Weekly pill could tame painful birthmarks with fewer side effects
NCT ID NCT04861064
First seen Mar 11, 2026 · Last updated Jun 23, 2026 · Updated 16 times
Summary
This study tests whether taking the drug sirolimus just once a week can safely treat venous and lymphatic malformations—abnormal clusters of blood vessels or lymph vessels that can cause pain and swelling. Current daily dosing works but often causes side effects like low white blood cell counts and mouth sores. The trial will enroll 24 people aged 2 and older, who will take sirolimus weekly for 6 months, with the option to continue longer. Researchers will measure changes in lesion size and track side effects to see if weekly dosing is a good alternative.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Medical University of South Carolina
RECRUITINGCharleston, South Carolina, 29403, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Sirolimus (a drug that slows abnormal cell growth)
What this could lead to
If it works, this could offer a safer, more convenient treatment option for people with venous and lymphatic malformations, with fewer side effects than daily dosing.
What could go wrong
This is a small, early-phase trial with only 24 participants. The weekly dosing may not be as effective as daily dosing, and side effects like infections or mouth sores are still possible.
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.