Calcilytix Therapeutics, Inc., a BridgeBio company
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)
Facility
City
Region
Status
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
More Calcilytix Therapeutics, Inc., a BridgeBio company trials in the USA
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.
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