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)
Calcilytix Therapeutics, Inc., a BridgeBio company
Summary
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.
Eligibility
- Age range
- 0–17 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugEncaleret
Oral tablets, age-appropriate pediatric formulation (currently under development).
Locations (6)
- Yale UniversityNew Haven, Connecticut
- Children's National Medical CenterWashington D.C., District of Columbia
- Nemours Children's HealthJacksonville, Florida
- The Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania
- Royal London HospitalLondon
- Royal Manchester Children's HospitalManchester