Study of the Disease Process of Lymphangioleiomyomatosis
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT00001465 |
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Recruitment Status :
Recruiting
First Posted : November 4, 1999
Last Update Posted : May 23, 2023
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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.
| Condition or disease | Intervention/treatment |
|---|---|
| Lung Disease Pneumothorax Tuberous Sclerosis Lymphangioleiomyomatosis | Device: Toshibia Aquilion One CT |
| Study Type : | Observational |
| Estimated Enrollment : | 2000 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | Characterization of the Pathogenesis of Lymphangioleiomyomatosis (LAM) |
| Actual Study Start Date : | December 18, 1995 |
| Group/Cohort | Intervention/treatment |
|---|---|
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LAM
Patients with tissue diagnosis of LAM may be admitted for evaluation every six months, or as deemed necessary for research
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Device: Toshibia Aquilion One CT
The Toshiba Aquilion ONE CT system is currently being used for studies in both general CT radiology and CT cardiac imaging. One of the unique aspects of the Aquilion ONE CT system is its ability to acquire whole organ volume images in a single rotation by utilizing an x-ray detector that is configured as 320 detector rows with a 0.5 mm width, providing a z-axis coverage of 16 cm of anatomy. In line with the evolutionary changes to CT systems, the Aquilion ONE will be upgraded with new technology that will expand its capabilities. The changes being made to the Aquilion ONE will provide enhancements to image acquisition capabilities, reduce ionizing radiation dose, and improve subject access to the system. All of these features assist in enhancing the safety of the currently installed Aquilion ONE CT system. |
- Define the clinical course of the disease LAM andelucidate the pathogenesis of LAM at cellular and molecular levels [ Time Frame: on going ]To define the molecular basis of the remarkable proliferation of immature appearing smooth muscle cells, which is the cause of many of the clinical manifestations, and perhaps thereby to improve our understanding of the mechanism of smooth muscle cell proliferation in other diseases, e.g., interstitial lung diseases, asthma, atherosclerosis, hypertension, and post-angioplastic coronary restenosis. To assess the contribution of proteins and genetic factors to cyst formation, airway obstruction, and clinical course. To evaluate the role of TSC genes in the pathogenesis of LAM.
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, Learn About Clinical Studies.
| Ages Eligible for Study: | 16 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
- INCLUSION CRITERIA:
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.
Pregnant and or nursing women can be included in accordance with Federal Regulations at Subpart B of 45 CFR 46 Subjects who are pregnant and or nursing will be excluded from procedures during their pregnancy that are greater than minimal risk, until they are no longer pregnant and/or nursing. Procedures that will not be completed while the subject is pregnant and/or nursing including: PFTs, Six Minute Walk Test, thoracentesis, bronchoscopy, and measurements with imaging modalities requiring contrast or with radiation exposure such as Chest x-ray, CT scan, MRI, bone densitometry (DEXA). Allowing subjects to be included in the study may glean important information about individuals with uncommon pulmonary disease during and post pregnancy.
EXCLUSION CRITERIA:
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00001465
| Contact: Tatyana Worthy, R.N. | (301) 827-1376 | worthyt@mail.nih.gov | |
| Contact: Joel Moss, M.D. | (301) 496-1597 | mossj@nhlbi.nih.gov |
| United States, Maryland | |
| National Institutes of Health Clinical Center | Recruiting |
| Bethesda, Maryland, United States, 20892 | |
| Principal Investigator: | Joel Moss, M.D. | National Heart, Lung, and Blood Institute (NHLBI) |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | National Heart, Lung, and Blood Institute (NHLBI) |
| ClinicalTrials.gov Identifier: | NCT00001465 |
| Other Study ID Numbers: |
950186 95-H-0186 |
| First Posted: | November 4, 1999 Key Record Dates |
| Last Update Posted: | May 23, 2023 |
| Last Verified: | May 8, 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Smooth Muscle Proliferation Bronchoscopy Female |
Pneumothorax Tuberous Sclerosis Natural History |
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Tuberous Sclerosis Lymphangioleiomyomatosis Lung Diseases Pneumothorax Sclerosis Pathologic Processes Respiratory Tract Diseases Pleural Diseases Hamartoma Neoplasms Neoplasms, Multiple Primary Neoplastic Syndromes, Hereditary Malformations of Cortical Development, Group I Malformations of Cortical Development Nervous System Malformations |
Nervous System Diseases Neurocutaneous Syndromes Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Congenital Abnormalities Genetic Diseases, Inborn Lymphangiomyoma Lymphatic Vessel Tumors Neoplasms by Histologic Type Perivascular Epithelioid Cell Neoplasms Neoplasms, Connective and Soft Tissue Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases |