The overall objective of this study is to evaluate the pharmacokinetics (PK), efficacy, and safety of encaleret in pediatric participants from birth to 17 years of age with ADH1.
Provide written informed consent (if legally permitted), or have written informed consent from a parent/legal guardian and provide assent (where required and as appropriate per local requirements)
Have a documented pathogenic or likely pathogenic activating variant, or variant of uncertain significance of the calcium-sensing receptor (CASR), associated with biochemical findings of hypoparathyroidism at screening or a documented history of hypoparathyroidism as manifested by hypocalcemia and intact parathyroid hormone (PTH) <40 picogram per milliliter (pg/mL) (4.2 picomoles per liter [pmol/L])
Have at least 1 symptom or sign of hypoparathyroidism at screening or a documented history of symptoms or signs of hypoparathyroidism
Be on ADH1 treatment for at least 6 months before screening for cohorts 1 to 3, or for at least 3 months before screening for cohort 4
History of thyroid or parathyroid surgery
History of renal transplantation
History of cancer (except thyroid cancer, basal cell skin cancer, or squamous cell skin cancer), skeletal malignancies, bone metastases, irradiation (radiotherapy) to the skeleton, chemotherapy with alkylating agents, Paget disease, fibrous dysplasia, chronic osteomyelitis, bone infarcts, benign bone tumors with curettage and bone grafts, retinoblastoma, or Li-Fraumeni syndrome within 5 years before screening
Received any investigational medicinal product within 30 days or 5 half-lives before Day 1, whichever is longer, or is in follow-up for another interventional clinical study during screening
Sequential
Encaleret
DrugOral tablets, age-appropriate pediatric formulation (currently under development).
Period 1: Maximum Plasma Concentration (Cmax) of Encaleret and Metabolites M1, and M3
Period 1: Area Under the Plasma Concentration-time Curve (AUC) of Encaleret and Metabolites M1, and M3
Period 3: Number of Participants with Albumin-corrected Blood Calcium (cCa) and Urinary Calcium (UCa) Excretion Response
Period 1: Change from Baseline in Blood cCa
Period 1: Change from Baseline in Blood Intact Parathyroid Hormone (iPTH) Concentration
Period 1: Change from Baseline in Blood Phosphate Concentration
Period 1: Change from Baseline in Blood 1,25-(OH)2 Vitamin D Concentration
Click on the lead sponsor to view all of their trials.
