Understanding the disease process of lymphangioleiomyomatosis
Characterization of the Pathogenesis of Lymphangioleiomyomatosis (LAM)
This study looks at how lymphangioleiomyomatosis affects the lungs in women and tries to find out what causes the disease to help develop better treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 2000 (estimated) |
| Ages | 16 Years to 100 Years |
| Sex | All |
| Sponsor | National Institutes of Health Clinical Center (CC) NIH |
| Drugs / interventions | radiation |
| Locations | 1 site (Bethesda, Maryland) |
| Trial ID | NCT00001465 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on pulmonary lymphangioleiomyomatosis (LAM), a severe lung disease primarily affecting women of childbearing age. The research aims to define the clinical course of LAM and investigate its underlying cellular and molecular mechanisms. By identifying specific proteins and genes involved in lung destruction, the study seeks to pave the way for more effective therapies. Participants will include individuals diagnosed with LAM and healthy controls for comparison.
Who should consider this trial
Good fit: Ideal candidates for this study are women diagnosed with pulmonary lymphangioleiomyomatosis or tuberous sclerosis.
Not a fit: Patients with other lung diseases or those who do not meet the diagnostic criteria for LAM may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to the development of more effective therapies for patients suffering from lymphangioleiomyomatosis.
How similar studies have performed: While this study explores the disease process of LAM, similar studies have shown promise in understanding other lung diseases at the molecular level.
Eligibility criteria
Show full inclusion / exclusion criteria
* 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.
Where this trial is running
Bethesda, Maryland
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)
Study contacts
- Principal investigator: Joel Moss, M.D. — National Heart, Lung, and Blood Institute (NHLBI)
- Study coordinator: Tatyana A Worthy, R.N.
- Email: worthyt@mail.nih.gov
- Phone: (301) 827-1376
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.