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Brief Title: Study of the Disease Process of Lymphangioleiomyomatosis
Official Title: Characterization of the Pathogenesis of Lymphangioleiomyomatosis (LAM)
Study ID: NCT00001465
Brief Summary: Pulmonary lymphangioleiomyomatosis (LAM) is a destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy for the disease and the prognosis is poor. This study is designed to determine the disease processes involved at the level of cells and molecules, in order to develop more effective therapy. Researchers intend to identify the proteins and genes that contribute to the process of lung destruction in affected individuals.
Detailed Description: Individuals with pulmonary lymphangioleiomyomatosis develop severe destructive lung disease. Most of them are females of childbearing age. Currently, there is no proven effective therapy and the prognosis is variable. This study is designed to (a) define the clinical course of the disease and (b) elucidate the pathogenesis of the disease at the cellular and molecular levels, in order to develop more effective therapy. To accomplish this, we intend to identify the proteins and genes that contribute to the process of lung destruction in affected individuals.
Minimum Age: 16 Years
Eligible Ages: CHILD, ADULT, OLDER_ADULT
Sex: ALL
Healthy Volunteers: No
National Institutes of Health Clinical Center, Bethesda, Maryland, United States
Name: Joel Moss, M.D.
Affiliation: National Heart, Lung, and Blood Institute (NHLBI)
Role: PRINCIPAL_INVESTIGATOR