CALIBRATE-PEDSEncaleret for Pediatric Autosomal Dominant Hypocalcemia Type 1
Encaleret
Treatment Study
Summary
Study start date: January 30, 2026
Actual date on which the first participant was enrolled.This study aims to explore how a medicine called encaleret works in children with a condition known as Autosomal Dominant Hypocalcemia Type 1 (ADH1), which affects calcium levels in the body. The study involves children from birth up to 17 years old who have this condition. Understanding the effects of encaleret could lead to better treatment options for managing calcium levels in these young patients, potentially improving their overall health and quality of life. Participants in this study will receive encaleret, and researchers will observe how the medicine moves through their bodies and its impact on their health. The medication will be given in a way that suits their age, which could involve taking it by mouth or another method. The focus will be on determining how well the drug works and ensuring it is safe for the children. Researchers will closely monitor the participants to track any changes in their condition and to assess any potential side effects of the treatment.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.28 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Until 17 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.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.
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 5 locations
Nemours Children's Health
Jacksonville, United StatesThe Children's Hospital of Philadelphia
Philadelphia, United StatesRoyal London Hospital
London, United Kingdom