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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Encaleret | Experimental | Participants will receive encaleret at a dose as needed based on calcium levels. |
|
| Standard of Care (SoC) | Other | Participants will continue receiving calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Encaleret | Drug | Administered as film-coated tablet for oral use |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Responders who Achieve Both Albumin-Corrected Blood Calcium (cCa) and 24-hour Urinary Calcium (UCa) Within the Target Range |
| Up to Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Intact Parathyroid Hormone (iPTH) Within or Greater than the Reference Range | Up to Week 24 | |
| Number of Participants who Achieve Blood Magnesium Within the Reference Range | Up to Week 24 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Calcilytix Medical Director | Calcilytix Therapeutics, Inc., a BridgeBio company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital, Oakland | Oakland | California | 94609 | United States | ||
| Children's Hospital Colorado |
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| Standard of Care | Dietary Supplement | Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.) |
|
| Number of Participants who Achieve Blood Phosphate Within the Reference Range | Up to Week 24 |
| Change From Baseline in Blood 1,25-(OH)2 Vitamin D | Baseline to Week 24 |
| Change From Baseline in cCa | Baseline to Week 24 |
| Change From Baseline in 24-hour UCa | Baseline to Week 24 |
| Change From Baseline in iPTH | Baseline to Week 24 |
| Change From Baseline in Blood Phosphate and Blood Magnesium | Baseline to Week 24 |
| Change From Baseline in Urine Magnesium, Phosphate, Sodium, and Citrate Handling | 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) | 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 | Baseline to Week 24 |
| Number of Participants in the Encaleret Arm Receiving Calcium and/or Vitamin D Supplements | Up to Week 24 |
| Steady State Encaleret Trough Concentration (Ctrough) | Up to Week 24 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Indiana University Health University Hospital | Indianapolis | Indiana | 46202 | United States |
| NIH | Bethesda | Maryland | 20892 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Mayo Clinic - Rochester | Rochester | Minnesota | 55905 | United States |
| Columbia University Irving Medical Center | New York | New York | 10032 | United States |
| Physicians East | Greenville | North Carolina | 27834 | United States |
| Ohio State University Medical Center | Columbus | Ohio | 43203 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Houston Methodist Hospital | Houston | Texas | 77030 | United States |
| Royal North Shore Hospital | Saint Leonards | New South Wales | 2065 | Australia |
| Royal Brisbane and Women's Hospital | Herston | Queensland | 4029 | Australia |
| Bone Research & Education Centre | Oakville | Ontario | L6M 1M1 | Canada |
| Vseobecna fakultni nemocnice v Praze | New Town | 128 08 | Czechia |
| Aarhus University Hospital | Aarhus | 8200 | Denmark |
| CHU Bicetre | Le Kremlin-Bicêtre | 94270 | France |
| Hôpital Edouard Herriot - HCL | Lyon | 69003 | France |
| IRCCS Ospedale San Raffaele | Milan | 20132 | Italy |
| University Hospital of Pisa | Pisa | 56124 | Italy |
| Fondazione Policlinico Universitario Campus Bio-Medico | Roma | 00128 | Italy |
| The University of Tokyo Hospital | Tokyo | 113-8655 | Japan |
| Eramus MC | Rotterdam | 3015 AA | Netherlands |
| Freeman Hospital | Newcastle upon Tyne | NE7 7DN | United Kingdom |
| Norfolk and Norwich University Hospital | Norwich | NR4 7UY | United Kingdom |
| ID | Term |
|---|---|
| C562783 | Hypercalciuric Hypocalcemia, Familial |
| D006996 | Hypocalcemia |
| D009140 | Musculoskeletal Diseases |
| D009135 | Muscular Diseases |
| D009139 | Musculoskeletal Abnormalities |
| D002128 | Calcium Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D007011 | Hypoparathyroidism |
| D053565 | Hypercalciuria |
| D009397 | Nephrocalcinosis |
| D053040 | Nephrolithiasis |
| ID | Term |
|---|---|
| D009750 | Nutritional and Metabolic Diseases |
| D014883 | Water-Electrolyte Imbalance |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D010279 | Parathyroid Diseases |
| D004700 | Endocrine System Diseases |
| D020924 | Urological Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D002114 | Calcinosis |
| D052878 | Urolithiasis |
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| ID | Term |
|---|---|
| D059039 | Standard of Care |
| ID | Term |
|---|---|
| D019984 | Quality Indicators, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
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