Anemia associated with chronic kidney disease in pediatric patients (CKD) on hemodialysis MedDRA version: 14.1 Level: LLT Classification code 10058124 Term: Nephrogenic anemia System Organ Class: 100000004851
Conditions
Interventions
Trade Name: MIRCERA®
Pharmaceutical Form: Solution for injection
INN or Proposed INN: methoxy polyethylene glycol-epoetin beta
CAS Number: 677324-53-7
Current Sponsor code: RO0503821
Concentration uni
Sponsors
F. Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Written informed consent 2. Pediatric patients 5 -17 years old with clinically stable chronic renal anemia 3. Hemodialysis treatment for at least 8 weeks 4. Body weight = 10 kg 5. Adequate hemodialysis: URR of > 65% or Kt/V >1.2 for patients on thrice weekly HD. Patients with fewer or with more HD sessions per week should have a weekly Kt/V = 3.6 6. Baseline pre-dialysis Hb concentration 10.0 – 12.0 g/dL determined from the mean of weekly Hb values measured between weeks -2 to -1 7. Intravenous maintenance epoetin alfa, epoetin beta, or darbepoetin alfa with same dosing interval for at least 8 weeks before screening 8. Stable maintenance epoetin alfa, epoetin beta, or darbepoetin alfa treatment with no weekly dose change = 25% (increase or decrease) during the 2-weeks of screening. Patients who had been previously treated by the sc route could only participate if they have been receiving their ESA by the iv route for at least 8 weeks before screening. 9. Adequate iron status defined as serum ferritin = 100 ng/mL or TSAT = 20% (or percentage of hypochromic red cells =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Overt gastrointestinal bleeding within 8 weeks before screening or during the screening period 2. RBC transfusions within 8 weeks before screening or during the screening period 3. Hemoglobinopathies (e.g., homozygous sickle-cell disease, thalassemia of all types) 4. Hemolysis 5. Active malignant disease 6. Chronic, uncontrolled or symptomatic inflammatory disease (e.g. systemic lupus erythematosus) 7. Uncontrolled hypertension as assessed by the investigator 8. Epileptic seizures within 3 months prior to screening and during the screening period 9. Administration of any investigational drug within 4 weeks prior to screening and planned during the study 10. Severe hyperparathyroidism (Intact PTH = 1000 pg/ml or whole PTH = 500 pg/ml) or biopsy-proven bone marrow fibrosis 11. Known hypersensitivity to recombinant human erythropoietin, polyethylene glycol, or to any constituent of the study drug formulation 12. Pure red cell aplasia (PRCA) or history of PRCA 13. High likelihood of early withdrawal or interruption of the study (e.g. planned living donor kidney transplant within 16 weeks after randomization) 14. Planned elective surgery during the entire study period (except hemodialysis access surgery)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - To determine the starting dose of MIRCERA® in pediatric patients with CKD on hemodialysis when switching from stable maintenance treatment with epoetin alfa, epoetin beta or darbepoetin alfa - To demonstrate changes in Hb over time in response to different iv doses of MIRCERA®;Secondary Objective: - To study the pharmacokinetics (PK) of MIRCERA® in pediatric patients - To explore MIRCERA® exposure response relationship - To assess the safety and tolerability of multiple doses of MIRCERA® in pediatric patients - To document long-term safety and efficacy of MIRCERA® administration in pediatric patients with anemia associated with CKD;Primary end point(s): The primary endpoint in this study will be the change in Hb concentration (g/dL) between the baseline and evaluation periods. The different MIRCERA® dose groups will be compared in an exploratory manner, using summary statistics based on means, standard deviations and percentiles. The analysis will be performed for all patients of the dose group combined as well as stratified by age category (=5 to <12 years versus =12 years).;Timepoint(s) of evaluation of this end point: Weeks 17-20 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Incidence of RBC transfusions, reticulocyte counts, AEs, laboratory parameters. 2. Number of patients with average Hb concentration above, below or within range of 10-12g/dL during evaluation period ;Timepoint(s) of evaluation of this end point: 1. Throughout study 2. Weeks 17-20 | — |
Countries
Australia, Belgium, France, Germany, Hungary, Italy, Poland, Spain, Thailand
Contacts
Public ContactTrial Information Support Line-TISL
F. Hoffmann-La Roche Ltd
Outcome results
None listed