CALIBRATE-PEDS
Research type
Research Study
Full 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)
IRAS ID
1012601
Contact name
Scott Adler
Contact email
Sponsor organisation
Calcilytix Therapeutics, Inc., a BridgeBio Company
Clinicaltrials.gov Identifier
Research summary
Autosomal Dominant Hypocalcemia Type 1 (ADH1) occurs when the calcium-sensing receptor (CaSR) is abnormal and senses that blood calcium is higher than it actually is. When this happens, blood calcium becomes low. Low levels of calcium in the blood can cause symptoms such as cramping or tingling in the hands, feet, or other parts of the body. A very low blood calcium can cause fainting or seizures and can have effects on the heart. The study medicine encaleret may help bring calcium levels in your blood back to a normal level by correcting how your CaSR is working. This research study will check how safe and effective encaleret is in children and adults under 18 years of age, or adults who began study participation while under 18 years of age, with ADH1.
Currently, there is no approved therapy is available for managing ADH1 in children. Similar to other forms of hypoparathyroidism, the conventional standard of care involves administering oral active vitamin D and calcium.
Approximately 28 children and adults under the age of 18, at the time of initial consent, will take part in this study at approximately 8 study sites globally. This is an open-label, single-arm study.
There are 5 stages to this study:
• Screening (up to 89 days to determine participant eligibility)
• Period 1 (up to 6-day in-patient stay in the hospital)
• Period 2 (20-week outpatient dose optimisation, to determine the best dose)
• Period 3 (4-week outpatient dose maintenance), during which time the participant will stay on the dose selected in period 2)
• Optional Long-Term Extension (up to 24-month outpatient continued study treatment)REC name
North West - Liverpool Central Research Ethics Committee
REC reference
25/NW/0281
Date of REC Opinion
29 Oct 2025
REC opinion
Further Information Favourable Opinion