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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05680818




Registration number
NCT05680818
Ethics application status
Date submitted
12/12/2022
Date registered
11/01/2023

Titles & IDs
Public title
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
Scientific title
CALIBRATE: A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Secondary ID [1] 0 0
CLTX-305-302
Universal Trial Number (UTN)
Trial acronym
CALIBRATE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autosomal Dominant Hypocalcemia (ADH) 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
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Encaleret
Treatment: Other - Standard of Care

Experimental: Encaleret - Participants will receive encaleret at a dose as needed based on calcium levels.

Other: Standard of Care (SoC) - Participants will continue receiving calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)


Treatment: Drugs: Encaleret
Administered as film-coated tablet for oral use

Treatment: Other: Standard of Care
Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Responders who Achieve Both Albumin-Corrected Blood Calcium (cCa) and 24-hour Urinary Calcium (UCa) Within the Target Range
Timepoint [1] 0 0
Up to Week 24
Secondary outcome [1] 0 0
Number of Participants With Intact Parathyroid Hormone (iPTH) Within or Greater than the Reference Range
Timepoint [1] 0 0
Up to Week 24
Secondary outcome [2] 0 0
Number of Participants who Achieve Blood Magnesium Within the Reference Range
Timepoint [2] 0 0
Up to Week 24
Secondary outcome [3] 0 0
Number of Participants who Achieve Blood Phosphate Within the Reference Range
Timepoint [3] 0 0
Up to Week 24
Secondary outcome [4] 0 0
Change From Baseline in Blood 1,25-(OH)2 Vitamin D
Timepoint [4] 0 0
Baseline to Week 24
Secondary outcome [5] 0 0
Change From Baseline in cCa
Timepoint [5] 0 0
Baseline to Week 24
Secondary outcome [6] 0 0
Change From Baseline in 24-hour UCa
Timepoint [6] 0 0
Baseline to Week 24
Secondary outcome [7] 0 0
Change From Baseline in iPTH
Timepoint [7] 0 0
Baseline to Week 24
Secondary outcome [8] 0 0
Change From Baseline in Blood Phosphate and Blood Magnesium
Timepoint [8] 0 0
Baseline to Week 24
Secondary outcome [9] 0 0
Change From Baseline in Urine Magnesium, Phosphate, Sodium, and Citrate Handling
Timepoint [9] 0 0
Baseline to Week 24
Secondary outcome [10] 0 0
Change From Baseline in QT Interval Corrected for Changes in the Heart Rate With Fridericia Formula (QTcF) as Assessed by Electrocardiogram (ECG)
Timepoint [10] 0 0
Baseline to Week 24
Secondary outcome [11] 0 0
Change from Baseline in 36-Item Short Form Health Survey (SF-36) Physical Component Score and Mental Component Score and Each of the Sub-Domains
Timepoint [11] 0 0
Baseline to Week 24
Secondary outcome [12] 0 0
Number of Participants in the Encaleret Arm Receiving Calcium and/or Vitamin D Supplements
Timepoint [12] 0 0
Up to Week 24
Secondary outcome [13] 0 0
Steady State Encaleret Trough Concentration (Ctrough)
Timepoint [13] 0 0
Up to Week 24

Eligibility
Key inclusion criteria
Key

1. Participants must have a documented pathogenic or likely pathogenic activating variant, or variant of uncertain significance, of the calcium sensing receptor (CASR) gene associated with biochemical findings of hypoparathyroidism.
2. Participants must have a documented history of symptoms or signs of ADH1.
3. Participants 16 to <18 years old must have closed growth plates on hand radiograph.
4. Participants treated with thiazide diuretics must discontinue thiazides for at least 14 days prior to SoC Optimization Visit 1 through Week 24 (Period 3). When the thiazide is being used as an antihypertensive, alternative therapy will be prescribed by the Investigator as needed.
5. Participants treated with phosphate binders (other than calcium salts) must discontinue the phosphate binders at least one day prior to the SoC Optimization Visit 1.
6. Participants treated with magnesium or potassium supplements must be willing to discontinue such treatment prior to the first dose of encaleret.
7. Participants treated with potassium-sparing diuretics must be willing to discontinue such treatment prior to the first dose of encaleret.
8. Participants must meet SoC Optimization criteria as defined in the protocol.

Key
Minimum age
16 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. History of hypocalcemic seizure within the past 3 months preceding Screening.
2. History of thyroid or parathyroid surgery.
3. History of renal transplantation.
4. Pregnant or nursing (lactating) women, where pregnancy is confirmed by a positive beta-human chorionic gonadotropin (ß-hCG) laboratory test.
5. History of treatment with parathyroid hormone (PTH) 1-84 or 1-34 within the 2 months preceding Screening and requiring SoC doses exceeding >1.2× their pre-PTH treatment total daily doses or bone turnover markers, Collagen cross-linked C-telopeptide (CTx )and Procollagen type 1 N-propeptide (P1NP), > upper limit of normal for sex, age (men only) and menopausal status (women only).
6. Blood 25-OH Vitamin D level <25 nanograms (ng)/milliliter (mL).
7. Estimated glomerular filtration rate (eGFR) <30 mL/minute/1.73 m^2 using chronic kidney disease-EPI creatinine equation refit without the race variable (chronic kidney disease-EPI creatinine equation refit without the race variable [CKD-EPIcr_R]) (for participants <18 years old the Bedside Schwartz equation should be used).
8. Participants with positive Hepatitis B surface antigen (HBsAg), Hepatitis A immunoglobulin M (IgM), or human immunodeficiency virus (HIV) viral serology test at the Screening Visit. Participants who are in complete remission from Hepatitis C virus (HCV) as evidenced by sensitive assay =12 weeks after completion of HCV therapy may participate in the study.

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
6/01/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2028
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Recruitment hospital [1] 0 0
Royal North Shore Hospital - Saint Leonards
Recruitment hospital [2] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment postcode(s) [1] 0 0
2065 - Saint Leonards
Recruitment postcode(s) [2] 0 0
4029 - Herston
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
Illinois
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
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
Canada
State/province [13] 0 0
Ontario
Country [14] 0 0
Czechia
State/province [14] 0 0
Nové Mesto
Country [15] 0 0
Denmark
State/province [15] 0 0
Aarhus
Country [16] 0 0
France
State/province [16] 0 0
Le Kremlin-Bicêtre
Country [17] 0 0
France
State/province [17] 0 0
Lyon
Country [18] 0 0
Italy
State/province [18] 0 0
Milano
Country [19] 0 0
Italy
State/province [19] 0 0
Pisa
Country [20] 0 0
Italy
State/province [20] 0 0
Roma
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
Taiwan
State/province [24] 0 0
Tainan
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Newcastle Upon Tyne
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Norwich

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
MedInfo@bridgebio.com
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/NCT05680818