ADH1 and ADH2 Disease Monitoring Study (DMS)

Last updated: April 2, 2025
Sponsor: Calcilytix Therapeutics, Inc., a BridgeBio company
Overall Status: Active - Not Recruiting

Phase

N/A

Condition

Hypoparathyroidism

Parathyroid Disorders

Treatment

N/A

Clinical Study ID

NCT05227287
CLTX-305-901
  • Ages < 90
  • All Genders

Study Summary

A global, multi-center, Disease Monitoring Study (DMS) in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1) or Autosomal Dominant Hypocalcemia Type 2 (ADH2) designed to characterize ADH1 and ADH2 disease presentation and progression through retrospective (past) and longitudinal prospective (over time into the future) data collection.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Have a documented activating variant or variant of uncertain significance of theCASR gene causative of ADH1 or documented activating variant or variant of uncertainsignificance of the GNA11 gene causative of ADH2 associated with a clinical syndromeof hypoparathyroidism prior to enrollment

Note: Acceptable documentation includes CASR or GNA11 genetic analysis report. If no prior documented CASR or GNA11 gene variant or variant of uncertain significance, potential participants can undergo CASR and GNA11 gene variant analysis at Screening.

  • Be willing and able to provide informed consent or assent after the nature of thestudy and its details have been explained, and prior to any research-relatedprocedures

  • Be willing and able to provide access to prior medical records including imaging,biochemical, and diagnostic and medical history data, if available

  • Be willing and able to comply with the study visit schedule and study procedures

Exclusion

Key Exclusion Criteria:

  • Have serious medical or psychiatric comorbidity that, in the opinion of theInvestigator, would present a concern for participant safety or compromise theability to provide consent or assent, or comply with the study visit schedule andstudy procedures

  • Enrollment in an interventional clinical study at the time of DMS Screening visit

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 95
Study Start date:
January 20, 2022
Estimated Completion Date:
December 31, 2028

Study Description

The ADH1 and ADH2 DMS is designed to better understand the disease burden of ADH1 and ADH2, how participants with ADH1 or ADH2 are managed with standard of care practices in a real-world setting, and how standard of care treatment impacts ADH1 and ADH2 symptoms.

The study will include adult and pediatric participants with a confirmed clinical diagnosis of ADH1 or ADH2. Each participant's data will be collected over a period of up to 5 years. In addition, retrospective (or past) data will be collected.

Connect with a study center

  • Royal North Shore Hospital

    Saint Leonards, New South Wales 2065
    Australia

    Site Not Available

  • Universitair Ziekenhuis Brussel

    Jette, Brussels-Capital 1090
    Belgium

    Site Not Available

  • Universitaire Ziekenhuizen Leuven

    Leuven, Flemish Brabant 3000
    Belgium

    Site Not Available

  • Bone Research and Education Centre

    Oakville, Ontario L6M 1M1
    Canada

    Site Not Available

  • Aarhus University Hospital

    Aarhus, 8200
    Denmark

    Site Not Available

  • Helsinki University Hospital (HUS) - The New Children's Hospital

    Helsinki, 00290
    Finland

    Site Not Available

  • HCL Hopital Femme Mere Enfant

    Bron, Auvergne-Rhône-Alpes 69500
    France

    Site Not Available

  • HCL Hopital Edouard Herriot

    Lyon, Auvergne-Rhône-Alpes 69003
    France

    Site Not Available

  • Departement d'Endocrinologie et Diabetes pour Enfants - AP-HP Hopital Bicetre

    Le Kremlin-Bicêtre, Île-de-France 94270
    France

    Site Not Available

  • CHU de Lille

    Lille, 59000
    France

    Site Not Available

  • Endokrinologikum Gottingen

    Göttingen, 37075
    Germany

    Site Not Available

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano

    Milano, Lombardy 20122
    Italy

    Site Not Available

  • IRCCS Ospedale San Raffaele

    Milano, Lombardy 20132
    Italy

    Site Not Available

  • Policlinico Universitario Campus Bio-Medico

    Rome, 00128
    Italy

    Site Not Available

  • Osaka University Hospital

    Osaka, 565-0871
    Japan

    Site Not Available

  • The University of Tokyo Hospital

    Tokyo, 113-8655
    Japan

    Site Not Available

  • Erasmus MC

    Rotterdam, 3015 GD
    Netherlands

    Site Not Available

  • Centro Hospitalar Universitario de Lisboa Norte - Hospital de Santa Maria

    Lisbon, 1649-028
    Portugal

    Site Not Available

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL
    United Kingdom

    Site Not Available

  • University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland

    Oakland, California 94609
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Nemours Children's Clinic

    Jacksonville, Florida 32207
    United States

    Site Not Available

  • Indiana University (IU) School of Medicine - University Hospital

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Johns Hopkins Bayview Medical Center

    Baltimore, Maryland 21224
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Mayo Clinic

    Rochester, Minnesota 55902
    United States

    Site Not Available

  • Physician's East Endocrinology

    Greenville, North Carolina 27834
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Ohio State University Medical Center (OSUMC)

    Columbus, Ohio 43210
    United States

    Site Not Available

  • The Diabetes & Thyroid Center of Fort Worth (DTC)

    Fort Worth, Texas 76132
    United States

    Site Not Available

  • Houston Methodist Hospital

    Houston, Texas 77030
    United States

    Site Not Available

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