ESPN 54th Annual Meeting

ESPN 2022


 
Safety and Efficacy of Vadadustat for the Treatment of Pediatric Patients With Chronic Kidney Disease (CKD)–Related Anemia
FRANZ SCHAEFER 1 ALICIA NEU 2 ROSA REAL 3 ANDREW BLAIR 4 CHRISTINE SOLINSKY 4 ZHIQUN ZHANG 4

1- HEIDELBERG UNIVERSITY
2- JOHNS HOPKINS UNIVERSITY
3- OTSUKA AMERICA PHARMACEUTICAL, INC.
4- AKEBIA THERAPEUTICS, INC.
 
Introduction:

Vadadustat (VADA) is an oral hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor. We aimed to assess the safety, efficacy, and pharmacokinetic (PK)/pharmacodynamic (PD) properties of once-daily VADA in pediatric patients with CKD-related anemia.

Material and methods:

Two phase 3, multicenter, single-arm, open-label studies are evaluating once-daily oral VADA for treatment of pediatric patients (≥4 months to <17 years) with dialysis-dependent (DD) or non–dialysis-dependent (NDD) CKD-related anemia either naïve to erythropoiesis-stimulating agents (ESA-naïve trial; NCT05082584) or after conversion from an ESA (ESA-treated trial; NCT05082571). Key inclusion criteria are diagnosis of anemia of CKD, estimated glomerular filtration rate of >10 and <60 mL/min/1.73 m2 or a diagnosis of DD-CKD, transferrin saturation ≥20%, and mean hemoglobin (Hb) ≥9.0 and ≤12.0 g/dL (ESA-treated trial) or <10.0 g/dL (ESA-naïve trial). Patients are being enrolled in staggered cohorts stratified by age (12-17 y, 6-11 y, 2-5 y, 4 mo-<2 y); target enrollment for each study is up to 71 patients. The treatment period will consist of a correction/conversion period (weeks 1–20), primary evaluation period (PEP) (weeks 21–28), and extension period (weeks 29–51), in which all patients will receive once-daily VADA from baseline through week 52. A 4-week safety follow-up period is planned after end of treatment (Figure 1). Primary efficacy endpoint is mean change in Hb between baseline and PEP. Additional endpoints include time to achieve Hb ≥10.0 g/dL and proportion of patients with mean Hb values of 10.0–12.0 g/dL during PEP and extension period; safety and tolerability, including adverse events; and PK/PD endpoints.

Results:

 These studies will be the first evaluation of VADA in pediatric patients with anemia of CKD.

Conclusions:

 Two studies are assessing safety, efficacy, and PK/PD of VADA in pediatric patients with CKD-related anemia (DD or NDD) either naïve to or after conversion from an ESA.