Trial record 2 of 15 for:    lymphangioleiomyomatosis OR LAM | Open Studies | NIH, U.S. Fed

Study of the Disease Process of Lymphangioleiomyomatosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by National Institutes of Health Clinical Center (CC)
Information provided by:
National Institutes of Health Clinical Center (CC) Identifier:
First received: November 3, 1999
Last updated: March 14, 2014
Last verified: January 2014

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.

Lung Disease
Tuberous Sclerosis

Study Type: Observational
Official Title: Characterization of the Pathogenesis of Lymphangioleiomyomatosis (LAM)

Resource links provided by NLM:

Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 99999999
Study Start Date: September 1995
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.


Ages Eligible for Study:   16 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

General admission criteria for patients include one or both of the following:

Findings on lung biopsy diagnostic of LAM;

Findings on chest x-ray and/or chest computed axial tomography consistent with LAM.

Patients with TSC and pulmonary LAM will be included in the study.

Normal non-smokers in the control group are defined as individuals who have not smoked for greater than or equal to 1 year and have no systemic or pulmonary disease.

Normal smokers defined as individuals with no systemic or pulmonary disease, who have smoked for greater than or equal to 1 year and have normal chest x-ray and normal pulmonary function tests may be included if needed as controls for a similar population of patients with LAM.


Exclusion criteria for patients include:

Age less than 16.

Advanced stage of a pulmonary or a systemic illness in which the risk of the study is judged to be significant even in the absence of a clear contraindication to the procedures.

Exclusion criteria for patients for the formal exercise study and the stress echocardiogram include patients on continuous oxygen. Patients may perform an exercise test that will assess the patient's exercise capacity with activities of daily living.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00001465

Contact: Mary Haughey, R.N. (301) 496-3632
Contact: Joel Moss, M.D. (301) 496-1597

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)    800-411-1222 ext TTY8664111010   
Sponsors and Collaborators
Principal Investigator: Joel Moss, M.D. National Heart, Lung, and Blood Institute (NHLBI)
  More Information

Additional Information:
Publications: Identifier: NCT00001465     History of Changes
Other Study ID Numbers: 950186, 95-H-0186
Study First Received: November 3, 1999
Last Updated: March 14, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Smooth Muscle Proliferation
Tuberous Sclerosis
Lymphangioleiomyomatosis (LAM)

Additional relevant MeSH terms:
Lung Diseases
Tuberous Sclerosis
Congenital Abnormalities
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Immune System Diseases
Immunoproliferative Disorders
Lymphatic Diseases
Lymphatic Vessel Tumors
Lymphoproliferative Disorders
Malformations of Cortical Development
Neoplasms by Histologic Type
Neoplasms, Connective and Soft Tissue
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Nervous System Diseases
Nervous System Malformations
Neurocutaneous Syndromes
Neurodegenerative Diseases
Pathologic Processes
Perivascular Epithelioid Cell Neoplasms
Pleural Diseases
Respiratory Tract Diseases processed this record on October 23, 2014