Could a cream replace pills for painful birthmarks? early trial hints at possibility

NCT ID NCT04172922

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This early-phase study tested a sirolimus (rapamycin) cream applied directly to skin lesions in children with vascular anomalies. The goal was to see if the cream could safely reduce pain, bleeding, and lesion size without the side effects of oral medication. Only 5 children were enrolled, and the study was terminated early, so the results are very preliminary.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

topical sirolimus (rapamycin) cream

What this could lead to

If it works, this could offer a safer, skin-only treatment for painful or bleeding vascular lesions in children, avoiding the side effects of oral medication.

What could go wrong

This was a very small, early-phase trial that was terminated early, so results are limited. Topical sirolimus may not work for all types of vascular anomalies, and long-term safety is not yet known.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

vascular malformation

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Nemours Children's Health, Jacksonville

    Jacksonville, Florida, 32207, United States

  • Nemours Children's Hospital, Florida

    Orlando, Florida, 32827, United States