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42-Day Repeat Oral Dose Study of AKB-6548 in Participants With Chronic Kidney Disease and Anemia

Phase 2
Completed
Conditions
Kidney Disease
Anemia
Interventions
Drug: Placebo
Registration Number
NCT01381094
Lead Sponsor
Akebia Therapeutics
Brief Summary

The purpose of this study is to evaluate the dose response (efficacy), pharmacodynamic response, pharmacokinetics, safety, and tolerability of orally administered AKB-6548 in pre-dialysis participants with anemia with repeat dosing for 42 days.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
93
Inclusion Criteria
  • 18 to 79 years of age, inclusive
  • Chronic Kidney Disease (eGFR <60 mL/min), not yet on dialysis
  • Hemoglobin (Hgb) ≤ 10.5 g/dL
  • Transferring saturation ≥ 20%
  • Ferritin ≥ 50 ng/mL

Key

Exclusion Criteria
  • Body mass index >42
  • Red blood cell transfusion within 12 weeks
  • Androgen therapy within the previous 21 days prior to study dosing
  • Therapy with any approved or experimental erythropoiesis stimulating agent (ESA) within the 11 weeks prior to the Screening visit
  • Participants meeting the criteria of ESA resistance within the previous 4 months
  • Individual doses of intravenous iron of greater than 250 mg within the past 21 days
  • Aspartate aminotransferase or alanine aminotransferase >1.8x upper limit of normal (ULN)
  • Alkaline phosphatase >2x ULN
  • Total bilirubin >1.5x ULN
  • Uncontrolled hypertension
  • New York Heart Association Class III or IV congestive heart failure
  • Myocardial infarction, acute coronary syndrome, or stroke within 6 months prior to dosing

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
AKB-6548 630 mgAKB-6548-
AKB-6548 240 mgAKB-6548-
AKB-6548 500 mgAKB-6548-
AKB-6548 370 mgAKB-6548-
PlaceboPlacebo-
Primary Outcome Measures
NameTimeMethod
Absolute Change From Baseline in Hemoglobin (Hgb) to End of Treatment (Week 6)Baseline, Week 6

Absolute change from Baseline was calculated as the Week 6 (end of treatment) value minus the Baseline value. Baseline Hgb was defined as the average of the last two measurements obtained prior to dosing. If there was only one measurement prior to dosing, this measurement served as Baseline. A positive change from Baseline indicated that hemoglobin concentration increased.

Secondary Outcome Measures
NameTimeMethod
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs ParameterUp to Week 8 (Follow-up Visit 2 weeks after last dose)

Parameters assessed for vital signs included sitting blood pressure, pulse, respiratory rate, and body temperature. The investigator was responsible for reviewing laboratory results for clinically significant changes.

Number of Participants With Clinically Significant Abnormal 12-Electrocardiogram (ECG) FindingsUp to Week 8 (Follow-up Visit 2 weeks after last dose)

A standard 12-lead ECG was performed following dosing in a supine position for approximately 10 minutes. ECGs were taken prior to blood draws when possible. Clinical significance is determined by the investigator. The investigator was responsible for reviewing laboratory results for clinical significance.

Number of Participants With Clinically Significant Changes From Baseline in Laboratory Parameter ValuesUp to Week 8 (Follow-up Visit 2 weeks after last dose)

Parameters assessed for laboratory values included hematology, serum chemistry, C-reactive protein, DHEA-S, VEGF, and cystatin C. The investigator was responsible for reviewing laboratory results for clinically significant changes.

Change From Baseline in Red Blood Cell (RBC) Count at Week 1, Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 1, Week 2, Week 4, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline RBC count was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated RBC count increased.

Change From Baseline in Reticulocyte Hgb Content at Week 6Baseline, Week 6

Change from Baseline was calculated as the Week 6 value minus the Baseline value. Baseline Reticulocyte count was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated reticulocyte Hgb content increased.

Change From Baseline in Absolute Reticulocyte Count at Week 1, Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 1, Week 2, Week 4, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline Reticulocyte count was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated reticulocyte count increased.

Maximum Change From Baseline in HgbBaseline; up to Week 8

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline Hgb was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated that hemoglobin concentration increased.

Maximum Change From Baseline in HCTBaseline; up to Week 8

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline HCT was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated that HCT concentration increased.

Maximum Change From Baseline in RBC CountBaseline; up to Week 8

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline RBC Count was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated that RBC count increased.

Maximum Change in Absolute Reticulocyte Count From BaselineBaseline; up to Week 8

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline absolute reticulocyte count was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated that reticulocyte count increased.

Number of Participants With Absolute Change From Baseline in Hgb ≥ 0.4, 0.6, 0.8, and 1.0 g/dL at the End of Dosing PeriodUp to Week 6 (End of the Dosing Period)

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline Hgb was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Number of Participants With Change From Baseline in HCT ≥5.0, 7.5, and 10.0% by the End of Dosing PeriodUp to Week 6 (End of The Dosing Period)

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline HCT was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Change From Baseline in Total Iron at Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 2, Week 4, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline total iron was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Number of Participants With Change From Baseline in RBC Count ≥5.0, 7.5, and 10.0% by the End of Dosing PeriodUp to Week 6 (End of The Dosing Period)

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline RBC Count was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Change From Baseline in Unsaturated Iron Binding Capacity at Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 2, Week 4, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline Unsaturated Iron Binding Capacity was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Change From Baseline in Iron Saturation at Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 2, Week 4, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline iron saturation was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Change From Baseline in Total Iron Binding Capacity (TIBC) at Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 2, Week 4, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline TIBC was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Change From Baseline in Ferritin at Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 2, Week 4, Week 6, Follow-up (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline ferritin was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Change From Baseline in Erythropoietin at Week 2, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 2, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline erythropoietin was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Change From Baseline in Hepcidin at Week 6Baseline, Week 6

Change from Baseline was calculated as the Week 6 value minus the Baseline value. Baseline hepcidin was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Mean Plasma Vadadustat Concentrations on Week 2 and Week 4Week 2: Pre-dose and post-dose; Week 4: Pre-dose

Plasma samples were collected for the analysis.

Mean Plasma Vadadustat Acyl-Glucuronide Concentrations on Week 2 and Week 4Week 2: Pre-dose; Week 4: Pre-dose

Plasma samples were collected for the analysis.

Mean Change From Baseline in PR Interval, QT Interval, QRS Interval, and QT Corrected (QTc) IntervalBaseline, Week 6

A standard 12-lead ECG was performed following dosing in a supine position for approximately 10 minutes. ECGs were taken prior to blood draws when possible. The parameters evaluated from the participant ECG trace included PR interval, QT interval, QRS interval, and QTc (corrected).

Number of Participants With Change From Baseline in Hgb ≥5.0, 7.5, and 10.0% by the End of Dosing PeriodUp to Week 6 (End of The Dosing Period)

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline Hgb was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

Change From Baseline in Hgb at Week 1, Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 1, Week 2, Week 4, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline Hgb was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated that hemoglobin concentration increased.

Change From Baseline in Hematocrit (HCT) at Week 1, Week 2, Week 4, Week 6, and Follow-up Visit (up to Week 8)Baseline, Week 1, Week 2, Week 4, Week 6, Follow-up Visit (up to Week 8)

Change from Baseline was calculated as the visit value minus the Baseline value. Baseline HCT was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline. A positive change from Baseline indicated HCT concentration increased.

Number of Participants Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Up to Week 8 (Follow-up Visit 2 weeks after last dose)

An adverse event (AE) was defined as any untoward medical occurrence (including a clinically significant abnormal laboratory finding) that occurred in the protocol-specified AE reporting period. A TEAE included medical conditions, signs, and symptoms not previously observed in the participant that emerged during the protocol-specified AE reporting period, including signs or symptoms associated with pre-existing underlying conditions that were not present prior to the AE reporting period. A SAE included AEs that met one or more of the following criteria/outcomes: death, life-threatening, in-patient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, and congenital anomaly/birth defect.

Number of Participants With Change From Baseline in Reticulocyte Count ≥6000, 12000, and 18000 Cells/uL by the End of Dosing PeriodUp to Week 6 (End of The Dosing Period)

Change from Baseline was calculated as the maximum value minus the Baseline value. Baseline reticulocytes count was defined as the average of the last two measurements obtained prior to dosing; if there was only one value prior to dosing, this value served as Baseline.

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