Clinical Trial: The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)

Brief Summary:

Lymphangioleiomyomatosis (LAM) is a rare lung disease that mostly affects women of childbearing age. In LAM, abnormal, muscle-like cells begin to grow out of control in the lungs. As a result, air can't move freely in and out of the lungs. In some cases, this means the lungs can't supply the body's other organs with enough oxygen.

This study is being conducted to find out what dose of a drug called saracatinib is best tolerated by people with LAM. This drug has been tested in patients with certain types of cancer but is not currently approved by the United States Food and Drug Administration (FDA). Saracatinib may work in cancer by preventing the growth, movement and invasiveness of cancer cells. The use of saracatinib to treat LAM is considered experimental. Preliminary testing already completed suggests that the study drug, saracatinib, may suppress certain substances in the lungs of patients with LAM thus may be effective in slowing down the disease process

Detailed Summary:

Tuberous sclerosis complex (TSC) is an autosomal dominant disorder caused by mutations in tuberous sclerosis complex 1 (TSC1) or tuberous sclerosis complex 2 (TSC2) tumor suppressor genes. TSC is characterized by tumors in a wide range of tissues, seizures, mental retardation, autism, and organ failure. Lymphangioleiomyomatosis (LAM), the major pulmonary manifestation in women with TSC, is a progressive lung disease characterized by infiltration of atypical smooth muscle like cells and formation of cysts.

The long term goal of this research is to devise novel therapeutic strategies for patients with LAM. Our preliminary data reveal an increase in active Src in lung tissues of patients with LAM as well as in laboratory cultured cells.

The focus of this study is to examine if Src inhibition represents a potential therapeutic strategy in LAM. In this study, we will evaluate the safety, tolerability of Src inhibition in subjects with LAM.

The Quality Assurance (QA) plan is put forth in the Manual of Operations for this study. This study is utilizing the services of the Data Coordinating Center (DCC) at the University of Southern Florida. The data collected and entered in the electronic clinical research form (CRF) at each site will be reviewed by the site clinical research associate and the DCC data manager. Data check will be made throughout the study for all 3 sites and for all patients enrolled. Data checks to compare data entered into the registry against predefined rules for range or consistency with other data fields in the registry.

Source data verification will be done to assess the accuracy, completeness, or representativeness of registry data. This will be done by a DCC Clinical Research Associate (CRA).

One of three escalating daily oral doses of saracatinib; 50, 125 and 175 mg. Saracatinib will be given orally once a day for four weeks. Adverse events will be monitored. The subject will receive only one of the doses as determined by the escalation of the study.