An open-label multi-center, multiple dose study to determine the optimum starting dose of intravenous MIRCERA® for maintenance treatment of anemia in pediatric patients with chronic kidney disease on hemodialysis

• Register: EUCTR
• Last refreshed on: 4 July 2016
• Main ID: EUCTR2007-007758-70-FR
• Date of registration: 06/06/2008
• Prospective Registration: Yes
• Primary sponsor: F. Hoffmann-La Roche Ltd
• Public title: An open-label multi-center, multiple dose study to determine the optimum starting dose of intravenous MIRCERA® for maintenance treatment of anemia in pediatric patients with chronic kidney disease on hemodialysis
• Scientific title: An open-label multi-center, multiple dose study to determine the optimum starting dose of intravenous MIRCERA® for maintenance treatment of anemia in pediatric patients with chronic kidney disease on hemodialysis
• Date of first enrolment: 25/08/2008
• Target sample size: 41
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-007758-70
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, France, Germany, Hungary, Italy, Spain

Key inclusion & exclusion 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 <10%); mean of two values measured during screening
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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. Poorly controlled hypertension (e.g. BP >95th percentile for age and sex on two consecutive hemodialysis sessions) despite adequate ultrafiltration
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)


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Anemia associated with chronic kidney disease in pediatric patients (CKD) on hemodialysis
MedDRA version: 9.1 Level: LLT Classification code 10058124 Term: Nephrogenic anemia

Intervention(s)

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 unit: µg/ml microgram(s)/millilitre
Concentration type: equal
Concentration number: 50-

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 unit: µg/ml microgram(s)/millilitre
Concentration type: equal
Concentration number: 100-

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 unit: µg/ml microgram(s)/millilitre
Concentration type: equal
Concentration number: 200-

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 unit: µg/ml microgram(s)/millilitre
Concentration type: equal
Concentration number: 400-

Primary Outcome(s)

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).

Secondary ID(s)

NH19707

2007-007758-70-BE