CONVERSION: Study to Evaluate the Safety and Efficacy of Oral Vadadustat in Pediatric Participants With Anemia of Chronic Kidney Disease

Sponsor

Akebia Therapeutics (Industry)

Overall Status

Suspended

CT.gov ID

NCT05082571

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

1.6

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Anemia of Chronic Kidney Disease	Drug: vadadustat	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

71 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Jan 1, 2023

Anticipated Primary Completion Date :

Jul 1, 2026

Anticipated Study Completion Date :

Oct 1, 2026

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

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

Time to Achieve Hb Levels of ≥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]
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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

4 Months to 16 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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