Vitamin d drug shows promise for rare bone disease without extra phosphate

NCT ID NCT03748966

First seen Oct 31, 2025 · Last updated May 29, 2026 · Updated 36 times

Summary

This early-phase study tested whether a high-dose vitamin D medication (calcitriol) alone could improve blood phosphate levels and bone health in 16 children and adults with X-linked hypophosphatemia (XLH), a genetic condition causing weak bones and poor growth. Participants took calcitriol for one year without the usual phosphate supplements. Researchers measured changes in phosphate levels, kidney function, and rickets severity. The goal was to see if this simpler treatment could work while avoiding kidney damage.

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Contacts and locations

Locations

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

Conditions

Explore the condition pages connected to this study.