of participants with physical examinations abnormalities and/or AEs
Number of participants with potentially clinically significant physical examination abnormalities.
Measured: Up to week 52
Percentage of participants with Hb response
Response is defined as a participant achieving 2 consecutive Hb values within 1 g/dL of baseline (ESA-treated participants only) or achieving 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.
Measured: Up to Week 24
Mean monthly intravenous (IV) iron use
Summaries of monthly IV iron usage (number of administrations) will be recorded.
Measured: Up to week 52
Mean Monthly total IV iron dosage
Summaries of monthly IV iron usage (total dosage) will be recorded.
Measured: Up to week 52
Percentage of participants using IV iron
Percentage of participants using IV iron by study period will be recorded.
Measured: Up to week 52
Percentage of participants using oral iron
Percentage of participants using oral iron by study period will be recorded.
Measured: Up to week 52
Percentage of participants using rescue therapy
Percentage of participants using rescue therapy by study period will be recorded.
Measured: Up to week 52
Palatability questionnaire
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.
Measured: Up to week 52
Change from baseline in Pediatric Quality of Life (PedsQL) Inventory Score
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.
Measured: Baseline and up to week 52
Change from baseline in PedsQL Multidimensional Fatigue Scale questionnaire QoL Score
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.
Measured: Baseline and up to week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2023
- Study start16 Jan 2024
- Primary completion30 Oct 2027 (estimated)
- Study completion30 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Kidney DiseaseStudies of the same condition, open and under way.
- More trials run by Astellas Pharma Global Development, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 98 participants (actual)
Sponsor and investigators
- Lead sponsor
- Astellas Pharma Global Development, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Astellas Pharma Global Development, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05970172
- Sponsor's protocol ID
- 1517-CL-1003
- Other IDs
- 2022-501980-42-00
- Status last verified
- Oct 2026
- First submitted
- 25 Jul 2023
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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 products 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. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.