secondary hyperparathyroidism in paediatric subjects with chronic kidney disease MedDRA version: 9.1 Level: LLT Classification code 10020708
Conditions
Interventions
Trade Name: MIMPARA
Pharmaceutical Form: Coated tablet
INN or Proposed INN: Cinacalcet
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 30-
Trade Name: MIMPARA
Phar
Sponsors
ISTITUTO GIANNINA GASLINI
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To be eligible for inclusion into this study, the subjects must fulfill all of the following criteria at study entry: - Parents/guardian written informed consent, and childs assent given before any studyrelated procedure not part of the subjects normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to his or her future medical care. - Age > 2 and 10.49 Kg in males and >9.95 Kg in females, respectively; - Inpatient or outpatient status at the time of enrolment; - Males or females. Female subjects sexually active must be neither pregnant nor breastfeeding, and must lack childbearing potential from screening visit to the end of the safety follow-up, as defined by using an effective form of contraception (i.e. condoms, oral contraceptives or intra-uterine device-IUD). Medically approved contraception could include abstinence. Oral contraception will be accompanied by a barrier method. A negative serum pregnancy test is required prior to initiation of treatment with study medication. - On stable hemodialysis (HD) or peritoneal dialysis (PD) for their CKD for at least one month before entering the 6-month pre-treatment period; - Plasma iPTH levels > 300 pg/mL, AND - Plasma calcium (Ca) levels > 9.4 mg/dL (with normal serum albumin level), AND - Plasma phosphorus (P) levels 60; - Records availability for the following parameters 6 months prior to study entry: demographic information, physical examination, height and dry weight, auxological/anthropometric indices, blood pressure values, Kt/V urea, plasma iPTH, calcium, phosphorus, and alkaline phosphatase levels, blood pH and bicarbonate, serum creatinine/urea, C reactive protein (CRP) levels, liver function tests, blood count, blood 25(OH) vitamin D3 level. 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
Exclusion criteria: To be eligible for inclusion in this study the subjects must not meet any of the following criteria at study entry: - The following laboratory values: Hb2 times the upper limit of normal values) requiring a concomitant corticosteroid or immunosuppressive therapy - History of infectious diseases (including opportunistic infections) within 4 weeks prior to study entry - Evidence as assessed by the Investigator of active or latent bacterial, viral or fungal infections at the time of potential enrollment, including subjects with evidence of Human Immunodeficiency virus (HIV) infection. - Hepatitis-B surface antigen-positive subjects only in subjects who are otherwise eligible for PK/PD assessments - Hepatits C antibody-positive subjects who are also PCR-positive or RIBA positive only in subjects who are otherwise eligible for PK/PD assessments - Use of recombinant human growth hormone therapy - Use of tricyclic antidepressant use - Use of drugs that interact with cinacalcet disposition, such as ketoconazole, itraconazole, telythromycin, voryconazole, ritonavir, rifampin - Previous use of cinacalcet - History of hypersensitivity to any of the ingredients of the study drug. - Use of any investigational drug, device or biologic within 4 weeks prior to study entry or during the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: -To determine the efficacy of cinacalcet HCl plus standard therapy for SHPT in paediatric subjects with CKD on dialysis by assessing the response to treatment (e.g., a reduction from baseline >25% in mean intact parathyroid hormone (iPTH) levels with concomitant values for P<6 mg/dL and Ca between 8.4-10.5 mg/dL or Ca x P product <60) after 6 months compared to standard therapy alone intra-subject; AND - To evaluate the overall safety and tolerability of cinacalcet HCl plus standard therapy for SHPT in paediatric patients with CKD on dialysis over 6 months compared to standard therapy alone intra-subject.;Secondary Objective: -to determine the efficacy of cinacalcet HCl plus standard therapy in the target population by assessing the response to treatment during the induction period over 6 months, and during the maintenance period over 12 months; - To evaluate the overall safety and tolerability of cinacalcet HCl plus standard therapy in the target population over 18 months; - To evaluate the effects of cinacalcet HCl on bone growth by measuring anthropometric indices (Height-for-Age, Weight-for-Age, Weight-for-Height, Body Mass Index-for-Age) and subject growth velocity over 18 months; - In a subset of adolescent subjects (12-18 yrs), to assess the pharmacokinetics (PK)/pharmacodynamic (PD) profile of cinacalcet HCl and its correlation with iPTH target levels and testosterone level when applicable; - In a subset of children (2-11 yrs), to explore the population clinical pharmacology of cinacalcet HCl.;Primary end point(s): The primary study endpoint (EP) will be the proportion of recruited patients who will have a reduction from baseline of at least 25% in mean iPTH levels with concomitant values for plasma P <6 mg/dL and Ca between 8.4 and 10.5 mg/dL or the Ca x P product <60 during the 6-month, intra subject-controlled, efficacy-assessment phase. | — |
Countries
Italy
Outcome results
None listed