E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
- Stage 5 chronic kidney disease
- Secondary to hyperparathyroidism |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Male diseases of the urinary and reproductive systems [C12] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064848 |
E.1.2 | Term | Chronic kidney disease |
E.1.2 | System Organ Class | 100000004857 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the safety of paricalcitol capsules for the treatment of secondary hyperparathyroidism in pediatric subjects with Stage 5 CKD, receiving peritoneal dialysis through the evaluation of the incidence of hypercalcemia.
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Subject must be receiving peritoneal dialysis or hemodialysis for at least 3 months prior to Screening
•Male or female subject between 10 and 16 years of age, inclusive at the time of Screening
•Subject is currently being treated for secondary hyperparathyroidism
•For entry into the Dosing Period (for subjects that are naïve to Vitamin D Receptor (VDR) Activators or those who have completed a 2 to 12 week washout), the subject must meet the following laboratory criteria prior to enrollment:
◦A corrected calcium value greater than or equal to 8.4 and less than or equal to 10.2 mg/dL
◦A phosphorus value less than or equal to 6.5 mg/dL
◦An intact parathyroid hormone value greater than 300 pg/mL and less than or equal to 2000 pg/mL |
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E.4 | Principal exclusion criteria |
•Subject is expected or scheduled to receive a living donor kidney transplant within 3 months of Screening or is a kidney transplant patient requiring full immunosuppressant therapy
•Subject is expected to stop peritoneal dialysis or hemodialysis within 4 months of Screening (per investigator discretion)
•Subject is expected to transfer to chronic hemodialysis within 4 months of Screening (per investigator discretion)
•Subject has had a parathyroidectomy within 12 weeks prior to Screening
•Subject has had symptomatic or significant hypocalcemia requiring VDR Activator therapy (i.e., calcitriol, paricalcitol, or doxercalciferol) within 2 months prior to Screening
•Subject is taking maintenance calcitonin, bisphosphonates, glucocorticoids in an equivalent dose of greater than 5 mg prednisone daily, or other drugs known to affect calcium or bone metabolism within 4 to 8 weeks prior to Dosing
•Subject is receiving cinacalcet at the time of Screening
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E.5 End points |
E.5.1 | Primary end point(s) |
Hypercalcemia [ Time Frame: From Day 1 to week 12 ]
Incidence of hypercalcemia (two consecutive, post baseline corrected calcium measures greater than 10.2 mg/dL). This data will be gathered via blood draws |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Time Frame: From Day 1 to week 12 |
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E.5.2 | Secondary end point(s) |
•Intact parathyroid hormone [ Time Frame: From Day 1 to week 12 ]
Proportion of subjects who achieve 2 consecutive intact parathyroid hormone values between 150 to 300 pg/mL (or greater than or equal to 30 percent reduction from baseline in intact parathyroid hormone).
•Changes from baseline in chemistry and hematology lab variables [ Time Frame: During the 12 week dosing period, up to week 12 ]
•Proportion of subjects experiencing adverse events [ Time Frame: From Day 1 to week 12 ]
•Changes from baseline in electrocardiogram (ECG) [ Time Frame: From Day 1 to week 12 ]
•Changes from baseline in vital signs [ Time Frame: From Day 1 to week 12 ] |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 9 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |