Weekly pill could tame painful birthmarks with fewer side effects

NCT ID NCT04861064

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

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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

  • Medical University of South Carolina

    RECRUITING

    Charleston, South Carolina, 29403, United States

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Other studies related to the condition(s) this trial covers.