New drug aims to fix calcium levels in rare genetic disease

NCT ID NCT05680818

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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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.

Contacts and locations

Locations

  • Aarhus University Hospital

    Aarhus, 8200, Denmark

  • Bone Research & Education Centre

    Oakville, Ontario, L6M 1M1, Canada

  • CHU Bicetre

    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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