Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Pediatric Patients With CKD

Study Overview
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Brief Summary
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This open-labeled, one-center study is designed to evaluate the efficacy and safety of roxadustat in ESA-naïve and ESA-treated pediatric patients with CKD Stages 3, 4, and 5 who are receiving dialysis or not. The study will enroll patients between the ages of 2 to <18 years. Approximately 30 patients will be enrolled.

Detailed Description
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This study is to evaluate the use of roxadustat for renal anemia in pediatric patients with CKD

Official Title
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An Open-label, One-center Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Pediatric Patients With Chronic Kidney Disease

Conditions
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Anemia Associated With Chronic Kidney Disease

Intervention / Treatment
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* Drug: Roxadustat


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Estimated glomerular filtration rate (Bedside Schwartz formula) of < 60 ml/min/1.73 m2 (stage 3, 4 and 5 CKD) for dialysis or non-dialysis patients. For ESA-naïve patients, mean of two most recent central laboratory Hb values during the screening period obtained at least 2 days apart must be < 11.0 g/dL. For patients currently receiving stable ESA dosing who will discontinue ESA and convert to roxadustat during study, mean of two most recent central laboratory Hb values during the screening period obtained at least 2 days apart must be ≥ 9.0 g/dL and ≤ 12.5 g/dL Ferritin >50 ng/mL and transferrin saturation >10% Exclusion Criteria: Uncontrolled hypertension as judged by the principal investigator prior to screening. Known hematologic disease related anemia (including PRCA) Known malignancy within the past 5 years before screening. History of severe anaphylaxis or known allergic to ingredient of roxadusta. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3x upper limit of normal (ULN) and total bilirubin (Tbili) ≤2x ULN(obtained from screening visit). Serum folate and vitamin B12 > LLN and cannot be corrected (obtained from screening visit). Any RBC transfusion during the past 4 weeks before screening. Weight<10kg Any prior exposure to roxadustat or any other HIF-PH inhibitor.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Roxadustat<br>Starting doses of 20, 50, 70 or 100 mg/30,70,90,or 120mg based on weight and dialysis or not. | Drug: Roxadustat<br>* Starting doses of 20, 50, 70 or 100 mg/30,70,90,or 120mg based on weight and dialysis or not.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of patients with mean Hb ≥ 11.0 g/dL | After the use of roxadustat, the proportion of patients with mean Hb ≥ 11.0 g/dL | weeks 16-24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change in Hb |  | weeks 16-24 |