A Study for Monthly Methoxy Polyethylene Glycol-Epoetin Beta Treatment in Patients With Chronic Renal Anaemia

Study Overview
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Brief Summary
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This open-label single-arm study will evaluate the efficacy, safety and tolerability of methoxy polyethylene glycol epoetin beta on long-term maintenance of haemoglobin levels in patients with chronic renal anaemia. Patients will receive methoxy polyethylene glycol-epoetin beta intravenously once monthly at initial doses of either 120 micrograms or 200 micrograms or 360 micrograms in the titration phase of 16 weeks with a potential dose adjustment in the evaluation phase of 8 weeks. The anticipated time on study treatment is 24 weeks. The target sample size is 50-100 patients.

Official Title
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A Single Arm Open Label Interventional Study to Assess the Efficacy, Safety and Tolerability of Once-monthly Administration of Intravenous Methoxy-polyethylene Glycol-epoetin Beta for the Maintenance of Haemoglobin Levels in Dialysis Patients With Chronic Renal Anaemia

Conditions
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Anemia

Intervention / Treatment
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* Drug: methoxy polyethylene glycol-epoetin beta [Mircera]


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adults >/=18 years of age Chronic renal anaemia Haemoglobin concentration between 10 and 12 g/dL at screening Adequate iron status Continuous intravenous maintenance short-acting therapy with same dosing interval for 8 weeks prior to screening Regular long-term haemodialysis therapy for at least 12 weeks prior to screening Exclusion Criteria: Change in haemoglobin concentration >/=2 g/dL during screening Transfusion of red blood cells less than 8 weeks prior to screening Poorly controlled hypertension Relevant acute or chronic bleeding requiring treatment less than 8 weeks prior to screening Active malignant disease Haemolysis Haemoglobinopathies

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Mircera<br> | Drug: methoxy polyethylene glycol-epoetin beta [Mircera]<br>* initial doses of either 120 micrograms or 200 micrograms or 360 micrograms, once monthly<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of Participants Maintaining Average Haemoglobin During the Efficacy Evaluation Period Within the Target Range (10-12 g/dl) | The proportion of participants with their mean haemoglobin (Hb) concentration (g/dL) within the target range during the efficacy evaluation period was assessed. The target range is the reference Hb not >12 g/dL and not < 10 g/dL. | Up to Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events and Serious Adverse Events | An adverse event (AE) was defined as any untoward medical occurrence that occurred during the course of the trial after study treatment had started. An adverse event was therefore any unfavourable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. A Serious adverse event (SAE) is any untoward medical occurrence that at any dose results in death, are life threatening, requires hospitalization or prolongation of hospitalization or results in disability/incapacity, and congenital anomaly/birth defect. The AEs were assessed from baseline to every visit throughout the treatment, post study drug discontinuation, and follow up period. | Up to Week 28 | 
| Mean Change in Haemoglobin Concentration From Screening Period and Efficacy Evaluation Period | The mean haemoglobin (Hb) concentration (g/dL) change from the baseline (Week 0) till efficacy evaluation period (EEP) was assessed and reported. | Up to Week 24 | 
| Proportion of Participants Maintaining Haemoglobin Concentration Within the Haemoglobin Range 10-12g/dL Throughout the Efficacy Evaluation Period | The proportion of participants maintaining haemoglobin concentration within the haemoglobin range 10-12g/dL throughout the efficacy evaluation period (EEP) was assessed and reported. | Up to Week 24 | 
| Mean Time Spent by Participants in the Haemoglobin Range of 10 - 12 g/dL During the Efficacy Evaluation Period | Mean time spent in the haemoglobin range 10 - 12 g/dL during the efficacy evaluation period (EEP) was assessed and reported. | Up to Week 24 | 
| Mean Number of Months Per Participant Requiring Dose Adjustment During the Dose Titration and Evaluation Periods | Mean number of months per participant requiring dose adjustment during the dose titration and evaluation periods was assessed and reported. | Up to Week 24 | 
| Mean Monthly Dose of Methoxy Polyethylene Glycol-epoetin Beta During the Dose Titration and Evaluation Periods | Mean monthly dose of methoxy polyethylene glycol-epoetin beta during the dose titration and evaluation periods was assessed and reported. | Baseline, Week 4, Week 8, Week 12, Week 16, and Week 20 | 
| Number of Participants With Marked Laboratory Abnormalities | A marked laboratory abnormality is defined as above and/or below the normal range of a laboratory parameter which was considered to be potentially clinically relevant. The number of participants with marked laboratory abnormality are presented. Marked laboratory abnormalities were analyzed according to the Roche specified limits for the following reference range: Haemoglobin (Hb) (11.7-17.3 g/dL), Haematocrit (Hct) (35-47%), White blood cells (WBC) (3.6-11.0 10^3/µL), Red blood cells (RBC) (3.8- 5.9 10^6/µL), MCV (80-100 fL) Platelets (150-440 10^3/µL), Iron (37-158 µg/dL), Ferritin (10-365 ng/mL), Transferrin (170-340 mg/dL), TIBC (250-450 µg/dL), TSAT (15-50%), Albumin (3.4-4.8 g/dL), hs-CRP (<= 10.000 mg/dL), Potassium (3.5-5.1 mmol/L), and Phosphorus (2.7-4.5 mg/dL). | Up to Week 28 |