MedPath

Roxadustat in the Treatment of Anemia in Chronic Kidney Disease Patients Not Requiring Dialysis

Phase 3
Completed
Conditions
Anemia in Chronic Kidney Disease in Non-dialysis Patients
Interventions
Drug: Placebo
Drug: Roxadustat
Registration Number
NCT01887600
Lead Sponsor
Astellas Pharma Europe B.V.
Brief Summary

This study was conducted to treat anemia in patients with chronic kidney disease. Anemia is a reduced number of red blood cells or hemoglobin. Hemoglobin is important for the transport of oxygen in your blood. The purpose of the study was to see if Roxadustat is both effective and safe as a treatment for anemia in patients with chronic kidney disease.

Detailed Description

The study consisted of three study periods as follows:

* Screening period: up to 6 weeks

* Treatment period: minimum 52 weeks (primary treatment period) up to a maximum of 104 weeks (extended treatment period)

* Post-Treatment Follow-Up period: 4 weeks

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
594
Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboParticipants received matching placebo according to the tiered weight-based approach, with starting doses of 70 mg given thrice weekly (TIW) to participants weighing up to 70 kg and 100 mg given TIW to participants weighing more than 70 kg. Dose-titration was performed based upon regular measurement of Hb levels until participants achieved central Hb value of ≥ 11.0 g/dL and Hb increase from baseline (BL) of ≥ 1.0 g/dL at two consecutive study visits, separated by at least 5 days. Once target Hb level was reached participants entered maintenance period during which roxadustat dosage was adjusted every 4 weeks to maintain participants Hb level within the target range of 10.0 g/dL and 12.0 g/dL. Participants received matching placebo for at least 52 weeks up to a maximum of 104 weeks.
RoxadustatRoxadustatParticipants received roxadustat according to the tiered weight-based approach, with starting doses of 70 mg given thrice weekly (TIW) to participants weighing up to 70 kg and 100 mg given TIW to participants weighing more than 70 kg. Dose-titration was performed based upon regular measurement of Hb levels until participants achieved central Hb value of ≥ 11.0 g/dL and Hb increase from baseline (BL) of ≥ 1.0 g/dL at two consecutive study visits, separated by at least 5 days. Once target Hb level was reached participants entered maintenance period during which roxadustat dosage was adjusted every 4 weeks to maintain participants Hb level within the target range of 10.0 g/dL and 12.0 g/dL. Participants received roxadustat for at least 52 weeks up to a maximum of 104 weeks.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants With a Hemoglobin (Hb) Response to Treatment at Two Consecutive Visits During the First 24 Weeks of Treatment Without Rescue Therapy Prior to Hb ResponseBaseline to week 24

Hemoglobin (Hb) response was measured as Yes or No. Response Yes (responders) was defined as: Hb ≥11.0 g/dL and Hb increase from baseline by ≥ 1.0 g/dL, for participants with baseline Hb \> 8.0 g/dL; or Hb increase from baseline by ≥ 2.0 g/dL, for participants with baseline Hb ≤ 8.0 g/dL at two consecutive visits with available data separated at least 5 days during the first 24 weeks of treatment without having received rescue therapy (red blood cell (RBC) transfusion, erythropoiesis-stimulating agent (ESA), or intravenous (IV) iron prior to Hb response. This was the primary efficacy endpoint for EU (EMA).

Hb Change From Baseline (BL) to the Average Hb in Weeks 28-52 Regardless of Rescue TherapyBaseline and weeks 28 to 52

The change from baseline to the average Hb values across weeks 28 to 52 without having received rescue therapy. The Hb values from visit windows at weeks 28, 32, 36, 40, 44, 48 and 52 were used for the calculation of the average of weeks 28 to 52. This was the primary efficacy endpoint for US (FDA).

Secondary Outcome Measures
NameTimeMethod
Hb Change From BL to the Average Hb in Weeks 28-36 Without Having Received Rescue Therapy Within 6 Weeks Prior to and During 8-Week Evaluation PeriodBaseline and weeks 28 to 36

The Hb values from visit windows at weeks 28, 32 and 36 were used for the calculation of the average of weeks 28 to 36.

Change From BL in Low-Density Lipoprotein (LDL) Cholesterol (Regardless of Fasting Status) to the Average LDL Cholesterol of Weeks 12 to 28Baseline and weeks 12 to 28

Analysis was completed on all values collected on day 1 and weeks 12, 20 and 28.

Time to First Use of Rescue Therapy (Composite of Red Blood Cell (RBC) Transfusions, Erythropoiesis-stimulating Agent (ESA) Use, and Intravenous (IV) Iron)Baseline to week 104 (End of Treatment [EOT])

The time to first use of rescue therapy was calculated (in years) as: (First event date - Analysis date of first dose intake + 1) / 365.25. The First event date was defined as Date of first dose of rescue medication during the efficacy emergent period and Analysis date of first dose intake was defined as date of first study drug dose intake collected on day 1. The Efficacy Emergent Period was defined as the evaluation period from the analysis date of first dose intake up to 7 days after the analysis date of last dose or the end of treatment (EOT) visit, whichever occurred first. Data reported was analysed by Kaplan-Meier estimate for cumulative proportion. Medication onset date was the date of the first use of rescue medication.

Change From BL in Short Form (SF)-36 Vitality (VT) Sub-score to the Average VT Sub-score of Weeks 12 to 28Baseline and weeks 12 to 28

Change from BL in SF-36 VT sub-score to the average value in weeks 12-28 was calculated using the physical component scores (PCS) of SF-36. The multi-purpose, short-form health survey has 36 questions with an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. The survey measures eight dimensions or scales: (1) physical functioning (PF) (10 items); (2) role limitations due to physical health problems (RP) (3 items); (3) bodily pain (BP) (2 items); (4) social functioning (SF) (2 items); (5) general health perceptions (GH) (5 items); (6) role limitations due to emotional problems (RE) (3 items); (7) vitality, energy or fatigue (VT) (4 items); and (8) mental health (MH) (5 items). The SF-36 scores ranged from 0-100 with higher scores indicating better health status.

Average Level of Hb Over Weeks 96 to 104 Without Use of Rescue Therapy Within 6 Weeks Prior to and During the Evaluation PeriodWeeks 96 to 104

All scheduled and unscheduled hemoglobin values from weeks 96 to 104 were taken into account for calculating the average values.

Change From BL in SF-36 Physical Functioning (PF) Sub-score to the Average PF Sub-score of Weeks 12 to 28Baseline and weeks 12 to 28

Change from baseline in SF-36 PF normalized sub-score compared to the average PF sub-score of weeks 12 to 28. The multi-purpose, short-form health survey has 36 questions with an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. Change from baseline in PF sub score of SF-36 to the average of weeks 12-28 was compared by treatment arm for all participants (primary analysis) and in the subsets of participants with baseline PF sub score below 35 and equal or above 35. The SF-36 scores ranged from 0-100 with higher scores indicating better health status. All available SF-36 PF values were used i.e., both scheduled and unscheduled for the calculation of the average PF sub-score of weeks 12 to 28.

Change From BL in Mean Arterial Pressure (MAP) to the Average MAP of Weeks 20 to 28Baseline and weeks 20 to 28

The MAP was derived for each visit from the average systolic (SBP) and diastolic blood pressure (DBP) calculated for each visit using the three readings and the following equation: MAP = (2/3) \* DBP + (1/3) \* SBP. Baseline assessment was the assessment on day 1 (average of the three readings). If the baseline assessment was missing, then the latest available value prior to first drug administration was used.

Time to First Occurrence of HypertensionBaseline and year 0.5, year 1, year 1.5 and year 2

Occurrence of hypertension was defined as SBP increase from BL ≥20 mmHg and SBP \>170 mmHg or DBP increase from BL ≥15 mmHg and DBP ≥110 mmHg. Time to first occurrence of hypertension was defined as first date where SBP criterion or DBP criterion is met, whichever occurred first. Data was analysed using Kaplan-Meier estimate for cumulative proportion.

Rate of Progression of CKD Measured by Annualized Estimated Glomerular Filtration Rate (eGFR) Slope Over TimeBaseline to week 108

Annualized eGFR slope over time was estimated by a random slopes and intercepts model using all available eGFR values (one baseline and all post-treatment values up to EOT period or start of dialysis adjusted on baseline Hb, region, CV history at baseline and the interaction terms (baseline eGFR by timepoint and baseline Hb by timepoint). All assessments collected after initiation of chronic dialysis (acute or chronic) are excluded from the analysis. Baseline assessment was the assessment from day 1 visit. If this value was missing, the value from screening visit was used.

Average Level of Hb Over Weeks 28 to 36 Without Use of Rescue Therapy Within 6 Weeks Prior to and During the Evaluation PeriodWeeks 28 to 36

All scheduled and unscheduled hemoglobin values from weeks 28 to 36 were taken into account for calculating the average values.

Average Level of Hb Over Weeks 44 to 52 Without Use of Rescue Therapy Within 6 Weeks Prior to and During the Evaluation PeriodWeeks 44 to 52

All scheduled and unscheduled hemoglobin values from weeks 44 to 52 were taken into account for calculating the average values.

Time to Achieve the First Hb Response Without Rescue Therapy, as Defined by Primary EndpointBaseline to week 24

Hb response was measured as Yes or No; Yes was defined as Hb ≥11.0 g/dL and Hb increase from baseline by ≥ 1.0 g/dL, for participants with baseline Hb \> 8.0 g/dL; or Hb increase from baseline by ≥ 2.0 g/dL, for participants with baseline Hb ≤ 8.0 g/dL. For a participant without rescue therapy before Hb response (defined in 1 primary outcome), the time to achieve Hb response was calculated (in weeks) as: (First event date - Analysis date of first dose intake + 1) / 7 where First event date was defined as First date of both values that met the criteria for response. Participants who discontinued or received rescue therapy prior to the first Hb response or before the second consecutive Hb value defined as a response were classified as non responders and were censored at week 24 or end of efficacy emergent period, whichever came first. Data analysis was completed using Kaplan-Meier estimate for cumulative proportion.

Hb Change From BL to Each Post-Dosing Time PointBaseline (day 1) and weeks 1, 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, 28, 32, 36, 40, 44, 48, 52, 56, 60, 64, 68, 72, 76, 80, 84, 88, 92, 96, 100, 104

All scheduled and unscheduled hemoglobin values that belong to each visit window were taken into account using one value per analysis window. Baseline Hb was defined as the mean of four latest central laboratory Hb values prior or on the same date as first study drug intake (pre-dose).

Hb Change From BL to the Average Hb Value of Weeks 28-36 Regardless of the Use of Rescue TherapyBaseline and weeks 28 to 36

The Hb values from visit windows from weeks 28 to 36 were used for the calculation of the average regardless of rescue therapy. Baseline Hb was defined as the mean of four latest central laboratory Hb values prior or on the same date as first study drug intake (pre-dosing).

Hb Change From BL to the Average Hb Value of Weeks 44-52 Regardless of the Use of Rescue TherapyBaseline and weeks 44 to 52

The Hb values from visit windows from weeks 44 to 52 were used for the calculation of the average regardless of rescue therapy. Baseline Hb was defined as the mean of four latest central laboratory Hb values prior or on the same date as first study drug intake (pre-dosing).

Hb Change From BL to the Average Hb Value of Weeks 96-104 Regardless of the Use of Rescue TherapyBaseline and weeks 96 to 104

The Hb values from visit windows from weeks 96 to 104 were used for the calculation of the average regardless of rescue therapy. Baseline Hb was defined as the mean of four latest central laboratory Hb values prior or on the same date as first study drug intake (pre-dosing).

Percentage of Hb Values Within 10.0-12.0 g/dL in Weeks 28-36 Without Use of Rescue TherapyBaseline and weeks 28 to 36

The percentage of Hb values measured during weeks 28-36 within 10.0 -12.0 g/dL, without having received rescue therapy within 6 weeks prior to and during the 8-week evaluation period is reported.

Percentage of Hb Values Within 10.0-12.0 g/dL in Weeks 44-52 Without Use of Rescue TherapyBaseline and weeks 44 to 52

The percentage of Hb values measured during weeks 44-52 with values within 10.0 - 12.0 g/dL, without having received rescue therapy within 6 weeks prior to and during the 8-week evaluation period is reported.

Percentage of Hb Values Within 10.0-12.0 g/dL in Weeks 96-104 Without Use of Rescue TherapyBaseline and weeks 96 to 104

The percentage of Hb values measured during weeks 96-104 within 10.0 -12.0 g/dL, without having received rescue therapy within 6 weeks prior to and during the 8-week evaluation period is reported.

Time to First HospitalizationBaseline to week 104

Time to first hospitalization was defined in years as the First event date during the Efficacy Emergent Period - (Analysis date of first dose intake +1)/365.25. The first event date was defined as the Date of first admission. The Efficacy Emergent Period was defined as the evaluation period from the analysis date of first dose intake up to 7 days after the analysis date of last dose or EOT visit, whichever occured first. Analysis date of first dose intake was defined as the date of first study drug intake collected on day 1 visit. For participants who experienced more than one hospitalization, only their first event following study treatment was used. Data analysis was completed using Kaplan-Meier estimate for cumulative proportion.

Number of Days of Hospitalization Per Patient Exposure Year (PEY)Baseline to week 104

The sum of the durations of all hospitalizations in days was adjusted for the duration of exposure. Derived only for participants with at least one hospitalization. The number of days of hospitalization per PEY was calculated as the sum of the durations of all hospitalizations in days \[Minimum (Date of discharge, End of Efficacy Emergent Period) - Date of admission + 1\] / \[Duration of Efficacy Emergent Period in days / 365.25\]. Participants can have more than one hospitalization.

Time to First Use of Rescue Therapy (Composite of RBC, Transfusions, ESA Use, and IV Iron) in the First 24 Weeks of TreatmentBaseline to week 24

Time to first use of rescue therapy in years. Data analysis was completed using Kaplan-Meier estimate for cumulative proportion.

Time to First Use of RBC TransfusionsBaseline to week 104

Time to First Use of RBC Transfusions during efficacy emergent period. For participants who have experienced more than one RBC transfusion, only their first event following study treatment was used. The efficacy emergent period was defined as the evaluation period from the analysis date of first dose intake up to 7 days after the analysis date of last dose or EOT visit, whichever occured first. Data analysis was completed using Kaplan-Meier estimate for cumulative proportion.

Mean Monthly Number of RBC PacksBaseline to week 104

During efficacy emergent period, the mean monthly number of RBC packs was calculated as the sum of units transfused between the first dose and up to the last dose in the period divided by duration of efficacy emergent period (in days) divided by 28 days. The efficacy emergent period was defined as the evaluation period from the analysis date of first dose intake up to 7 days after the analysis date of last dose or EOT visit, whichever occured first. In case of missing number of packs, values were estimated based on 1 unit for packed cells = 250 mL or 1 unit for whole blood = 500 mL. Participants without RBC transfusion were included with a value of zero. No estimation if values were missing.

Mean Monthly Volume of Blood TransfusedBaseline to week 104

During efficacy emergent period, the mean monthly volume of blood transfused was calculated as the sum of blood volume transfused between the first dose and up to the last dose in the period divided by duration of efficacy emergent period (in days) divided by 28 days. The efficacy emergent period was defined as the evaluation period from the analysis date of first dose intake up to 7 days after the analysis date of last dose or EOT visit, whichever occurred first. The mean monthly volume transfused was calculated as the sum of the volume transfused between the first dose and up to the last dose in the period divided by duration (in days) and multiplied by 28 days. Participants without RBC transfusion were included with a value of zero.

Time to First Use of ESA Rescue TherapyBaseline to week 104

Time to First Use of ESA Rescue Therapy during efficacy emergent period. For participants with use of ESA, the time to first use of ESA was calculated as (First event date - Analysis date of first dose intake + 1) / 365.25. The efficacy emergent period was defined as the evaluation period from the analysis date of first dose intake up to 7 days after the analysis date of last dose or EOT visit, whichever occured first. Data analysis was completed using Kaplan-Meier estimate for cumulative proportion. Participants without ESA rescue were censored at the end of treatment.

Time to First Use of IV IronBaseline to week 104

Time to first use of IV iron during efficacy emergent period in years. For participants with use of IV iron, the time to first use of IV iron was calculated as (First event date - Analysis date of first dose intake + 1) / 365.25. The efficacy emergent period was defined as the evaluation period from the analysis date of first dose intake up to 7 days after the analysis date of last dose or EOT visit, whichever occurred first.Data analysis was completed using Kaplan-Meier estimate for cumulative proportion.

Change From BL to Each Post-Dosing Visit in Total CholesterolBaseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 68, 84, 104

Change from baseline to each planned assessment for total cholesterol is reported. Baseline was defined as the value on day 1. If the value was missing, the latest value prior to first study drug administration was used.

Change From BL to Each Post-Dosing Visit in Low Density Lipoprotein (LDL)/High-Density Lipoprotein (HDL) RatioBaseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 68, 84, 104

Change from baseline to each planned assessment for LDL/HDL ratio is reported. Baseline was defined as the value on Day 1. If this value was missing, the latest value prior to first study drug administration was used.

Percentage of Participants Who Have Achieved Antihypertensive Treatment Goal in CKD Participants Over Weeks 12-28Weeks 12 to 28

Occurrence of achieved antihypertensive treatment goal was defined as the average SBP \< 130 mmHg and the average DBP \< 80 mmHg over the period of weeks 12-28. Participants without any blood pressure measurement were excluded.

Change From BL to the Average Value of Weeks 12-28 in Quality of Life (QoL) SF-36 Physical Component Score (PCS)Baseline and weeks 12 to 28

The 36-Item short-form health survey (SF-36) is a multi-purpose survey with 36 questions. It provides an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. For each scale scores range from 0-100. The physical component score was calculated based on the results of the SF-36 scores. Higher scores indicate better health status.

Change From BL to the Average Value of Weeks 12-28 in Anemia Subscale (Ans) of Functional Assessment of Cancer Therapy (FACT-An) ScoreBaseline and weeks 12 to 28

Baseline FACT-An AnS was defined as the FACT-An AnS value on Day 1. Together with the Functional Assessment of Cancer Therapy - General (FACT-G), the Anemia Subscale (AnS) is referred to as the FACT-An Total. The AnS scale contains 13 fatigue specific items (the Fatigue Score) plus 7 items related to anemia. The Anemia AnS score range is 0 to 80. For the above score, a higher score indicates better QoL.

Change From BL to the Average Value of Weeks 12-28 in Total FACT-An ScoreBaseline and weeks 12 to 28

Baseline FACT-An Total Score was defined as the FACT-An Total score on Day 1. Total Fact-An score is composed of FACT-G and Ans scales. FACT-G contains 27 items that cover four dimensions of well-being: physical (PWB) - 7 items, functional (FWB) - 7 items, social/family (SWB) - 7 items, and emotional (EWB) - 6 items. The AnS scale contains 13 fatigue specific items (the Fatigue Score) plus 7 items related to anemia. The total score is obtained by summation of the scores from PWB, SWB, EWB, FWB and AnS. The FACT-An Total Score scale range is 0-188. A higher score indicates better QoL.

Change From BL to Each Study Visit in Serum Creatinine (Cr) RatioBaseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 60, 68, 76, 84, 92, 100, 104

Baseline assessment was the assessment from day 1 visit. If baseline value was missing, the value from screening visit was used. In case of missing data, no imputation rules were applied. To compute the geometric mean of serum Cr (ratio) and associated 95% CI, the mean of log-transformed serum Cr (ratio) values and associated 95% CI are back-transformed to the raw scale. All assessments collected after initiation of dialysis (acute or chronic) were excluded from the analysis.

Change From BL to the Average Value of Weeks 12-28 in the Euroqol Questionnaire - 5 Dimensions 5 Levels (EQ-5D 5L) Visual Analogue Scale (VAS) ScoreBaseline and weeks 12 to 28

The Euroqol Questionnaire - 5 Dimensions 5 Levels (EQ-5D 5L) is a self-reported questionnaire. The EQ-5D 5L is used as a measure of respondents' Health Related Quality of Life (HRQoL). The EQ-5D 5L consists of the EQ-5D descriptive system and the EQ visual analogue scale (VAS). The EQ-5D 5L descriptive system comprises of 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, extreme problems. The VAS records the respondent's self-rated health status on a graduated (0-100) scale, where the answers are labeled 'Best imaginable health state' and 'Worst imaginable health state' with higher scores for higher HRQoL.

Change From BL to the Average Value of Weeks 12-28 in Overall Work Impairment Due to Anaemic SymptomsBaseline and weeks 12 to 28

Work productivity and activity impairment: anemic symptoms (WPAI:ANS) questionnaire version 2 was used to measure work and activity impairment during the last seven days due to anemia. It is self-assessed questionnaire which consists of 6 questions covering work and daily activities. Questions include asking if participant is working, how many hours the person missed work due to anemic symptoms, how many hours the person missed work due to other reasons, how many hours participant actually worked and how the anemic symptoms impacted their productivity and ability to do daily activities. For the last 2 questions, they were scored from 0-10 with 0 identifying no effect on work and 10 completely prevented from working. Overall work impairment due to ANS was calculated as 100 x Q2/(Q2+Q4)+\[(1-Q2/(Q2+Q4))x(Q5/10)\]. Scores were calculated with the formula to derive the overall work impairment on each timepoints in percentage, and then changes of the percentage from baseline are reported.

Percentage of Participants in Each Category in Patients' Global Impression of Change (PGIC)Week 12 to 28

The Patients' Global Impression of Change (PGIC) is a participant-rated instrument that measures change in participants overall status on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse).

Change From BL to Each Post-Dosing Visit in Non-HDL CholesterolBaseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 68, 84, 104

Change from baseline to each planned assessment for non-HDL is reported. Baseline was defined as the value on day 1. If this value was missing, the latest value prior to first study drug administration was used.

Change From BL to Each Post-Dosing Visit in Apolipoproteins A1 (ApoA1)Baseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 68, 84, 104

Change from baseline to each planned assessment for apolipoproteins A1 is reported. Baseline was defined as the value on day 1. If this value was missing, the latest value prior to first study drug administration was used.

Change From BL to Each Post-Dosing Visit in Apolipoproteins B (ApoB)Baseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 68, 84, 104

Change from baseline to each planned assessment for apolipoproteins B is reported. Baseline was defined as the value on day 1. If this value was missing, the latest value prior to first study drug administration was used.

Change From BL to Each Post-Dosing Visit in Ratio ApoB/ApoA1Baseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 68, 84, 104

Change from baseline to each planned assessment for ratio of ApoB/ApoA1 is reported. Baseline was defined as the value on day 1. If this value was missing, the latest value prior to first study drug administration was used.

Percentage of Participants With Mean LDL Cholesterol <100 mg/dL Calculated Over Weeks 12 to 28Weeks 12 to 28

Mean LDL cholesterol \<100 mg/dL over weeks 12 to 28 was defined as a binary variable (Yes/No), where Yes was defined as mean LDL cholesterol \<100 mg/dL over weeks 12 to 28. Participants without any LDL value within this duration were excluded.

Change From BL to Each Study Visit in Serum HepcidinBaseline and weeks 4,12,20,36,52,104

Baseline was assessed on Day 1 visit. If baseline value was missing, the value from screening visit was used. In case of missing data, no imputation rules were applied.

Change From BL to Each Study Visit in Serum Transferrin Saturation (TSAT)Baseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 60, 68, 76, 84, 92, 100, 104

Baseline was assessed on Day 1 visit. If baseline value was missing, the value from screening visit was used. In case of missing data, no imputation rules were applied.

Change From BL to Each Study Visit in Albumin/Creatinine Ratio in UrineBaseline and weeks 12, 24, 36, 52, 64, 76, 88, 104

Baseline assessment was the assessment from day 1 visit. If baseline value was missing, value from screening visit was used. In case of missing data, no imputation rules were applied. To compute the geometric mean of albumin/creatinine ratio in urine and associated 95% CI, the mean of log-transformed albumin/creatinine ratio in urine values (ratio) and associated 95% CI are back-transformed to the raw scale. All assessments collected after initiation of dialysis (acute or chronic) were excluded from the analysis.

Time to CKD Progression (Composite of Doubling Serum Creatinine, Chronic Dialysis or Renal Transplant, and Death)Baseline and year 0.5, year 1, year 1.5 and year 2

CKD progression was defined as date of occurrence of chronic dialysis or date of renal transplant or doubled serum creatinine or date of death, whichever came first. The time to CKD progression was calculated (in years) as (First event date - Analysis date of first dose intake + 1) / 365.25. First event date was defined as first occurrence of serum creatinine being doubled compared with baseline, first occurrence of chronic dialysis or renal transplant, occurrence of participants who died (whichever occurred first). Analysis date of first dose intake was defined as date of first study drug dose intake collected on day 1 visit. Data analysis was completed using Kaplan-Meier estimate for cumulative proportion. The data evaluated until the safety emergent period, which was defined as the evaluation period from analysis date of first drug intake up to 28 days after the analysis last dose, and results were presented for every 6 months up to 2 years.

Time to at Least 40% Decrease in eGFR From Baseline, Chronic Dialysis or Renal TransplantBaseline and year 0.5, year 1, year 1.5 and year 2

All eGFR values collected during the safety emergent period are considered, excluding those collected on or after initiation of dialysis (acute or chronic). The First event date was defined as First occurrence of 40% decrease in eGFR from baseline, first occurrence of chronic dialysis or renal transplant (whichever occurred first and Analysis date of first dose intake was defined as date of first study drug dose intake collected on day 1 visit. The safety emergent period was defined as the evaluation period from the analysis date of first drug intake up to 28 days after the analysis date of last dose or end of study (EOS), whichever occurred first. Data analysis was completed using Kaplan-Meier estimate for cumulative proportion. The data evaluated until the safety emergent period, which was defined as the evaluation period from analysis date of first drug intake up to 28 days after the analysis last dose, and results were presented for every 6 months up to 2 years.

Change From BL to Each Study Visit in Serum FerritinBaseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 60, 68, 76, 84, 92, 100, 104

Baseline assessment was assessment from Day 1 visit. If baseline value was missing, the value from screening visit was used. In case of missing data, no imputation rules were applied.

Change From BL to Each Study Visit in Serum HbA1c LevelBaseline and weeks 12, 28, 36, 44, 60, 84, 104

HbA1c was measured at each timepoint and presented in 'fraction of 1' unit by dividing the values in percentage by 100, in order to fit for CDISC (Clinical Data Interchange Standards Consortium) standard terminology. Changes from baseline to each timepoint were reported in unit 'fraction of 1'. Baseline was assessed on Day 1 visit. If baseline value was missing, the value from screening visit was used. In case of missing data, no imputation rules were applied.

Change From BL to Each Study Visit in Fasting Blood GlucoseBaseline and weeks 4, 8, 12, 20, 28, 36, 44, 52, 60, 68, 76, 84, 92, 100, 104

Baseline was assessed on Day 1 visit. If baseline value was missing, the value from screening visit was used. In case of missing data, no imputation rules were applied.

Time to Doubling of Serum Creatinine or Chronic Dialysis or Renal Transplant Compared to BaselineBaseline and year 0.5, year 1, year 1.5 and year 2

The endpoint was defined as time to doubling serum creatinine or chronic dialysis or renal transplant what ever came first. Time to event was defined as (First event date - Analysis date of first dose intake + 1) / 365.25. First event date was defined as date of dialysis or date of renal transplant (whichever occurred first) and analysis date of first dose intake was defined as date of first study drug dose intake collected on day 1 visit. Data analysis was completed using Kaplan-Meier estimate for cumulative proportion. The data evaluated until the safety emergent period, which was defined as the evaluation period from analysis date of first drug intake up to 28 days after the analysis last dose, and results were presented for every 6 months up to 2 years.

Trial Locations

Locations (138)

Site ES34011

🇪🇸

Galdakao, Vizcaya, Spain

Site ES34003

🇪🇸

Barcelona, Spain

Site UA38015

🇺🇦

Kherson, Ukraine

Site UA38019

🇺🇦

Odessa, Ukraine

Site RS38104

🇷🇸

Belgrade, Serbia

Site BE32004

🇧🇪

Brussels, Flemish Brabant, Belgium

Site TR90016

🇹🇷

Edirne, Turkey

Site BY37505

🇧🇾

Minsk, Belarus

Site RO40005

🇷🇴

Timisoara, Timis, Romania

Site PL48001

🇵🇱

Krakow, Malopolska, Poland

Site PL48057

🇵🇱

Nowy Tomyśl, Poland

Site PL48005

🇵🇱

Warszawa, Poland

Site PL48006

🇵🇱

Wroclaw, Poland

Site IT39036

🇮🇹

Pavia, Italy

Site RU70004

🇷🇺

Omsk, Russian Federation

Site PA50703

🇵🇦

Ciudad de Colon, Panama

Site PL48007

🇵🇱

Tarnow, Poland

Site PL48004

🇵🇱

Warszawa, Poland

Site ES34007

🇪🇸

Ciudad Real, Spain

Site RU70048

🇷🇺

Moscow, Russian Federation

Site RU70047

🇷🇺

Moscow, Russian Federation

Site RU70024

🇷🇺

Chelyabinsk, Russian Federation

Site UA38021

🇺🇦

Cherkasy, Ukraine

Site RU70003

🇷🇺

Nizhny Novgorod, Russian Federation

Site ZA27006

🇿🇦

Parow, South Africa

Site ES34002

🇪🇸

Badalona-Barcelona, Spain

Site PE51002

🇵🇪

Trujillo, Peru

Site RS38117

🇷🇸

Krusevac, Serbia

Site PL48012

🇵🇱

Lodz, Poland

Site RU70005

🇷🇺

Moscow, Russian Federation

Site RU70002

🇷🇺

Saint Petersburg, Russian Federation

Site RU70011

🇷🇺

Saint Petersburg, Russian Federation

Site RU70045

🇷🇺

Saint-Petersburg, Russian Federation

Site UA38006

🇺🇦

Dnepropetrovsk, Ukraine

Site UA38011

🇺🇦

Kharkiv, Ukraine

Site UA38012

🇺🇦

Kyiv, Ukraine

Site TR90020

🇹🇷

Malatya, Turkey

Site RU70014

🇷🇺

Rostov-on-don, Russian Federation

Site UA38016

🇺🇦

Ivano-Frankivsk, Ukraine

Site UA38018

🇺🇦

Uzhgorod, Ukraine

Site ES34006

🇪🇸

Barcelona, Spain

Site TR90003

🇹🇷

Ankara, Turkey

Site RU70057

🇷🇺

Yaroslavl, Russian Federation

Site GB44001

🇬🇧

Swansea, United Kingdom

Site UA38010

🇺🇦

Kyiv, Ukraine

Site UA38007

🇺🇦

Mykolaiv, Ukraine

Site UA38009

🇺🇦

Lviv, Lvivska, Ukraine

Site UA38017

🇺🇦

Kyiv, Ukraine

Site UA38008

🇺🇦

Odessa, Ukraine

Site BE32013

🇧🇪

Liege, Belgium

Site CO57008

🇨🇴

Barranquilla, Colombia

Site PE51001

🇵🇪

Iquitos, Peru

Site BY37506

🇧🇾

Minsk, Belarus

Site GT50202

🇬🇹

Ciudad de Guatemala, Guatemala

Site GT50207

🇬🇹

Ciudad de Guatemala, Guatemala

Site BY37503

🇧🇾

Brest, Belarus

Site BY37507

🇧🇾

Minsk, Belarus

Site GE99504

🇬🇪

Tbilisi, Georgia

Site GT50208

🇬🇹

Ciudad de Guatemala, Guatemala

Site GT50201

🇬🇹

Ciudad de Guatemala, Guatemala

Site PA50701

🇵🇦

Ciudad de Panama, Panama

Site RO40012

🇷🇴

Bucuresti, Romania

Site ZA27004

🇿🇦

Bloemfontein, Free State, South Africa

Site DO17104

🇩🇴

Santo Domingo, Dominican Republic

Site RS38101

🇷🇸

Nis, Serbia

Site RS38105

🇷🇸

Belgrade, Serbia

Site GB44005

🇬🇧

Welwyn Garden City, United Kingdom

Site RS38103

🇷🇸

Belgrade, Serbia

Site BY37504

🇧🇾

Gomel, Belarus

Site BY37501

🇧🇾

Grodno, Belarus

Site BY37502

🇧🇾

Vitebsk, Belarus

Site BE32002

🇧🇪

Antwerp, Belgium

Site BE32017

🇧🇪

Bonheiden, Belgium

Site BE32012

🇧🇪

Baudour, Belgium

Site BE32014

🇧🇪

Ieper, Belgium

Site BG35923

🇧🇬

Pazardjik, Bulgaria

Site BG35906

🇧🇬

Sofia, Bulgaria

Site BG35908

🇧🇬

Sofia, Bulgaria

Site BG35910

🇧🇬

Sofia, Bulgaria

Site BG35903

🇧🇬

Veliko Tarnovo, Bulgaria

Site BG35907

🇧🇬

Stara Zagora, Bulgaria

Site BG35916

🇧🇬

Varna, Bulgaria

Site CO57002

🇨🇴

Pereira, Colombia

Site DO17101

🇩🇴

La Fe Santo Domingo, Dominican Republic

Site CO57007

🇨🇴

Barranquilla, Colombia

Site DO17102

🇩🇴

Santiago de los Caballeros, Dominican Republic

Site GR30001

🇬🇷

Thessaloniki, Greece

Site GE99503

🇬🇪

Tbilisi, Georgia

Site GE99502

🇬🇪

Tbilisi, Georgia

Site GR30003

🇬🇷

Heraklion, Greece

Site EE37201

🇪🇪

Tallinn, Estonia

Site DO17103

🇩🇴

Santo Domingo, Dominican Republic

Site GR30002

🇬🇷

Patras, Greece

Site GT50209

🇬🇹

Chiquimula, Guatemala

Site GT50206

🇬🇹

Guatemala, Guatemala

Site HU36029

🇭🇺

Budapest, Hungary

Site GT50205

🇬🇹

Guatemala, Guatemala

Site GT50203

🇬🇹

Guatemala, Guatemala

Site HU36008

🇭🇺

Pecs, Hungary

Site HU36025

🇭🇺

Gyor, Hungary

Site HU36027

🇭🇺

Kistarcsa, Hungary

Site HU36028

🇭🇺

Szent, Hungary

Site HU36026

🇭🇺

Kaposvar, Hungary

Site HU36003

🇭🇺

Zalsaegerszeg, Hungary

Site IT39001

🇮🇹

Bari, Italy

Site IT39008

🇮🇹

Lecco, Italy

Site IT39006

🇮🇹

Milano, Italy

Site IT39037

🇮🇹

Modena, Italy

Site PL48008

🇵🇱

Lodz, Poland

Site PL48013

🇵🇱

Szczecin, Poland

Site RO40001

🇷🇴

Brasov, Romania

Site PL48014

🇵🇱

Zamosc, Poland

Site RO40021

🇷🇴

Bucharest, Romania

Site RO40004

🇷🇴

Oradea, Romania

Site RO40003

🇷🇴

Bucuresti, Romania

Site RU70054

🇷🇺

Irkutsk, Russian Federation

Site RU70008

🇷🇺

Kaluga, Russian Federation

Site RU70007

🇷🇺

Moscow, Russian Federation

Site RU70051

🇷🇺

Moscow, Russian Federation

Site RU70043

🇷🇺

Petrozavodsk, Russian Federation

Site RU70022

🇷🇺

Saint Petersburg, Russian Federation

Site RU70060

🇷🇺

Saratov, Russian Federation

Site RU70006

🇷🇺

Smolensk, Russian Federation

Site RS38102

🇷🇸

Belgrade, Serbia

Site RU70001

🇷🇺

Yaroslavl, Russian Federation

Site RS38116

🇷🇸

Zrenjanin, Serbia

Site ZA27001

🇿🇦

Observatory, Cape Town, South Africa

Site ZA27002

🇿🇦

Durban, Kwa Zulu Natal, South Africa

Site ZA27008

🇿🇦

Durban, South Africa

Site ZA27007

🇿🇦

Port Elizabeth, South Africa

Site ES34010

🇪🇸

Alcorcon, Madrid, Spain

Site UA38003

🇺🇦

Chernivtsi, Ukraine

Site TR90024

🇹🇷

Kocaeli, Turkey

Site UA38002

🇺🇦

Zaporizhzhya, Ukraine

Site UA38001

🇺🇦

Ternopil, Ukraine

Site GB44004

🇬🇧

Westcliff-on-Sea, United Kingdom

Site GB44008

🇬🇧

Cambridge, United Kingdom

Site PL48002

🇵🇱

Katowice, Poland

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