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Recruiting Phase 2/3 NCT07080385

Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Phase 2/3 – combined trial
Conditions: Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Sponsor: Calcilytix Therapeutics, Inc., a BridgeBio company

trial.available_in: БГ
Overview
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
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.
Interventions
Encaleret
DRUG
Locations 6
United Kingdom (2)
Royal London Hospital
London
Royal Manchester Children's Hospital
Manchester
United States (4)
Nemours Children's Health
Jacksonville , Florida
Yale University
New Haven , Connecticut
The Children's Hospital of Philadelphia
Philadelphia , Pennsylvania
Children's National Medical Center
Washington D.C. , District of Columbia
Technical details
Status
Recruiting
Phase
Phase 2/3
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
0 Years
Maximum age
17 Years
Healthy volunteers
No
Start date
30.01.2026
Completion date
01.12.2030
Registry ID
NCT07080385
Source
clinicaltrials.gov
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