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New drug aims to fix calcium levels in rare genetic disease

NCT ID NCT05680818

Disease control Sponsor: Calcilytix Therapeutics, Inc., a BridgeBio company Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 30, 2026 · Updated 2 times

Summary

This study tests a new drug called encaleret against usual treatments for people with a rare genetic condition called autosomal dominant hypocalcemia type 1 (ADH1), which causes low blood calcium and high urine calcium. About 67 participants will receive either encaleret or standard care to see if the drug can safely bring both blood and urine calcium into normal ranges. The goal is to better control the disease, not cure it.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

67 people

The number who actually took part.

Started

Jan 2023

Expected to finish

Aug 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

16 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. Participants must have a documented pathogenic or likely pathogenic activating variant, or variant of uncertain significance, of the calcium sensing receptor (CASR) gene associated with biochemical findings of hypoparathyroidism. 2. Participants must have a documented history of symptoms or signs of ADH1. 3. Participants 16 to \<18 years old must have closed growth plates on hand radiograph. 4. Participants treated with thiazide diuretics must discontinue thiazides for at least 14 days prior to SoC Optimization Visit 1 through Week 24 (Period 3). When the thiazide is being used as an antihypertensive, alternative therapy will be prescribed by the Investigator as needed. 5. Participants treated with phosphate binders (other than calcium salts) must discontinue the phosphate binders at least one day prior to the SoC Optimization Visit 1. 6. Participants treated with magnesium or potassium supplements must be willing to discontinue such treatment prior to the first dose of encaleret. 7. Participants treated with potassium-sparing diuretics must be willing to discontinue such treatment prior to the first dose of encaleret. 8. Participants must meet SoC Optimization criteria as defined in the protocol. Key Exclusion Criteria: 1. History of hypocalcemic seizure within the past 3 months preceding Screening. 2. History of thyroid or parathyroid surgery. 3. History of renal transplantation. 4. Pregnant or nursing (lactating) women, where pregnancy is confirmed by a positive beta-human chorionic gonadotropin (β-hCG) laboratory test. 5. History of treatment with parathyroid hormone (PTH) 1-84 or 1-34 within the 2 months preceding Screening and requiring SoC doses exceeding \>1.2× their pre-PTH treatment total daily doses or bone turnover markers, Collagen cross-linked C-telopeptide (CTx )and Procollagen type 1 N-propeptide (P1NP), \> upper limit of normal for sex, age (men only) and menopausal status (women only). 6. Blood 25-OH Vitamin D level \<25 nanograms (ng)/milliliter (mL). 7. Estimated glomerular filtration rate (eGFR) \<30 mL/minute/1.73 m\^2 using chronic kidney disease-EPI creatinine equation refit without the race variable (chronic kidney disease-EPI creatinine equation refit without the race variable \[CKD-EPIcr\_R\]) (for participants \<18 years old the Bedside Schwartz equation should be used). 8. Participants with positive Hepatitis B surface antigen (HBsAg), Hepatitis A immunoglobulin M (IgM), or human immunodeficiency virus (HIV) viral serology test at the Screening Visit. Participants who are in complete remission from Hepatitis C virus (HCV) as evidenced by sensitive assay ≥12 weeks after completion of HCV therapy may participate in the study. Note: Other protocol defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

autosomal dominant hypocalcemia autosomal dominant hypocalcemia 1 calcium metabolic disease Hypercalciuria Hypercalciuric Hypocalcemia, Familial Hypocalcemia hypoparathyroidism metabolic disease muscle tissue disorder Musculoskeletal Abnormalities musculoskeletal system disorder nephrocalcinosis nephrolithiasis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Autosomal dominant hypocalcemia (ADH)

Contacts and locations

Locations

  • Aarhus University Hospital

    Aarhus, 8200, Denmark

  • Bone Research & Education Centre

    Oakville, Ontario, L6M 1M1, Canada

  • CHU Bicetre

    Le Kremlin-Bicêtre, Le Kremlin-Bicêtre, 94270, France

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Eramus MC

    Rotterdam, 3015 AA, Netherlands

  • Fondazione Policlinico Universitario Campus Bio-Medico

    Roma, 00128, Italy

  • Freeman Hospital

    Newcastle upon Tyne, NE7 7DN, United Kingdom

  • Houston Methodist Hospital

    Houston, Texas, 77030, United States

  • Hôpital Edouard Herriot - HCL

    Lyon, 69003, France

  • IRCCS Ospedale San Raffaele

    Milan, 20132, Italy

  • Indiana University Health University Hospital

    Indianapolis, Indiana, 46202, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905, United States

  • NIH

    Bethesda, Maryland, 20892, United States

  • Norfolk and Norwich University Hospital

    Norwich, NR4 7UY, United Kingdom

  • Ohio State University Medical Center

    Columbus, Ohio, 43203, United States

  • Physicians East

    Greenville, North Carolina, 27834, United States

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4029, Australia

  • Royal North Shore Hospital

    Saint Leonards, New South Wales, 2065, Australia

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • The University of Tokyo Hospital

    Tokyo, 113-8655, Japan

  • UCSF Benioff Children's Hospital, Oakland

    Oakland, California, 94609, United States

  • University Hospital of Pisa

    Pisa, 56124, Italy

  • Vseobecna fakultni nemocnice v Praze

    New Town, 128 08, Czechia

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