Phase 2 Completed N=51 Randomized Quadruple-blind Treatment

Dose-Finding Study of Vadadustat in Japanese Subjects With Anemia Secondary to Non-Dialysis Dependent Chronic Kidney Disease (NDD-CKD)

Anemia · Non-dialysis Dependent Chronic Kidney Disease

Source: ClinicalTrials.gov NCT03054337 ↗

Enrolled (actual)

Serious AEs

10.8%

Results posted

Apr 2021

Primary outcomePrimary: Mean Change in Hemoglobin (Hb) Levels From Pre-treatment to the End of the Primary Efficacy Period — 0.43; 1.13; 1.62; -0.47 grams per deciliter (g/dL) — p=0.0045

Summary

This is a Phase 2, randomized, double-blind, placebo-controlled, dose-finding study to assess the efficacy, safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of orally administered vadadustat in Japanese participants with anemia secondary to Non-dialysis Dependent Chronic Kidney Disease (NDD-CKD).

Outcome Measures

Outcome	Result	p-value
PRIMARY Mean Change in Hemoglobin (Hb) Levels From Pre-treatment to the End of the Primary Efficacy Period	0.43; 1.13; 1.62; -0.47	0.0045 sig
SECONDARY Time to Reach the Target Hb Level of 10.0 to 12.0 g/dL From Baseline up to Week 16	56.8; 39.0; 25.6; 79.0; 71.0; 54.5	—
SECONDARY Mean Hb Levels at the End of the Primary Efficacy Period	10.392; 10.675; 11.162; 9.421	—
SECONDARY Mean Hb Levels at the End of the Dose Adjustment and Maintenance Period	10.973; 11.230; 11.342; 10.860; 11.425; 11.950	—
SECONDARY Number of Participants Who Achieved the Target Hb Level of 10.0 to 12.0 g/dL at the End of the Dose Adjustment and Maintenance Period	11; 8; 11; 5; 3; 2	—
SECONDARY Mean Change in Hb Between Pre-treatment and the End of the Dose Adjustment and Maintenance Period	0.982; 1.610; 1.779; 0.790; 1.538; 2.075	—
SECONDARY Mean Change in Red Blood Cell (RBC) Count and Absolute Reticulocyte Count From Baseline to the End of the Primary Efficacy Period	0.17; 0.34; 0.48; -0.17; 0.01; 0.01	0.0018 sig
SECONDARY Mean Change in RBC Count and Absolute Reticulocyte Count From Baseline to the End of the Dose Adjustment and Maintenance Period	0.305; 0.476; 0.593; 0.186; 0.438; 0.608	—
SECONDARY Mean Change in Hematocrit and Reticulocytes From Baseline to the End of the Primary Efficacy Period	2.13; 4.13; 6.07; -1.17; 0.16; 0.28	0.0010 sig
SECONDARY Mean Change in Hematocrit and Reticulocytes From Baseline to the End of the Dose Adjustment and Maintenance Period	3.28; 5.17; 5.39; 2.32; 5.80; 6.05	—
SECONDARY Mean Change in Iron and Total Iron Binding Capacity (TIBC) From Baseline to the End of the Primary Efficacy Period	-1.8; -2.4; 4.4; -7.4; 44.9; 75.2	0.3702
SECONDARY Mean Change in Iron and TIBC From Baseline to the End of the Dose Adjustment and Maintenance Period	11.7; 3.5; 11.5; -1.4; 5.5; 5.8	—
SECONDARY Mean Change in Transferrin Saturation (TSAT) From Baseline to the End of the Primary Efficacy Period	-4.96; -7.31; -6.78; -4.72	0.9092
SECONDARY Mean Change in TSAT From Baseline to the End of the Dose Adjustment and Maintenance Period	-1.47; -2.68; -0.19; -7.00; -5.03; -2.68	—
SECONDARY Mean Change in Ferritin and Hepcidin From Baseline to the End of the Primary Efficacy Period	-38.48; -69.36; -101.54; -11.44; -24.622; -40.819	0.1080
SECONDARY Mean Change in Ferritin and Hepcidin From Baseline to the End of the Dose Adjustment and Maintenance Period	-79.45; -62.35; -59.68; -69.26; -146.63; -59.43	—
SECONDARY Number of Participants Who Required Rescue With Erythropoiesis-stimulating Agents (ESAs) From Baseline to the End of the Primary Efficacy Period	0; 0; 0; 0	—
SECONDARY Number of Participants Who Required Rescue With ESAs From Baseline to the End of the Dose Adjustment and Maintenance Period	0; 2; 1; 0; 0; 0	—
SECONDARY Number of Participants Who Required Rescue With a RBC Transfusion From Baseline to the End of the Primary Efficacy Period	0; 0; 0; 0	—
SECONDARY Number of Participants Who Required Rescue With a RBC Transfusion From Baseline to the End of the Dose Adjustment and Maintenance Period	0; 1; 1; 0; 0; 1	—
SECONDARY Number of the Participants With the Indicated Number of Dose Adjustments From Baseline to the End of the Dose Adjustment and Maintenance Period	3; 1; 2; 0; 0; 1	—
SECONDARY Number of Participants Who Maintained Iron Sufficiency From Baseline to Week 6	7; 4; 4; 9; 5; 8	0.0895
SECONDARY Number of Participants Who Maintained Iron Sufficiency From Baseline to Week 16	6; 2; 4; 1; 1; 0	—
SECONDARY Plasma Concentration Profile of Vadadustat and Its Metabolites Using a Pre-dose Sample From Week 4	5530.9; 12955.8; 19291.5; 3914.7; 12358.6; 16586.2	—
SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (SAEs) in the Primary Efficacy Period	4; 7; 7; 5; 0; 0	—
SECONDARY Number of Participants With TEAEs and Treatment-emergent SAEs in the Dose Adjustment and Maintenance Period	9; 10; 6; 3; 3; 3	—

Eligibility Criteria

Inclusion Criteria

Male and female Japanese participants ≥20 years of age
Diagnosis of chronic kidney disease (CKD) based on an estimated glomerular filtration rate ≤60 milliliters per minute per 1.73 meters squared (mL/min/1.73 m^2)
Hemoglobin (Hb) ≤10.5 grams per deciliter (g/dL)
Not currently being treated with dialysis and not expected to start dialysis within 3 months of screening

Exclusion Criteria

Anemia due to a cause other than CKD or presence of active bleeding or recent blood loss
Sickle cell disease, myelodysplastic syndromes, bone marrow fibrosis, hematologic malignancy, myeloma, hemolytic anemia, thalassemia, or pure red cell aplasia
Red blood cell transfusion within 4 weeks prior to or during screening
Intravenous iron within 4 weeks prior to or during screening
Any use of erythropoiesis-stimulating agents within 6 weeks prior to or during screening

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03054337). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.