Could a transplant drug tame tough vascular birthmarks?

NCT ID NCT00975819

Completed Disease control Sponsor: Denise Martin Adams Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for CAPILLARY LYMPHATIC ARTERIAL VENOUS MALFORMATIONS are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

capillary malformation-arteriovenous malformation syndrome kaposiform hemangioendothelioma lymphangiectasis lymphangioma lymphatic malformation microcystic lymphatic malformation multifocal lymphangioendotheliomatosis-thrombocytopenia syndrome PTEN hamartoma tumor syndrome thrombocytopenia tufted angioma venous hemangioma

As listed by the trial registrant

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

CAPILLARY LYMPHATIC ARTERIAL VENOUS MALFORMATIONS CAPILLARY VENOUS LYMPHATIC MALFORMATION KAPOSIFORM HEMANGIOENDOTHELIOMAS LYMPHANGIECTASIA SYNDROMES MICROCYSTIC LYMPHATIC MALFORMATION MUCOCUTANEOUS LYMPHANGIOMATOSIS AND THROMBOCYTOPENIA PTEN OVERGROWTH SYNDROME WITH VASCULAR ANOMALY TUFTED ANGIOMA VENOUS LYMPHATIC MALFORMATION

Contacts and locations

Locations

Children's Hospital Boston

Boston, Massachusetts, 02115, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

More trials for these conditions

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