Could a transplant drug tame tough vascular birthmarks?
NCT ID NCT00975819
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tested the drug sirolimus (rapamycin) in 61 children and young adults with complicated vascular anomalies—abnormal blood vessel growths that can cause pain, bleeding, or organ problems. The goal was to see if sirolimus could safely shrink these anomalies and improve quality of life. Participants received liquid sirolimus, and doctors measured response using MRI scans, function tests, and quality-of-life surveys.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
sirolimus (also known as rapamycin)
What this could lead to
If successful, sirolimus could offer a safe and effective treatment option to control complicated vascular anomalies and improve quality of life in children and young adults.
What could go wrong
This is a small, single-group Phase 2 study with no placebo comparison. Results may not apply to all types of vascular anomalies, and side effects from sirolimus are possible.
Disclaimer
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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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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.
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Other studies related to the condition(s) this trial covers.
Contacts and locations
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Locations
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Children's Hospital Boston
Boston, Massachusetts, 02115, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States