Phase 3, Randomized, Open-label, Controlled, Multiple-Dose, Efficacy, Safety, Pharmacokinetic, and Pharmacodynamic Study of Etelcalcetide in Pediatric Subjects 28 days to < 18 Years of age With Secondary Hyperparathyroidism and Chronic Kidney Disease Receiving Maintenance Hemodialysis
Study ID: STU-2019-0502
This is a multi-center, phase 3, randomized, open-label, controlled, multi-dose, titration study to assess the efficacy, safety, PK, and pharmacodynamics (PD) of etelcalcetide in the treatment of SHPT in pediatric subjects between 28 days and [Less Than] 18 years of age, with CKD on hemodialysis. approximately 56 subjects will be randomized in a 3:1 ratio (42 subjects will receive etelcalcetide in addition to standard of care [etelcalcetide arm] and 14 subjects will receive standard of care alone [control arm]) in the study. it is planned that at least 8 subjects [Less Than] 6 years of age will be enrolled. Part 1 of the study allows for enrollment of subjects 2 to [Less Than] 18 years of age and Part 2 allows for enrollment of subjects 28 days to [Less Than] 2 years of age. etelcalcetide must be determined to be reasonably well tolerated (appendix 3) in the first 4 etelcalcetide-treated subjects in Part 1 (subjects 2 to [Less Than] 18 years of age) with at least 2 subjects 2 to [Less Than] 6 years of age, before enrollment in Part 2 will be opened to subjects 28 days to [Less Than] 2 years of age.
To evaluate the efficacy of etelcalcetide in reducing the intact parathyroid hormone level (iPTH) by [GreaterThanorequalTo] 30% in children ages 28 days to [Less Than] 18 years with secondaryhyperparathyroidism (SHPT) receiving maintenance hemodialysis