First-Ever trial of sapropterin for rare smooth muscle syndrome begins
NCT ID NCT07574034
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests the drug sapropterin (Kuvan) in a single child with multisystem smooth muscle dysfunction syndrome (MSMDS), a rare genetic disorder with no approved treatment. The drug, already approved for another condition, aims to improve growth, blood pressure, and prevent brain complications. Because it involves only one participant, results will be very limited.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- sapropterin (Kuvan)
- What this could lead to
- If it works, this could provide a treatment option for children with MSMDS and support approval for this rare disease.
- What could go wrong
- This is a single-patient study with no control group, so results may not apply to others. The drug is untested for this condition in humans, and risks are unknown.
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
-
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5, Canada