Can a drug for one bone disease help another? a Rare-Disorder trial puts it to the test
NCT ID NCT03993821
First seen Jul 30, 2026 · Last updated Jul 31, 2026 · Updated 1 time
Summary
This early-phase trial is testing whether burosumab, a drug already approved for X-linked hypophosphatemia (XLH), can safely raise phosphate levels in a single patient with cutaneous skeletal hypophosphatemia syndrome (CSHS). Both conditions involve abnormally high FGF23 activity leading to low phosphate. The patient will receive burosumab injections over 52 weeks, with the main goal of measuring changes in blood phosphate levels.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- burosumab (Crysvita), an antibody that blocks FGF23 to raise phosphate levels
- What this could lead to
- If successful, burosumab could offer a treatment option for people with CSHS, a rare condition with no approved therapies.
- What could go wrong
- This is a very early, single-patient trial, so results may not apply to others. Burosumab is not yet approved for CSHS, and side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States