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Recruiting Phase 2/3

Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)

NCT07080385 · tracked via the Priya Life Science USA tracker
Phase
Phase 2/3
Started
2026-01-30
Last updated
2026-04-22

Condition(s) studied

Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Investigational drug(s) / intervention(s)

Encaleret →

Encaleret: Oral tablets, age-appropriate pediatric formulation (currently under development).

Study summary

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.

Eligibility

Sex
ALL
Min age
0 Years
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • 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

Trial sites (6)

FacilityCityRegionStatus
Yale University New Haven Connecticut Recruiting
Children's National Medical Center Washington D.C. District of Columbia Recruiting
Nemours Children's Health Jacksonville Florida Recruiting
The Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
Royal London Hospital London United Kingdom Recruiting
Royal Manchester Children's Hospital Manchester United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07080385 on ClinicalTrials.gov ↗ ← All trials in the USA