Encaleret for children with autosomal dominant hypocalcemia type 1
Official title: A Phase 2/3, Multicenter, Single-Arm, Open-Label Study Evaluating the Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Run by Calcilytix Therapeutics, Inc., a BridgeBio company · NCT07080385 · Data updated 2026-11-12
This study tests whether encaleret is safe, how the body processes it, and if it helps newborns through 17-year-olds with ADH1.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Ages | 0 Years to 17 Years |
| Sex | All |
| Enrollment | 28 (estimated) |
| Sponsor | Calcilytix Therapeutics, Inc., a BridgeBio company Industry-sponsored |
| Drugs / interventions | chemotherapy |
| Locations | 6 sites (New Haven, Connecticut and 5 other locations) |
| Status | Recruiting |
| Started | January 30, 2026 |
| Main results expected | December 2028 |
| Study ends | December 2030 |
| Registry record updated | April 22, 2026 |
| Trial ID | NCT07080385 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Children from birth to 17 years with a pathogenic or likely pathogenic CASR variant (or compatible biochemical hypoparathyroidism), who are symptomatic and have been on ADH1 treatment for the required pre-enrollment period, are ideal candidates.
✕Probably not a fit
Patients with prior thyroid or parathyroid surgery, a history of renal transplant, or without confirmed CASR-related ADH1 are unlikely to be eligible or to benefit from this intervention.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This interventional Phase 2/3 trial gives encaleret to pediatric participants from birth to 17 years who have genetically or biochemically confirmed ADH1. Investigators will measure pharmacokinetics, monitor calcium and PTH levels, record symptoms of hypoparathyroidism, and collect safety data. Participants must be symptomatic and have been on ADH1 treatment for a specified period before enrollment, with multiple age cohorts included. The trial is conducted at U.S. pediatric centers and uses scheduled clinic visits and laboratory testing to track response and adverse events.
Why it matters
Potential benefit: If successful, encaleret could reduce hypocalcemia symptoms and decrease reliance on conventional calcium and active vitamin D therapies in children with ADH1.
How similar studies have gone: Calcilytic drugs like encaleret represent a relatively novel targeted approach with encouraging preclinical data and limited early adult clinical evidence but little established pediatric experience.
Who can join: the full rules
Show full inclusion / exclusion 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.
Where it's running
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Yale University
New Haven, Connecticut, United States Recruiting -
Children's National Medical Center
Washington D.C., District of Columbia, United States Recruiting -
Nemours Children's Health
Jacksonville, Florida, United States Recruiting -
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States Recruiting -
Royal London Hospital
London, United Kingdom Recruiting -
Royal Manchester Children's Hospital
Manchester, United Kingdom Recruiting
Study contacts
- Study coordinator: Medical Information
- Email: MedInfo@bridgebio.com
- Phone: 650-600-3610
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.