Ignite Creation Date:
2025-12-25 @ 4:37 AM
Ignite Modification Date:
2025-12-26 @ 3:39 AM
Study NCT ID:
NCT05680818
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-31
First Post:
2022-12-12
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Taiwan']}, 'conditionBrowseModule': {'meshes': [{'id': 'C562783', 'term': 'Hypercalciuric Hypocalcemia, Familial'}, {'id': 'D006996', 'term': 'Hypocalcemia'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009139', 'term': 'Musculoskeletal Abnormalities'}, {'id': 'D002128', 'term': 'Calcium Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D007011', 'term': 'Hypoparathyroidism'}, {'id': 'D053565', 'term': 'Hypercalciuria'}, {'id': 'D009397', 'term': 'Nephrocalcinosis'}, {'id': 'D053040', 'term': 'Nephrolithiasis'}], 'ancestors': [{'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D014883', 'term': 'Water-Electrolyte Imbalance'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D010279', 'term': 'Parathyroid Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D020924', 'term': 'Urological Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D002114', 'term': 'Calcinosis'}, {'id': 'D052878', 'term': 'Urolithiasis'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D059039', 'term': 'Standard of Care'}], 'ancestors': [{'id': 'D019984', 'term': 'Quality Indicators, Health Care'}, {'id': 'D011787', 'term': 'Quality of Health Care'}, {'id': 'D006298', 'term': 'Health Services Administration'}, {'id': 'D017530', 'term': 'Health Care Quality, Access, and Evaluation'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 67}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2023-01-06', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2029-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-30', 'studyFirstSubmitDate': '2022-12-12', 'studyFirstSubmitQcDate': '2023-01-09', 'lastUpdatePostDateStruct': {'date': '2025-10-31', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-01-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-08-22', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Responders who Achieve Both Albumin-Corrected Blood Calcium (cCa) and 24-hour Urinary Calcium (UCa) Within the Target Range', 'timeFrame': 'Up to Week 24', 'description': '* cCa within 8.3-10.7 mg/dL (2.08-2.68 millimoles per liter \\[mmol/L\\])\n* 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\\])'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Intact Parathyroid Hormone (iPTH) Within or Greater than the Reference Range', 'timeFrame': 'Up to Week 24'}, {'measure': 'Number of Participants who Achieve Blood Magnesium Within the Reference Range', 'timeFrame': 'Up to Week 24'}, {'measure': 'Number of Participants who Achieve Blood Phosphate Within the Reference Range', 'timeFrame': 'Up to Week 24'}, {'measure': 'Change From Baseline in Blood 1,25-(OH)2 Vitamin D', 'timeFrame': 'Baseline to Week 24'}, {'measure': 'Change From Baseline in cCa', 'timeFrame': 'Baseline to Week 24'}, {'measure': 'Change From Baseline in 24-hour UCa', 'timeFrame': 'Baseline to Week 24'}, {'measure': 'Change From Baseline in iPTH', 'timeFrame': 'Baseline to Week 24'}, {'measure': 'Change From Baseline in Blood Phosphate and Blood Magnesium', 'timeFrame': 'Baseline to Week 24'}, {'measure': 'Change From Baseline in Urine Magnesium, Phosphate, Sodium, and Citrate Handling', 'timeFrame': 'Baseline to Week 24'}, {'measure': 'Change From Baseline in QT Interval Corrected for Changes in the Heart Rate With Fridericia Formula (QTcF) as Assessed by Electrocardiogram (ECG)', 'timeFrame': 'Baseline to Week 24'}, {'measure': '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', 'timeFrame': 'Baseline to Week 24'}, {'measure': 'Number of Participants in the Encaleret Arm Receiving Calcium and/or Vitamin D Supplements', 'timeFrame': 'Up to Week 24'}, {'measure': 'Steady State Encaleret Trough Concentration (Ctrough)', 'timeFrame': 'Up to Week 24'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Autosomal Dominant Hypocalcemia Type 1 (ADH1)', 'Hypocalcemia', 'Musculoskeletal Diseases', 'Muscular Diseases', 'Musculoskeletal Abnormalities', 'Calcium Metabolism Disorders', 'Metabolic Diseases', 'Hypoparathyroidism', 'Hypocalcemic Seizures', 'Hypercalciuria', 'Nephrocalcinosis', 'Nephrolithiasis', 'Calcium Sensing Receptor', 'Encaleret', 'Hypopara'], 'conditions': ['Autosomal Dominant Hypocalcemia (ADH)']}, 'descriptionModule': {'briefSummary': '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).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. 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.\n2. Participants must have a documented history of symptoms or signs of ADH1.\n3. Participants 16 to \\<18 years old must have closed growth plates on hand radiograph.\n4. 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.\n5. 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.\n6. Participants treated with magnesium or potassium supplements must be willing to discontinue such treatment prior to the first dose of encaleret.\n7. Participants treated with potassium-sparing diuretics must be willing to discontinue such treatment prior to the first dose of encaleret.\n8. Participants must meet SoC Optimization criteria as defined in the protocol.\n\nKey Exclusion Criteria:\n\n1. History of hypocalcemic seizure within the past 3 months preceding Screening.\n2. History of thyroid or parathyroid surgery.\n3. History of renal transplantation.\n4. Pregnant or nursing (lactating) women, where pregnancy is confirmed by a positive beta-human chorionic gonadotropin (β-hCG) laboratory test.\n5. 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).\n6. Blood 25-OH Vitamin D level \\<25 nanograms (ng)/milliliter (mL).\n7. 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).\n8. 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.\n\nNote: Other protocol defined Inclusion/Exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT05680818', 'acronym': 'CALIBRATE', 'briefTitle': 'Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1', 'organization': {'class': 'INDUSTRY', 'fullName': 'Calcilytix Therapeutics, Inc., a BridgeBio company'}, 'officialTitle': '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)', 'orgStudyIdInfo': {'id': 'CLTX-305-302'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Encaleret', 'description': 'Participants will receive encaleret at a dose as needed based on calcium levels.', 'interventionNames': ['Drug: Encaleret']}, {'type': 'OTHER', 'label': 'Standard of Care (SoC)', 'description': 'Participants will continue receiving calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)', 'interventionNames': ['Dietary Supplement: Standard of Care']}], 'interventions': [{'name': 'Encaleret', 'type': 'DRUG', 'otherNames': ['CLTX-305', 'Encaleret Sulfate'], 'description': 'Administered as film-coated tablet for oral use', 'armGroupLabels': ['Encaleret']}, {'name': 'Standard of Care', 'type': 'DIETARY_SUPPLEMENT', 'description': 'Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.)', 'armGroupLabels': ['Standard of Care (SoC)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "UCSF Benioff Children's Hospital, Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University Health University Hospital', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '20892', 'city': 'Bethesda', 'state': 'Maryland', 'country': 'United States', 'facility': 'NIH', 'geoPoint': {'lat': 38.98067, 'lon': -77.10026}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Clinic - Rochester', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Irving Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27834', 'city': 'Greenville', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Physicians East', 'geoPoint': {'lat': 35.61266, 'lon': -77.36635}}, {'zip': '43203', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'Ohio State University Medical Center', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "The Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Houston Methodist Hospital', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '2065', 'city': 'Saint Leonards', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Royal North Shore Hospital'}, {'zip': '4029', 'city': 'Herston', 'state': 'Queensland', 'country': 'Australia', 'facility': "Royal Brisbane and Women's Hospital", 'geoPoint': {'lat': -27.44453, 'lon': 153.01852}}, {'zip': 'L6M 1M1', 'city': 'Oakville', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Bone Research & Education Centre', 'geoPoint': {'lat': 43.45011, 'lon': -79.68292}}, {'zip': '128 08', 'city': 'New Town', 'country': 'Czechia', 'facility': 'Vseobecna fakultni nemocnice v Praze', 'geoPoint': {'lat': 50.07829, 'lon': 14.42089}}, {'zip': '8200', 'city': 'Aarhus', 'country': 'Denmark', 'facility': 'Aarhus University Hospital', 'geoPoint': {'lat': 56.15674, 'lon': 10.21076}}, {'zip': '94270', 'city': 'Le Kremlin-Bicêtre', 'state': 'Le Kremlin-Bicêtre', 'country': 'France', 'facility': 'CHU Bicetre', 'geoPoint': {'lat': 48.81471, 'lon': 2.36073}}, {'zip': '69003', 'city': 'Lyon', 'country': 'France', 'facility': 'Hôpital Edouard Herriot - HCL', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'zip': '20132', 'city': 'Milan', 'country': 'Italy', 'facility': 'IRCCS Ospedale San Raffaele', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '56124', 'city': 'Pisa', 'country': 'Italy', 'facility': 'University Hospital of Pisa', 'geoPoint': {'lat': 43.70853, 'lon': 10.4036}}, {'zip': '00128', 'city': 'Roma', 'country': 'Italy', 'facility': 'Fondazione Policlinico Universitario Campus Bio-Medico', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '113-8655', 'city': 'Tokyo', 'country': 'Japan', 'facility': 'The University of Tokyo Hospital', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': '3015 AA', 'city': 'Rotterdam', 'country': 'Netherlands', 'facility': 'Eramus MC', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'zip': 'NE7 7DN', 'city': 'Newcastle upon Tyne', 'country': 'United Kingdom', 'facility': 'Freeman Hospital', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}, {'zip': 'NR4 7UY', 'city': 'Norwich', 'country': 'United Kingdom', 'facility': 'Norfolk and Norwich University Hospital', 'geoPoint': {'lat': 52.62783, 'lon': 1.29834}}], 'overallOfficials': [{'name': 'Calcilytix Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Calcilytix Therapeutics, Inc., a BridgeBio company'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Calcilytix Therapeutics, Inc., a BridgeBio company', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}