Ignite Creation Date:
2025-12-25 @ 3:20 AM
Ignite Modification Date:
2026-03-02 @ 3:44 PM
Study NCT ID:
NCT03651505
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-31
First Post:
2018-08-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
X-linked Hypophosphatemia Disease Monitoring Program
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D053098', 'term': 'Familial Hypophosphatemic Rickets'}, {'id': 'D063730', 'term': 'Rickets, Hypophosphatemic'}, {'id': 'D017674', 'term': 'Hypophosphatemia'}], 'ancestors': [{'id': 'D012279', 'term': 'Rickets'}, {'id': 'D001851', 'term': 'Bone Diseases, Metabolic'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D007015', 'term': 'Hypophosphatemia, Familial'}, {'id': 'D015499', 'term': 'Renal Tubular Transport, Inborn Errors'}, {'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': 'D008664', 'term': 'Metal Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D002128', 'term': 'Calcium Metabolism Disorders'}, {'id': 'D010760', 'term': 'Phosphorus Metabolism Disorders'}, {'id': 'D014808', 'term': 'Vitamin D Deficiency'}, {'id': 'D001361', 'term': 'Avitaminosis'}, {'id': 'D003677', 'term': 'Deficiency Diseases'}, {'id': 'D044342', 'term': 'Malnutrition'}, {'id': 'D009748', 'term': 'Nutrition Disorders'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 782}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2018-07-16', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2032-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-29', 'studyFirstSubmitDate': '2018-08-24', 'studyFirstSubmitQcDate': '2018-08-28', 'lastUpdatePostDateStruct': {'date': '2025-10-31', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2018-08-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2032-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Long-Term Safety of Burosumab', 'timeFrame': '10 years', 'description': 'To assess the long-term safety of burosumab treatment in adult and pediatric patients with XLH, including overall renal health, the presence and/or progression of nephrocalcinosis and spinal stenosis, and pregnancy outcomes.'}, {'measure': 'Long-Term Effectiveness of Burosumab', 'timeFrame': '10 years', 'description': 'To evaluate the long-term effectiveness of burosumab treatment on key manifestations of XLH, including skeletal health, stiffness, mobility and physical functioning.'}, {'measure': 'Clinical Course of XLH Disease', 'timeFrame': '10 years', 'description': 'To illustrate the clinical, radiological, biochemical manifestations and progression of XLH over time in both untreated and treated patients with XLH.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['X-linked Hypophosphatemia', 'Fibroblast growth factor 23 (FGF23)', 'KRN23', 'XLH', 'Hypophosphatemia', 'Familial Hypophosphatemic Rickets', 'FGF23', 'Crysvita'], 'conditions': ['X-linked Hypophosphatemia', 'Hypophosphatemic Rickets']}, 'referencesModule': {'references': [{'pmid': '40962182', 'type': 'DERIVED', 'citation': 'Carpenter TO, Fukumoto S, Haffner D, Imel EA, Ozono K, Ishii H, Li Z, Sandilands K, Joos-Vandewalle P, Lee C, Kanematsu M, McCullough KP, Brandi ML. Advancing Patient Evidence in XLH (APEX): Baseline analysis of a global data unification program. Bone. 2025 Dec;201:117649. doi: 10.1016/j.bone.2025.117649. Epub 2025 Sep 15.'}], 'seeAlsoLinks': [{'url': 'http://www.ultragenyx.com/our-research/our-clinical-trial-transparency-commitment/', 'label': 'Ultragenyx Transparency Commitment'}]}, 'descriptionModule': {'briefSummary': 'The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.', 'detailedDescription': 'The XLH-DMP is a global, prospective, multicenter, longitudinal, long-term outcomes program for subjects on or off any treatment designed to characterize XLH disease presentation and progression, assess long-term safety and effectiveness of burosumab, as well as prospectively investigate longitudinal change over time across biomarker(s), clinical assessments, and patient/caregiver-reported outcome measures in a representative population. The XLH-DMP will collect demographic, biochemical, physiologic, disease severity, and progression data in patients taking burosumab and those not taking burosumab. In this DMP, patients will only have access to burosumab through authorized prescribed use. The Sponsor will not provide any treatments as part of the DMP.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Approximately 800 eligible adult and pediatric patients with XLH will be enrolled globally, with a minimum of 200 pediatric patients. Patients can enter the XLH-DMP regardless of how their XLH is being treated. Patients on Crysvita (burosumab) via prescription may begin taking Crysvita, per standard of care before or after enrolling in the XLH DMP.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Willing and able to provide informed consent or, in the case of patients under the age of 18 years (or 16 years, depending on the region), provide assent (if required) and informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.\n* Clinical diagnosis of XLH based on family history, OR confirmed PHEX mutation, OR biochemical profile consistent with XLH.\n* Willing and able to comply with the study visit schedule and study procedures.\n\nExclusion Criteria:\n\n* Concurrent enrollment in an Ultragenyx-sponsored clinical trial is NOT permitted.\n* Serious medical or psychiatric comorbidity.\n* Less than one year of life expectancy.'}, 'identificationModule': {'nctId': 'NCT03651505', 'briefTitle': 'X-linked Hypophosphatemia Disease Monitoring Program', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ultragenyx Pharmaceutical Inc'}, 'officialTitle': 'X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)', 'orgStudyIdInfo': {'id': 'UX023-CL401'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Prior Burosumab Clinical Trial Participants', 'description': 'Patients who participated in burosumab clinical trials and continue to receive burosumab via prescription from their physician.', 'interventionNames': ['Other: No intervention']}, {'label': 'Not from Prior Burosumab Clinical Trial', 'description': 'Patients may take other treatments for XLH and may start burosumab treatment at any time as prescribed by a physician.', 'interventionNames': ['Other: No intervention']}], 'interventions': [{'name': 'No intervention', 'type': 'OTHER', 'description': 'Access to any treatment is through authorized commercial use and not as a part of this DMP', 'armGroupLabels': ['Not from Prior Burosumab Clinical Trial', 'Prior Burosumab Clinical Trial Participants']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': "Children's Hospital of Alabama", 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '92123', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': "Rady Children's Hospital San Diego", 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '94158', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital of Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '06520', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale University School of Medicine', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '46202-5167', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University School of Medicine', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '21218', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins University', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '27708', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'The Ohio State University Wexner Medical Center', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '37212', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Vanderbilt University - Department of Medicine', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Vanderbilt University - Department of Pediatrics', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Houston Methodist Research Institute', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas, Health Science Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '22903', 'city': 'Charlottesville', 'state': 'Virginia', 'country': 'United States', 'facility': 'University of Virgina School of Medicine', 'geoPoint': {'lat': 38.02931, 'lon': -78.47668}}, {'zip': '23507', 'city': 'Norfolk', 'state': 'Virginia', 'country': 'United States', 'facility': "Children's Hospital of The King's Daughter", 'geoPoint': {'lat': 36.84681, 'lon': -76.28522}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '53792', 'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'facility': "American Family Children's Hospital", 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}, {'zip': 'C1270AAN', 'city': 'Buenos Aires', 'state': 'Buenos Aires F.D.', 'country': 'Argentina', 'facility': 'Hospital General de Niños Pedro de Elizalde', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'zip': 'C1425EFD', 'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Hospital de Niños Dr. Ricardo Gutierrez', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Córdoba', 'country': 'Argentina', 'facility': 'Hospital Privado Universitario de Córdoba', 'geoPoint': {'lat': -31.40648, 'lon': -64.18853}}, {'zip': '58400-398', 'city': 'São José', 'state': 'Campina Grande - PB', 'country': 'Brazil', 'facility': 'Hospital Universitário Alcides Carneiro (HUAC)', 'geoPoint': {'lat': -27.61528, 'lon': -48.6275}}, {'zip': '05437-000', 'city': 'São Paulo', 'state': 'São Paulo', 'country': 'Brazil', 'facility': 'Instituto de Medicina Avancada (IMA Brasil)', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'zip': '80030-110', 'city': 'Curitiba', 'country': 'Brazil', 'facility': 'CETI - Centro de Estudos em Terapias Inovadoras', 'geoPoint': {'lat': -25.42778, 'lon': -49.27306}}, {'zip': '05403-000', 'city': 'São Paulo', 'country': 'Brazil', 'facility': 'Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'zip': 'V6H 3N1', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': "British Columbia Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'ON L6M 1M1', 'city': 'Oakville', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Bone Research and Education Centre', 'geoPoint': {'lat': 43.45011, 'lon': -79.68292}}, {'zip': 'H4A 0A9', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': "Shiner's Hospital for Children - Canada", 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': 'K1H 5B2', 'city': 'Ottawa', 'country': 'Canada', 'facility': "Children's Hospital Eastern Ontario Research Institute", 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'city': 'Santiago', 'state': 'Santiago Metropolitan', 'country': 'Chile', 'facility': 'Pontificia Universidad Católica de Chile', 'geoPoint': {'lat': -33.45694, 'lon': -70.64827}}, {'city': 'Medellín', 'state': 'Antioquia', 'country': 'Colombia', 'facility': 'Hospital Universitario de San Vicente Fundación', 'geoPoint': {'lat': 6.245, 'lon': -75.57151}}, {'city': 'Bogotá', 'state': 'Cundinamarca', 'country': 'Colombia', 'facility': 'Hospital Infantil Universitario de San José'}], 'overallOfficials': [{'name': 'Ultragenyx Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Ultragenyx Pharmaceuticals Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ultragenyx Pharmaceutical Inc', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Kyowa Kirin Co., Ltd.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}