NCT07080385 · Calcilytix Therapeutics, Inc., a BridgeBio company
Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)
(CALIBRATE-PEDS)
What this study is about
The overall objective of this study is to evaluate the how the drug moves through the body (PK), effectiveness, and safety of encaleret in pediatric participants from birth to 17 years of age with ADH1.
View original scientific description
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- New Haven, Connecticut
- Washington D.C., District of Columbia
- Jacksonville, Florida
- Philadelphia, Pennsylvania
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 22, 2026 · Source of record for eligibility and locations