CLARIFY: ADH1 and ADH2 Disease Monitoring Study (DMS)

Sponsor

Calcilytix Therapeutics, Inc., a BridgeBio company (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05227287

Collaborator

(none)

Enrollment

Locations

81.4

Anticipated Duration (Months)

8.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

A global, multi-center, non-interventional 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.

Condition or Disease	Intervention/Treatment	Phase
Autosomal Dominant Hypocalcemia

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 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.

Study Design

Study Type:

Observational

Anticipated Enrollment :

60 participants

Observational Model:

Cohort

Time Perspective:

Other

Official Title:

CLARIFY: Autosomal Dominant Hypocalcemia Types 1 And 2 (ADH1/2) Disease Monitoring Study (DMS)

Actual Study Start Date :

Jan 20, 2022

Anticipated Primary Completion Date :

Nov 1, 2028

Anticipated Study Completion Date :

Nov 1, 2028

Outcome Measures

Primary Outcome Measures

Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of blood calcium homeostasis over time
Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of phosphorus homeostasis over time
Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of magnesium homeostasis over time
Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of albumin over time
Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of intact PTH (iPTH) homeostasis over time
Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of intact 1,25-dihydroxyvitamin D homeostasis over time
Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of urine calcium homeostasis over time
Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of urine phosphorus homeostasis over time
Retrospective and prospective determination of the biochemical manifestations and progression of ADH1 and ADH2 over time [Initial diagnosis through the duration of the study (Month 60)]
Retrospective and prospective assessment of urine creatinine over time

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 90 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Have a documented activating variant of the CASR gene for ADH1 or documented activating variant of the GNA11 gene for ADH2 associated with a clinical syndrome of hypoparathyroidism prior to enrollment
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 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 Criteria:

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 ADH1 or ADH2 interventional clinical study at the time of DMS Screening visit or at any point during the DMS

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Indiana University (IU) School of Medicine - University Hospital	Indianapolis	Indiana	United States	46202
2	Physician's East Endocrinology	Greenville	North Carolina	United States	27834
3	Ohio State University Medical Center (OSUMC)	Columbus	Ohio	United States	43210
4	The Diabetes & Thyroid Center of Fort Worth (DTC)	Fort Worth	Texas	United States	76132
5	Bone Research and Education Centre	Oakville	Ontario	Canada	L6M 1M1
6	HCL Hopital Femme Mere Enfant	Bron	Auvergne-Rhône-Alpes	France	69500
7	HCL Hopital Edouard Herriot	Lyon	Auvergne-Rhône-Alpes	France	69003