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

Phase 3

Suspended

• Conditions: Anemia of Chronic Kidney Disease
• Interventions: Drug: Vadadustat

• Registration Number: NCT05082584
• Lead Sponsor: Akebia Therapeutics

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-09-28
• Study First Submitted QC: 2021-10-14
• Study First Posted: 2021-10-19
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-09
• Last Update Posted: 2023-10-10
• Study Start: 2025-01
• Primary Completion: 2026-07
• Study Completion: 2026-10

Recruitment & Eligibility

• Status: SUSPENDED
• Sex: All
• Target Recruitment: 71

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%

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Vadadustat	Vadadustat	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

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Number of Participants With Mean Hb Values Within the Target Range During the Extension Period	From Week 29 to Week 52
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 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 Treatment-emergent Adverse Events and who Discontinued From the Study due to Adverse Events	Up to Week 56
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

Trial Locations

Locations (1)

Research Site; 🇺🇸 Hackensack, New Jersey, United States