Saracatinib will be given orally at a dose of 125 milligrams once daily for 9 months. Saracatinib is provided as a pink tablet.

Pulmonary Lymphangioleiomyomatosis

Subjects will receive enough tablets for 90 days +/- 14 days at each visit. Subject will have visits every 90 days for drug accountability as well as safety and efficacy testing to include pulmonary function testing, laboratory testing to include liver and kidney profile, urine pregnancy testing at each visit, vital signs, physical examination - any medically significant changes from baseline visit will be recorded, all adverse events will be monitored until resolution.

saracatinib

Stanford University

Loyola Medical Center

Laboratory of Translational Research NHLBI

University of Cincinnati

Baylor College of Medicine - Ben Taub General Hospital

Safety and Efficacy of Saracatinib In Subjects With Lymphangioleiomyomatosis

Tony Eissa

Principal Investigator

Baylor College of Medicine

Brigham and Women's Hospital

Loyola University

University of South Florida

National Institutes of Health (NIH)

Only one arm: Intervention is Saracatinib. We plan to study three escalating doses of oral saracatinib; 50, 125 and 175 mg. Saracatinib is given orally once a day.
More regarding dose escalation is included in intervention below.

Saracatinib is escalated as follows: Three dose levels will be administered for 1 month each: 50 mg, 125 mg and 175 mg. Three subjects will be treated at the lowest daily dose of 50 mg. If no subject experiences DLT (dose limiting toxicity), the dose level is escalated to 125 mg/day for the next cohort of 3 different subjects and so on to next dose.

Saracatinib

University of Texas Health Science Center-Houston

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

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.

This objective is to generate a safety profile utilizing the data collected along with the adverse events. Subjects will be followed closely during the 4 weeks while taking drug and again for four weeks after stopping the study drug.

explore the efficacy measurements (e.g., pulmonary function test, six minute walk test, and VEGF-D)

The subject is aware of his/her data at the time of the study. They are told if they are/are not using the correct technique for MDI. There is no more data to share with the subject.

lymphangioleiomyomatosis/seizures

Safety and Efficacy of Saracatinib In Subjects With Lymphangioleiomyomatosis

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

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