Pharmacokinetics, Efficacy, and Safety of Encaleret in Pe... | NCT07080385

Status Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Encaleret	Experimental	Participants will receive encaleret dosing in Period 1 (6 days), Period 2 (20 weeks) and Period 3 (4 weeks). Following completion of Period 3, participants will have the option to enter a long-term extension (LTE) to continue encaleret for an additional approximately 24 months or until the sponsor decides to end the study, whichever occurs first.	Drug: Encaleret

Name	Type	Description	Arm Group Labels	Other Names
Encaleret	Drug	Oral tablets, age-appropriate pediatric formulation (currently under development).	Encaleret

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Period 1: Maximum Plasma Concentration (Cmax) of Encaleret and Metabolites M1, and M3		5 days
Period 1: Area Under the Plasma Concentration-time Curve (AUC) of Encaleret and Metabolites M1, and M3		5 days
Period 3: Number of Participants with Albumin-corrected Blood Calcium (cCa) and Urinary Calcium (UCa) Excretion Response		Week 25

Secondary Outcomes

Measure	Description	Time Frame
Period 1: Change from Baseline in Blood cCa		Baseline up to Day 5
Period 1: Change from Baseline in Blood Intact Parathyroid Hormone (iPTH) Concentration		Baseline up to Day 5

Eligibility Module

Eligibility Criteria

Key Inclusion Criteria:

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

Key Exclusion Criteria:

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
Treatment with a strong P-glycoprotein (P-gp) inhibitor within 300 days before screening for amiodarone or within 30 days before screening for any other strong P-gp inhibitor
Treatment with cardiac glycosides, or is being breastfed while the participant's nursing mother is treated with cardiac glycosides, within 30 days before screening
Presence or history of any disease or condition (eg, drug or alcohol dependence) that would affect the participant's safety, treatment compliance, or ability to complete the study, in the opinion of the investigator

Other protocol defined inclusion/exclusion criteria apply.

Facility	Status	City	State	ZIP	Country	Contacts
Yale University	Recruiting	New Haven	Connecticut	06511	United States
Children's National Medical Center