Rapamycin (Sirolimus) Safety in Complicated Vascular Anomalies
Rapamycin is also known as Sirolimus- it is an agent with known anti-proliferative activity. It has been shown to slow the progression of tumor cells. The purpose of this study is to determine if Rapamycin is safe and effective in young adults and children with complicated vascular anomalies. (KHE, Tufted Angioma, LM)
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.
Diagnosis of one of the following vascular anomalies: KHE with our without Kasabach Merritt Phenomenon, Tufted Angioma with or without Kasabach Merritt Phenomenon, Capillary Venous Lymphatic Malformation (CVLM), Venous Lymphatic Malformation (VLM), Microcystic Lymphatic Malformation (MLM), Mucocutaneous Lymphangiomatosis and Thrombocytopenia (MLT), Capillary Lymphatic Arterial Venous Malformation (CLAVM), PTEN Overgrowth syndrome with vascular anomaly, Lymphangietasia Syndromes.
Patients must have vascular anomalies that have the potential to cause significant morbidity: coagulopathy, chronic pain, recurrent cellulitis, ulceration, visceral or bone involvment, cardiac dysfunction.
Patients must be 0-31 years of age at study entry.
For more information contact:
Mary Sue Wetzel, RN at CCHMC, 513-636-0944, email@example.com
Matthew Alen at CCHMC 513-803-2082, firstname.lastname@example.org