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

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

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.

Study Overview

• Status: Suspended
• Conditions: Anemia of Chronic Kidney Disease
• Intervention / Treatment: Drug: Vadadustat

• Study Type: Interventional
• Enrollment (Estimated): 71
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 months to 16 years (Child)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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	Drug: Vadadustat; Vadadustat tablet orally once a day for 52 weeks; Other Names: AKB-6548

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	Time Frame
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
Time to Achieve First Hb Levels ≥10.0 grams/deciliters (g/dL)	Up to Week 52

Collaborators and Investigators

• Sponsor: Akebia Therapeutics
• Investigators: Study Director: Chief Medical Officer, Akebia Therapeutics Inc.

Study record dates

Study Major Dates

• Study Start (Estimated): January 1, 2025
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): October 1, 2026

Study Registration Dates

• First Submitted: September 28, 2021
• First Submitted That Met QC Criteria: October 14, 2021
• First Posted (Actual): October 19, 2021

Study Record Updates

• Last Update Posted (Actual): October 10, 2023
• Last Update Submitted That Met QC Criteria: October 9, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: children; anemia; kidney disease; vadadustat; pediatric population; chronic kidney disease; erythropoiesis stimulating agent; dialysis; hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor
• Additional Relevant MeSH Terms: Pathologic Processes; Urologic Diseases; Disease Attributes; Hematologic Diseases; Renal Insufficiency; Chronic Disease; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Renal Insufficiency, Chronic; Anemia
• Other Study ID Numbers: AKB-6548-CI-0042

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No