Status:

ACTIVE_NOT_RECRUITING

ADH1 and ADH2 Disease Monitoring Study (DMS)

Lead Sponsor:

Calcilytix Therapeutics, Inc., a BridgeBio company

Conditions:

Autosomal Dominant Hypocalcemia

Eligibility:

All Genders

Up to 90 years

Brief 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...

Detailed 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 ...

Eligibility Criteria

Inclusion

  • Key
  • Have a documented activating variant or variant of uncertain significance of the CASR gene causative of ADH1 or documented activating variant or variant of uncertain significance of the GNA11 gene causative of ADH2 associated with a clinical syndrome of 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 the study and its details have been explained, and prior to any research-related procedures
  • 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
  • Key

Exclusion

  • Have serious medical or psychiatric comorbidity that, in the opinion of the Investigator, would present a concern for participant safety or compromise the ability to provide consent or assent, or comply with the study visit schedule and study procedures
  • Enrollment in an interventional clinical study at the time of DMS Screening visit
  • Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Key Trial Info

Start Date :

January 20 2022

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

December 1 2028

Estimated Enrollment :

95 Patients enrolled

Trial Details

Trial ID

NCT05227287

Start Date

January 20 2022

End Date

December 1 2028

Last Update

April 4 2025

Active Locations (27)

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Page 1 of 7 (27 locations)

1

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

Oakland, California, United States, 94609

2

Children's Hospital Colorado

Aurora, Colorado, United States, 80045

3

Nemours Children's Clinic

Jacksonville, Florida, United States, 32207

4

Indiana University (IU) School of Medicine - University Hospital

Indianapolis, Indiana, United States, 46202