Safety and Efficacy of Saracatinib In Subjects With Lymphangioleiomyomatosis
This study is being done to determine if there is a potential benefit of saracatinib in LAM subjects. Based on the information of this trial, additional clinical development trials will be needed. The study will also test the tolerability of 125 mg of saracatinib given once daily over a 9 month period.
• Female patients. It should be noted that LAM occurs almost exclusively in women.
• 18 to 65 years of age.
• All patients must have a diagnosis of LAM as defined by compatible cystic changes on chest computed tomography (CT) and one of the following:
• Open lung, transbronchial or thoracic needle biopsy consistent with LAM
• Open or needle abdominal biopsy findings consistent with LAM
• Clinical findings of tuberous scleroma complex (TSC), renal angiomyolipoma, cystic abdominal lymphangiomas, or history of chylous effusion in the chest or abdomen
• Serum vascular endothelial growth factor D (VEGF-D) \> 800 pg/ml
• Subjects must have had a recent reduction in forced expiratory volume at 1-second (FEV1) of \> 50ml/year, as shown by at least two pulmonary function testing (PFT) measured at least 6 months apart in the last 24 months prior to enrolling study.