The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
67
Administered as film-coated tablet for oral use
Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)
UCSF Benioff Children's Hospital, Oakland
Oakland, California, United States
Number of Responders who Achieve Both Albumin-Corrected Blood Calcium (cCa) and 24-hour Urinary Calcium (UCa) Within the Target Range
* cCa within 8.3-10.7 mg/dL (2.08-2.68 millimoles per liter \[mmol/L\]) * 24-hr UCa within the reference range (\< 300 mg/day for men \[7.5 mmol/day\], \< 250 mg/day for women \[6.25 mmol/day\])
Time frame: Up to Week 24
Number of Participants With Intact Parathyroid Hormone (iPTH) Within or Greater than the Reference Range
Time frame: Up to Week 24
Number of Participants who Achieve Blood Magnesium Within the Reference Range
Time frame: Up to Week 24
Number of Participants who Achieve Blood Phosphate Within the Reference Range
Time frame: Up to Week 24
Change From Baseline in Blood 1,25-(OH)2 Vitamin D
Time frame: Baseline to Week 24
Change From Baseline in cCa
Time frame: Baseline to Week 24
Change From Baseline in 24-hour UCa
Time frame: Baseline to Week 24
Change From Baseline in iPTH
Time frame: Baseline to Week 24
Change From Baseline in Blood Phosphate and Blood Magnesium
Time frame: Baseline to Week 24
Change From Baseline in Urine Magnesium, Phosphate, Sodium, and Citrate Handling
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Children's Hospital Colorado
Aurora, Colorado, United States
Indiana University Health University Hospital
Indianapolis, Indiana, United States
NIH
Bethesda, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Columbia University Irving Medical Center
New York, New York, United States
Physicians East
Greenville, North Carolina, United States
Ohio State University Medical Center
Columbus, Ohio, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
...and 15 more locations
Time frame: Baseline to Week 24
Change From Baseline in QT Interval Corrected for Changes in the Heart Rate With Fridericia Formula (QTcF) as Assessed by Electrocardiogram (ECG)
Time frame: Baseline to Week 24
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
Time frame: Baseline to Week 24
Number of Participants in the Encaleret Arm Receiving Calcium and/or Vitamin D Supplements
Time frame: Up to Week 24
Steady State Encaleret Trough Concentration (Ctrough)
Time frame: Up to Week 24