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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05227287




Registration number
NCT05227287
Ethics application status
Date submitted
18/01/2022
Date registered
7/02/2022

Titles & IDs
Public title
ADH1 and ADH2 Disease Monitoring Study (DMS)
Scientific title
Autosomal Dominant Hypocalcemia Types 1 And 2 (ADH1/2) Disease Monitoring Study (DMS)
Secondary ID [1] 0 0
CLTX-305-901
Universal Trial Number (UTN)
Trial acronym
CLARIFY
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autosomal Dominant Hypocalcemia 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
ADH 1/2 DMS - Participants with ADH1 or ADH2. No investigational product will be administered to participants in this study. Participants will only receive standard of care (SoC) treatment as directed by the participants' treating physicians.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Blood Calcium Homeostasis
Timepoint [1] 0 0
Up to 60 months
Primary outcome [2] 0 0
Phosphorus Homeostasis
Timepoint [2] 0 0
Up to 60 months
Primary outcome [3] 0 0
Magnesium Homeostasis
Timepoint [3] 0 0
Up to 60 months
Primary outcome [4] 0 0
Intact Parathyroid Hormone (iPTH) Homeostasis
Timepoint [4] 0 0
Up to 60 months
Primary outcome [5] 0 0
Mineral Homeostasis as Assessed by 1,25-dihydroxyvitamin D Homeostasis
Timepoint [5] 0 0
Up to 60 months
Primary outcome [6] 0 0
Urine Calcium Homeostasis
Timepoint [6] 0 0
Up to 60 months
Primary outcome [7] 0 0
Urine Phosphorus Homeostasis
Timepoint [7] 0 0
Up to 60 months
Primary outcome [8] 0 0
Urine Magnesium Homeostasis
Timepoint [8] 0 0
Up to 60 months
Secondary outcome [1] 0 0
Blood Creatinine Levels
Timepoint [1] 0 0
Up to 60 months
Secondary outcome [2] 0 0
Estimated Glomerular Filtration Rate (eGFR)
Timepoint [2] 0 0
Up to 60 months
Secondary outcome [3] 0 0
Number of Participants With Nephrocalcinosis and Nephrolithiasis as Assessed by Renal Ultrasound
Timepoint [3] 0 0
Up to 60 months
Secondary outcome [4] 0 0
Bone Mineral Density as Assessed by Dual-Energy X-Ray Absorptiometry (DXA)
Timepoint [4] 0 0
Up to 60 months
Secondary outcome [5] 0 0
Change from Baseline in 36-Item Short Form Health Survey (SF-36v2) Physical Component Score and Mental Component Score in Participants = 16 years
Timepoint [5] 0 0
Up to 60 months
Secondary outcome [6] 0 0
Change from Baseline in 10-Item Short-Form 10 Healthy Survey for Children (SF-10) Score in participants = 6 years and <16 years
Timepoint [6] 0 0
Up to 60 months
Secondary outcome [7] 0 0
Number of Participants Receiving One or More ADH1/2 Treatment Regimens
Timepoint [7] 0 0
Up to 60 months

Eligibility
Key inclusion criteria
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
Minimum age
No limit
Maximum age
90 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 interventional clinical study at the time of DMS Screening visit

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

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/01/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2028
Actual
Sample size
Target
Accrual to date
Final
95
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Royal North Shore Hospital - Saint Leonards
Recruitment postcode(s) [1] 0 0
2065 - Saint Leonards
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
North Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
Belgium
State/province [11] 0 0
Flemish Brabant
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
Denmark
State/province [13] 0 0
Aarhus
Country [14] 0 0
Finland
State/province [14] 0 0
Helsinki
Country [15] 0 0
France
State/province [15] 0 0
Auvergne-Rhône-Alpes
Country [16] 0 0
France
State/province [16] 0 0
Lille
Country [17] 0 0
France
State/province [17] 0 0
Île-de-France
Country [18] 0 0
Germany
State/province [18] 0 0
Göttingen
Country [19] 0 0
Italy
State/province [19] 0 0
Lombardy
Country [20] 0 0
Italy
State/province [20] 0 0
Rome
Country [21] 0 0
Japan
State/province [21] 0 0
Osaka
Country [22] 0 0
Japan
State/province [22] 0 0
Tokyo
Country [23] 0 0
Netherlands
State/province [23] 0 0
Rotterdam
Country [24] 0 0
Portugal
State/province [24] 0 0
Lisbon
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Calcilytix Therapeutics, Inc., a BridgeBio company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Calcilytix Medical Director
Address 0 0
Calcilytix Therapeutics, Inc., a BridgeBio company
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Information
Address 0 0
Country 0 0
Phone 0 0
650-600-3610
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05227287