Clinical trial NCT05082571: Study to Evaluate the Safety and Efficacy of Oral Vadadustat in Pediatric Participants With Anemia of Chronic Kidney Disease
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 After Conversion From an Erythropoiesis-stimulating Agent
Study type: INTERVENTIONAL
Overall status: SUSPENDED
Phase: PHASE3
Conditions: Anemia of Chronic Kidney Disease
Keywords: anemia
kidney disease
vadadustat
pediatric population
children
chronic kidney disease
erythropoiesis stimulating agent
dialysis
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) after conversion from an Erythropoiesis Stimulating Agent (ESA).


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 hemoglobin (Hb) between 9.0 and 12.0 grams/deciliters (g/dL) (inclusive)
* Transferrin Saturation ≥ 20%

Exclusion Criteria:

* Anemia due to a cause other than CKD
* Active bleeding or recent clinically significant blood loss
* 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 less than 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 Hb Levels of ≥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: 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: 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: 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: