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Calcitriol Monotherapy for X-Linked Hypophosphatemia
Sponsor: Massachusetts General Hospital
Summary
Children and adults with XLH recruited will be treated with calcitriol alone (without phosphate supplementation) for one year, during which the calcitriol dose will be escalated during the first 3 months of therapy. The investigators hypothesize that treatment of adults and children with XLH alone will improve serum phosphate levels and skeletal mineralization without causing an increase in kidney calcifications. The study will also examine if calcitriol therapy will improve growth in children.
Official title: Calcitriol Monotherapy for X-Linked Hypophosphatemia: Effects on Mineral Ions, Growth and Skeletal Parameters
Key Details
Gender
All
Age Range
3 Years - 70 Years
Study Type
INTERVENTIONAL
Enrollment
20
Start Date
2019-03-28
Completion Date
2026-05
Last Updated
2025-10-01
Healthy Volunteers
No
Conditions
Interventions
Calcitriol
Adults and children (age 3-17) with X-linked hypophosphatemia will be treated with calcitriol therapy without phosphate supplementation. Doses of calcitriol will be escalated and optimized in the first three months of the study. Calcitriol is an oral medication taken once a day.
Locations (1)
Massachusetts General Hospital
Boston, Massachusetts, United States