Study to Evaluate the Safety and Efficacy of Oral Vadadustat in Pediatric Participants With Anemia of Chronic Kidney Disease Naive to Erythropoiesis-Stimulating Agents

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) naive to erythropoiesis-stimulating agent (ESA) treatment.

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 Naive to Erythropoiesis-stimulating Agents

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 screening hemoglobin (Hb) <10.0 grams/deciliters (g/dL) Transferrin Saturation ≥ 20% Exclusion Criteria: Anemia due to a cause other than CKD Active bleeding or recent clinically significant blood loss Treatment with an erythropoiesis-stimulating agents (ESA) within 8 weeks prior to Screening 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 <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 First Hb Levels ≥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 | 
| 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 | 
| Time to Reach Cmax (Tmax) 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, chronic kidney disease, erythropoiesis stimulating agent, dialysis, vadadustat, pediatric population, children, kidney disease, hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor