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

NCT05082584 | Withdrawn Phase 3 DMC FDA Drug

• 1 other identifier: AKB-6548-CI-0042
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Anemia of Chronic Kidney Disease

Keywords

anemia; chronic kidney disease; erythropoiesis stimulating agent; dialysis; vadadustat; pediatric population; children; kidney disease; hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (11)

• 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

Study Arms (1)

Vadadustat

EXPERIMENTAL

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

Interventions

Vadadustat DRUG

Vadadustat tablet orally once a day for 52 weeks

Also known as: AKB-6548

Vadadustat

Eligibility Criteria

• Age: 4 Months - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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%

You may not qualify if:

• 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

Sponsors & Collaborators

• Akebia Therapeutics: lead

Study Sites (1)

Research Site

Hackensack, New Jersey, 07601, United States

Location

MeSH Terms

Conditions

Anemia; Renal Insufficiency, Chronic; Kidney Diseases

Interventions

vadadustat

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Renal Insufficiency; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Chief Medical Officer

  Akebia Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 28, 2021
• First Posted: October 19, 2021
• Study Start: January 1, 2025
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: October 1, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)