Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies

Microcystic Lymphatic Malformation Clinical Trial

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

Status Active, not recruiting

Clinical Trial 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)

Clinical Trial 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. ;

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Capillary Lymphatic Arterial Venous Malformations
• Capillary Venous Lymphatic Malformation
• Congenital Abnormalities
• Hemangioendothelioma
• Kaposiform Hemangioendotheliomas
• Kasabach-Merritt Syndrome
• Lymphangiectasia Syndromes
• Lymphatic Abnormalities
• Microcystic Lymphatic Malformation
• Mucocutaneous Lymphangiomatosis and Thrombocytopenia
• PTEN Overgrowth Syndrome With Vascular Anomaly
• Sarcoma, Kaposi
• Syndrome
• Thrombocytopenia
• Thrombocytosis
• Tufted Angioma
• Vascular Malformations
• Venous Lymphatic Malformation

• NCT number: NCT00975819
• Study type: Interventional
• Source: Children's Hospital Medical Center, Cincinnati
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: October 2009
• Completion date: October 2019