Clinical trial NCT05082584: Study to Evaluate the Safety and Efficacy of Oral Vadadustat in Pediatric Participants With Anemia of Chronic Kidney Disease Naive to Erythropoiesis-Stimulating Agents
Official title: 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
Study type: INTERVENTIONAL
Overall status: SUSPENDED
Phase: PHASE3
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
Lead sponsor: Akebia Therapeutics
Sponsor class: INDUSTRY
First posted: 2021-10-19
Enrollment: 71
Sex: ALL
Minimum age (years): 4
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Mean Change in Hemoglobin (Hb) Values Between Baseline and the Primary Evaluation Period (Average Hb From Weeks 21 to 28)
timeFrame: Baseline; Weeks 21 to 28
Secondary outcomes:
measure: Time to Achieve First Hb Levels ≥10.0 grams/deciliters (g/dL)
timeFrame: Up to Week 52
measure: Number of Participants With Mean Hb Values Within the Target Range During the Primary Evaluation Period
timeFrame: From Week 21 to Week 28
measure: Number of Participants With Mean Hb Values Within the Target Range During the Extension Period
timeFrame: From Week 29 to Week 52
measure: Number of Participants With Treatment-emergent Adverse Events and who Discontinued From the Study due to Adverse Events
timeFrame: Up to Week 56
measure: Maximum Observed Plasma Concentration (Cmax) of Vadadustat and its Metabolites
timeFrame: Pre-dose and post-dose at intermediate time points up to 28 weeks
measure: Area Under the Plasma Concentration-Time Curve From 0 to Last Quantifiable Concentration (AUC 0-t) of Vadadustat and its Metabolites
timeFrame: Pre-dose and post-dose at intermediate time points up to 28 weeks
measure: Time to Reach Cmax (Tmax) of Vadadustat and its Metabolites
timeFrame: Pre-dose and post-dose at intermediate time points up to 28 weeks
measure: Terminal Elimination Half-Life (t1/2) of Vadadustat and its Metabolites
timeFrame: Pre-dose and post-dose at intermediate time points up to 28 weeks
measure: Change From Baseline in Serum Erythropoietin (EPO)
timeFrame: Pre-dose and post-dose at intermediate time points up to 28 weeks
measure: Change From Baseline in Reticulocyte Count
timeFrame: Pre-dose and post-dose at intermediate time points up to 28 weeks
measure: Change From Baseline in Hb levels
timeFrame: Pre-dose and post-dose at intermediate time points up to 28 weeks
Other outcomes:
Locations:
city: Hackensack
country: United States
facility: Research Site
state: New Jersey
Study officials:
affiliation: Akebia Therapeutics Inc.
name: Chief Medical Officer
role: STUDY_DIRECTOR
References: