Clinical Trials in the USA / NCT07080385
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
Condition(s) studied
Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Investigational drug(s) / intervention(s)
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
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
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
Active, not recruiting
Efficacy, Safety, and Tolerability of Encaleret in Participants With Chronic Hypoparathyroidism
Recruiting
ADH1 and ADH2 Disease Monitoring Study (DMS)
Active, not 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