NCT00975819

Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies

A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 61 (actual)
• Sponsor: Denise Martin Adams · Academic / Other
• Sex: All
• Age: 31 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine if the use of sirolimus in the treatment of children and young adults with complicated vascular anomalies will prove to be safe and provide objective response resulting in improved clinical status and quality of life. Funding Source - FDA OOPD (Food and Drug Administration - Office of Orphan Products Development)

Detailed description

Patients with vascular anomalies (VA) have a spectrum of diseases that can be broadly classified into vascular tumors and malformations. Complicated vascular anomalies can cause disfigurement, chronic pain, and organ dysfunction with significant morbidity and mortality. Despite the severity of potential complications, we lack uniform guidelines for the treatment and response to treatment of children and young adults with these diseases. There are pre-clinical and clinical data supporting the essential regulatory function of the PI3K/Akt/mTOR pathway in vascular growth and organization, and suggest a therapeutic target for patients with complicated vascular anomalies. The overall goal of this trial is to objectively determine the effectiveness and safety of the mTOR inhibitor Rapamycin\* in the treatment of children and young adults diagnosed with complicated vascular anomalies. We propose a Phase 2 trial with the diagnostic, therapeutic and response criteria experimentally determined in this study used as a framework for future Phase 3 clinical trials.

Conditions

• Kaposiform Hemangioendotheliomas
• Tufted Angioma
• Capillary Venous Lymphatic Malformation
• Venous Lymphatic Malformation
• Microcystic Lymphatic Malformation
• Mucocutaneous Lymphangiomatosis and Thrombocytopenia
• Capillary Lymphatic Arterial Venous Malformations
• PTEN Overgrowth Syndrome With Vascular Anomaly
• Lymphangiectasia Syndromes

Interventions

Type	Name	Description
DRUG	sirolimus	liquid dosing based on trough levels

Timeline

• Start date: 2009-10-01
• Primary completion: 2014-03-01
• Completion: 2020-10-01
• First posted: 2009-09-11
• Last updated: 2026-01-06
• Results posted: 2026-01-06

Locations

2 sites across 1 country: United States