Study to Evaluate the Safety and Efficacy of Oral Vadadustat in Pediatric Participants With Anemia of Chronic Kidney Disease

Study Overview
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Brief Summary
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This study will assess the safety and efficacy of once daily dosing of vadadustat for the treatment of pediatric participants with anemia of Chronic Kidney Disease (CKD) after conversion from an Erythropoiesis Stimulating Agent (ESA).

Official Title
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A Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Once Daily Oral Vadadustat for the Treatment of Pediatric Subjects With Anemia of Chronic Kidney Disease After Conversion From an Erythropoiesis-stimulating Agent

Conditions
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Anemia of Chronic Kidney Disease

Intervention / Treatment
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* Drug: vadadustat


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of anemia of chronic kidney disease (CKD) Diagnosis of non-dialysis-dependent (NDD) CKD with an estimated glomerular filtration rate of greater than (>) 10 and less than (<) 60 milliliters/minute/1.73 meters^2 (mL/min/1.73 m^2) or diagnosis of dialysis dependent (DD) CKD Mean hemoglobin (Hb) between 9.0 and 12.0 grams/deciliters (g/dL) (inclusive) Transferrin Saturation ≥ 20% Exclusion Criteria: Anemia due to a cause other than CKD Active bleeding or recent clinically significant blood loss History of sickle cell disease, myelodysplastic syndromes, bone marrow fibrosis, hematologic malignancy, myeloma, hemolytic anemia, thalassemia, or pure red cell aplasia Red Blood Cells transfusion within 4 weeks Serum albumin level less than 2.5 g/dL Uncontrolled hypertension Active malignancy or treatment for malignancy within the past 2 years prior to Screening Evidence of iron overload or diagnosis of hemochromatosis Known hypersensitivity to vadadustat or any excipients in vadadustat tablet

Ages Eligible for Study
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Minimum Age: 4 Months
Maximum Age: 16 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 |
| --- | --- |
| Experimental: Vadadustat<br>Cohort 1: participants with ≥12 years to <17 years; Cohort 2: participants with ≥6 years to <12 years; Cohort 3(a): participants with ≥2 years to <6 years; and Cohort 3(b): participants with ≥4 months to <2 years | Drug: vadadustat<br>* Vadadustat tablet orally once a day for 52 weeks<br>* Other names: AKB-6548;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean Change in Hemoglobin (Hb) Values Between Baseline and the Primary Evaluation Period (Average Hb From Weeks 21 to 28) |  | Baseline; Weeks 21 to 28 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Achieve Hb Levels of ≥10.0 grams/deciliters (g/dL) |  | Up to Week 52 | 
| Number of Participants With Mean Hb Values Within the Target Range During the Primary Evaluation Period |  | From Week 21 to Week 28 | 
| Number of Participants With Mean Hb Values Within the Target Range During the Extension Period |  | From Week 29 to Week 52 | 
| Number of Participants With Treatment-emergent Adverse Events and who Discontinued From the Study due to Adverse Events |  | Up to Week 56 | 
| Maximum Observed Plasma Concentration (Cmax) of Vadadustat and its Metabolites |  | Pre-dose and post-dose at intermediate time points up to 28 weeks | 
| Time to Reach Cmax (Tmax) of Vadadustat and its Metabolites |  | Pre-dose and post-dose at intermediate time points up to 28 weeks | 
| Area Under the Plasma Concentration-Time Curve From 0 to Last Quantifiable Concentration (AUC 0-t) of Vadadustat and its Metabolites |  | Pre-dose and post-dose at intermediate time points up to 28 weeks | 
| Terminal Elimination Half-Life (t1/2) of Vadadustat and its Metabolites |  | Pre-dose and post-dose at intermediate time points up to 28 weeks | 
| Change From Baseline in Serum Erythropoietin (EPO) |  | Pre-dose and post-dose at intermediate time points up to 28 weeks | 
| Change From Baseline in Reticulocyte Count |  | Pre-dose and post-dose at intermediate time points up to 28 weeks | 
| Change From Baseline in Hb levels |  | Pre-dose and post-dose at intermediate time points up to 28 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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anemia, kidney disease, vadadustat, pediatric population, children, chronic kidney disease, erythropoiesis stimulating agent, dialysis, hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor