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The purpose of this study is to better understand how X-linked hypophosphatemia (XLH) affects the body and daily life. Phosphate levels are critical in managing XLH, we aim to study how these levels change in patients taking burosumab and how they relate to bone health, overall disease burden and XLH-related conditions.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adults with XLH | Adults with XLH, confirmed genetically or clinically as determined by the investigator, who have been treated with burosumab for at least 90 days prior to the screening/baseline visit and whose treatment is still ongoing will be followed for a maximum of three years, continuing until end of trial visit, withdrawal of consent or loss to follow-up, whichever occurs first. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No treatment given | Other | No treatment given |
|
| Measure | Description | Time Frame |
|---|---|---|
| Average Trough Serum Phosphate Concentration | Average trough serum phosphate concentration, defined as the area under the concentration-time curve (AUC) divided by the total duration of the assessment period (AUC divided by time). Measured in milligrams per deciliter (mg/dL). | From baseline to last assessment (Up to 3 years) |
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Inclusion Criteria:
Exclusion Criteria:
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Adults with XLH, confirmed genetically or clinically as determined by the investigator, who have been treated with burosumab for at least 90 days prior to the screening/baseline visit and whose treatment is still ongoing. Participants will be followed for a maximum of three years, continuing until end of trial visit, withdrawal of consent or loss to follow-up, whichever occurs first.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Novo Nordisk | Contact | (+1) 866-867-7178 | clinicaltrials@novonordisk.com |
| Name | Affiliation | Role |
|---|---|---|
| Clinical Transparency dept. 2834 | Novo Nordisk A/S | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yale University School of Medicine | New Haven | Connecticut | 06510 | United States | ||
| Indiana University Hospital |
According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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| Indianapolis |
| Indiana |
| 46202-5271 |
| United States |
| The Ohio State University Wexner Medical Center (OSUWMC) - CarePoint East | Columbus | Ohio | 43203 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232-8148 | United States |
| Northern Sydney Cancer Centre-Royal North Shore Hospital (RNSH) | St Leonards | New South Wales | 2065 | Australia |
| Westmead Hospital | Westmead | New South Wales | 2145 | Australia |
| Monash University - Monash Medical Centre (MMC) | Clayton | Victoria | 3168 | Australia |
| Bone Research and Education Centre | Oakville | Ontario | L6M 1M1 | Canada |
| CHU de Bicetre | Le Kremlin | 94275 | France |
| Hopital Cochin - APHP | Paris | 75014 | France |
| Charite Campus Virchow | Berlin | 13353 | Germany |
| Endokrinologikum Goettingen | Göttingen | 37075 | Germany |
| Universitat Wurzburg Koenig-Ludwig-Haus | Wuezburg | 97074 | Germany |
| Office of Francesco Di Costanzo, MD | Sesto Fiorentino | Florence | 50019 | Italy |
| Istituto Auxologico Italiano | Milan | 20145 | Italy |
| Azienda Ospedaliera di Padova - U.O.C. di Gastroenterologia | Padova | 35128 | Italy |
| Fondazione Policlinico Universitario Campus Bio-Medico | Rome | 00128 | Italy |
| Fukuoka University - Fukuoka University Hospital | Fukuoka | Fukuoka | 814-0180 | Japan |
| Okayama Saiseikai Outpatient Center Hospital | Okayama | Okayama-ken | 700-0013 | Japan |
| The University of Tokyo Hospital | Bunkyo-ku | Tokyo | 113-8655 | Japan |
| Iseikai International General Hospital | Osaka | 530-0052 | Japan |
| Japan Community Healthcare Organization Osaka Hospital | Osaka | 553-0003 | Japan |
| Leiden University Medical Center (Leids Universitair Medisch Centrum (LUMC)) | Leiden | 2333 ZA | Netherlands |
| ID | Term |
|---|---|
| D053098 | Familial Hypophosphatemic Rickets |
| ID | Term |
|---|---|
| D063730 | Rickets, Hypophosphatemic |
| D012279 | Rickets |
| D001851 | Bone Diseases, Metabolic |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D007015 | Hypophosphatemia, Familial |
| D015499 | Renal Tubular Transport, Inborn Errors |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D002128 | Calcium Metabolism Disorders |
| D017674 | Hypophosphatemia |
| D010760 | Phosphorus Metabolism Disorders |
| D014808 | Vitamin D Deficiency |
| D001361 | Avitaminosis |
| D003677 | Deficiency Diseases |
| D044342 | Malnutrition |
| D009748 | Nutrition Disorders |
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