X-linked Hypophosphatemia (XLH)
Conditions
Brief summary
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.
Interventions
OTHERNo treatment given
No treatment given
Sponsors
Novo Nordisk A/S
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 64 Years
Healthy volunteers
No
Inclusion criteria
* Informed consent obtained before any trial-related activities, i.e., any procedure related to recording of data according to the protocol. * Male or female and aged 18-64 years (both inclusive) at the time of signing informed consent. * Diagnosis of congenital XLH, confirmed genetically or clinically as determined by the investigator, supported by documentation. * Participants have been treated with burosumab for at least 90 days prior to the screening/baseline visit and treatment is still ongoing. * Participant confirms ability and willingness to attend visits according to routine clinical care and mandatory assessments at the site.
Exclusion criteria
* Previous rescreening for this trial. * Current participation in any interventional clinical trial. Participation in another non-interventional trial, such as a patient registry trial, is permitted. * Any condition not associated with XLH, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Average Trough Serum Phosphate Concentration | From baseline to last assessment (Up to 3 years) | 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). |
Countries
Australia, Canada, France, Germany, Italy, Japan, Netherlands, United States
Contacts
STUDY_DIRECTORClinical Transparency dept. 2834
Novo Nordisk A/S
Outcome results
None listed