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NCT Number: NCT07080385

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

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.

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Key information

Age range

0 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Royal London Hospital, London, United Kingdom

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

encaleret

Drug

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

Primary outcomes

  1. Period 1: Maximum Plasma Concentration (Cmax) of Encaleret and Metabolites M1, and M3

    Time frame: 5 days

  2. Period 1: Area Under the Plasma Concentration-time Curve (AUC) of Encaleret and Metabolites M1, and M3

    Time frame: 5 days

  3. Period 3: Number of Participants with Albumin-corrected Blood Calcium (cCa) and Urinary Calcium (UCa) Excretion Response

    Time frame: Week 25

Secondary outcomes

  1. Period 1: Change from Baseline in Blood cCa

    Time frame: Baseline up to Day 5

  2. Period 1: Change from Baseline in Blood Intact Parathyroid Hormone (iPTH) Concentration

    Time frame: Baseline up to Day 5

  3. Period 1: Change from Baseline in Blood Phosphate Concentration

    Time frame: Baseline up to Day 5

  4. Period 1: Change from Baseline in Blood 1,25-(OH)2 Vitamin D Concentration

    Time frame: Baseline up to Day 5

  5. Period 1: Change from Baseline in Blood Magnesium Concentration

    Time frame: Baseline up to Day 5

  6. Period 1: Change from Baseline in 24-hour UCa in Toilet Trained Participants

    Time frame: Baseline up to Day 5

  7. Period 1: Change from Baseline in Spot Ratio of UCa/Urinary Creatinine (UCr) In Non-toilet Trained Participants

    Time frame: Baseline up to Day 5

  8. Period 3: Number of Participants with Blood iPTH Within the Reference Range

    Time frame: Week 25 (post-dose)

  9. Period 3: Number of Participants with Blood Phosphate Within the Reference Range

    Time frame: Week 25

  10. Period 3: Number of Participants with Blood Magnesium Within the Reference Range

    Time frame: Week 25

  11. Period 3: Number of Participants with Blood 1,25-(OH)2 Vitamin D Within the Reference Range

    Time frame: Week 25

  12. Periods 1, 2, and 3: Change from Baseline in Short Form-10 Health Survey for Children (SF-10) in Participants Aged ≥6 Years

    Time frame: Baseline up to Week 25

  13. Periods 1, 2, and 3: Dose of Calcium Supplements and/or Active Vitamin D Analogs Used as Rescue Therapy

    Time frame: 25 weeks

  14. Periods 1, 2, and 3: Dosing Frequency of Calcium Supplements and/or Active Vitamin D Analogs Used as Rescue Therapy

    Time frame: 25 weeks

  15. Periods 1, 2, 3, and LTE: Number of Participants with Adverse Events (AEs) and Serious AEs (SAEs)

    Time frame: 145 weeks

  16. LTE: Change from Baseline in Blood cCa

    Time frame: Baseline up to 145 weeks

  17. LTE: Change from Baseline in Blood iPTH Concentration

    Time frame: Baseline up to 145 weeks

  18. LTE: Change from Baseline in Blood Phosphate Concentration

    Time frame: Baseline up to 145 weeks

  19. LTE: Change from Baseline in Blood 1,25-(OH)2 Vitamin D Concentration

    Time frame: Baseline up to 145 weeks

  20. LTE: Change from Baseline in Blood Magnesium Concentration

    Time frame: Baseline up to 145 weeks

  21. LTE: Change from Baseline in 24-hour UCa In Toilet Trained Participants

    Time frame: Baseline up to 145 weeks

  22. LTE: Change from Baseline in Spot Ratio of UCa/UCr In Non-toilet Trained Participants

    Time frame: Baseline up to 145 weeks

  23. LTE: Change from Baseline in SF-10 in Participants Aged ≥6 Years

    Time frame: Baseline up to 145 weeks

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Information

CONTACT

[email protected]

650-600-3610

Sponsors and collaborators

Lead sponsor

Calcilytix Therapeutics, Inc., a BridgeBio company

Industry

Registry information

Official study 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)

Acronym: CALIBRATE-PEDS

Important dates

Study start
2026
Primary completion
2028
Study completion
2030
First posted
Jul 23, 2025
Registry last updated
Apr 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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