X-linked Hypophosphatemia Disease Monitoring Program - (XLH-DMP)
The purpose of this disease monitoring program is to collect information on the long-term effects of XLH on children and adults. Special focus will be on skeletal health in children and adult clinical trial patients, growth, balance, mobility, stiffness, pain, quality of life, disability, and difficulties performing activities of daily living.
The XLH-DMP is a long-term prospective outcomes study aiming to collect information on the disease for up to 10 years. Patients not taking burosumab will serve to illustrate the natural history of XLH. The patients who rollover from the clinical trial setting will have at least 2 to 3 years of burosumab treatment and will provide long-term information on safety and effectiveness of burosumab. The patients initiating treatment with burosumab via his/her clinician decision will capture information illustrating the pharmacodynamics and clinical response of burosumab in a real-life setting. Including pediatric patients is important in order to assess and/or confirm long-term effect on growth for which years of observation are necessary to address the impact of burosumab on the near final height. In adult patients, long-term effectiveness in fracture healing and prevention of new fractures or pseudo fractures is also relevant for describing long-term benefits of burosumab treatment.
Participants will be compensated for their participation.
- IRB Number: 1804148239
- Research Study Identifier: TX9006
- Principal Investigator: Erik Imel, MD