Recruiting
Phase 2
Phase 3

Encaleret

Sponsor:

Calcilytix Therapeutics, Inc., a BridgeBio company

Code:

NCT07080385

Conditions

Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Eligibility Criteria

Sex: All

Age: 0 - 17

Healthy Volunteers: Not accepted

Interventions

Encaleret

Study Details

Brief 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.

Conditions

Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Study ID

NCT07080385

Start date

Jan 30, 2026

Status verified date

Mar, 2026

Completion date

Dec, 2030

Anticipated

Primary completion date

Dec, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 17

Healthy Volunteers: Not accepted

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 Design

Enrollment

28 participants

Anticipated

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Encaleret

Participants will receive encaleret dosing in Period 1 (6 days), Period 2 (20 weeks) and Period 3 (4 weeks). Following completion of Period 3, participants will have the option to enter a long-term extension (LTE) to continue encaleret for an additional approximately 24 months or until the sponsor decides to end the study, whichever occurs first.

Interventions

Encaleret

Oral tablets, age-appropriate pediatric formulation (currently under development).

Primary outcome measure

  • Period 1: Maximum Plasma Concentration (Cmax) of Encaleret and Metabolites M1, and M3 [ Time Frame: 5 days ]
  • Period 1: Area Under the Plasma Concentration-time Curve (AUC) of Encaleret and Metabolites M1, and M3 [ Time Frame: 5 days ]
  • Period 3: Number of Participants with Albumin-corrected Blood Calcium (cCa) and Urinary Calcium (UCa) Excretion Response [ Time Frame: Week 25 ]

Central Contacts and Locations

Central contacts

Medical Information

650-600-3610MedInfo@bridgebio.com

Locations

Yale University

Recruiting

New Haven, Connecticut, United States, 06511

Children's National Medical Center

Recruiting

Washington D.C., District of Columbia, United States, 20010

Nemours Children's Health

Recruiting

Jacksonville, Florida, United States, 32207

The Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

More Information

Sponsor

Calcilytix Therapeutics, Inc., a BridgeBio company

Last update posted

Apr 22, 2026

Last verified

Mar, 2026

Keywords

  • Autosomal dominant hypocalcemia type 1
  • ADH1
  • Encaleret

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Calcilytix Therapeutics, Inc., a BridgeBio company on 2026-04-22.