- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00001465
Study of the Disease Process of Lymphangioleiomyomatosis
Characterization of the Pathogenesis of Lymphangioleiomyomatosis (LAM)
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.
Study Overview
Status
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Tatyana Worthy, R.N.
- Phone Number: (301) 827-1376
- Email: worthyt@mail.nih.gov
Study Contact Backup
- Name: Joel Moss, M.D.
- Phone Number: (301) 496-1597
- Email: mossj@nhlbi.nih.gov
Study Locations
-
-
Maryland
-
Bethesda, Maryland, United States, 20892
- Recruiting
- National Institutes of Health Clinical Center
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
- 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.
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
LAM
Patients with tissue diagnosis of LAM may be admitted for evaluation every six months, or as deemed necessary for research
|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
on going
|
Collaborators and Investigators
Investigators
- Principal Investigator: Joel Moss, M.D., National Heart, Lung, and Blood Institute (NHLBI)
Publications and helpful links
General Publications
- Matthew BP, Hasani AM, Chen YC, Pirooznia M, Stylianou M, Rollison SF, Machado TR, Quade NM, Jones AM, Julien-Williams P, Taveira-DaSilva A, Chen MY, Moss J, Wen H. Ultra-Small Lung Cysts Impair Diffusion Without Obstructing Air Flow in Lymphangioleiomyomatosis. Chest. 2021 Jul;160(1):199-208. doi: 10.1016/j.chest.2021.01.077. Epub 2021 Feb 5.
- Hu-Wang E, Schuzer JL, Rollison S, Leifer ES, Steveson C, Gopalakrishnan V, Yao J, Machado T, Jones AM, Julien-Williams P, Moss J, Chen MY. Chest CT Scan at Radiation Dose of a Posteroanterior and Lateral Chest Radiograph Series: A Proof of Principle in Lymphangioleiomyomatosis. Chest. 2019 Mar;155(3):528-533. doi: 10.1016/j.chest.2018.09.007. Epub 2018 Oct 3.
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Respiratory Tract Diseases
- Immune System Diseases
- Neoplasms, Connective and Soft Tissue
- Neoplasms by Histologic Type
- Neoplasms
- Lymphoproliferative Disorders
- Lymphatic Diseases
- Immunoproliferative Disorders
- Congenital Abnormalities
- Pleural Diseases
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Heredodegenerative Disorders, Nervous System
- Neoplastic Syndromes, Hereditary
- Malformations of Cortical Development, Group I
- Malformations of Cortical Development
- Nervous System Malformations
- Lymphangiomyoma
- Lymphatic Vessel Tumors
- Perivascular Epithelioid Cell Neoplasms
- Neurocutaneous Syndromes
- Hamartoma
- Neoplasms, Multiple Primary
- Sclerosis
- Pneumothorax
- Lung Diseases
- Lymphangioleiomyomatosis
- Tuberous Sclerosis
Other Study ID Numbers
- 950186
- 95-H-0186
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Pneumothorax
-
Tabriz University of Medical SciencesAJA University of Medical SciencesUnknownPneumothorax, Spontaneous | Pneumothorax Spontaneous Primary | Pneumothorax, Recurrent | Pneumothorax Spontaneous TensionIran, Islamic Republic of
-
Peking University Third HospitalNot yet recruitingPrimary Spontaneous Pneumothorax
-
Rennes University HospitalRecruiting
-
Dow University of Health SciencesUnknownSecondary PneumothoraxPakistan
-
University Hospital, Strasbourg, FranceRecruiting
-
Chinese University of Hong KongRecruitingPneumothorax, SpontaneousHong Kong
-
Johns Hopkins UniversityRecruitingPneumothorax | Tension PneumothoraxUnited States
-
University of RochesterWithdrawn
-
IsalaRecruitingPneumothorax and Air LeakNetherlands
-
Far Eastern Memorial HospitalNational Taiwan University Hospital; Department of HealthUnknown
Clinical Trials on Toshibia Aquilion One CT
-
National Heart, Lung, and Blood Institute (NHLBI)Recruiting
-
National Heart, Lung, and Blood Institute (NHLBI)TerminatedMyocardial Infarction | Myocardial Ischemia | Acquired Heart Disease | Atherosclerosis | Congenital Heart DiseaseUnited States
-
Johns Hopkins UniversityToshiba America Medical Systems, Inc.CompletedIschemia | Myocardial Ischemia | Heart Diseases | Vascular Diseases | Coronary Artery Disease | Coronary Disease | Arteriosclerosis | Arterial Occlusive DiseasesUnited States, Canada, Singapore, Netherlands, Brazil, Denmark, Germany, Japan
-
Rigshospitalet, DenmarkCompletedPET/CT Imaging of Angiogenesis in Patients With Neuroendocrine Tumors Using 68Ga-NODAGA-E[c(RGDyK)]2Neuroendocrine TumorsDenmark
-
Rigshospitalet, DenmarkCompletedNeuroendocrine TumorsDenmark
-
University of BergenOslo University HospitalCompletedSacroiliac InstabilityNorway
-
Medical University of WarsawRecruitingKnee Osteoarthritis | Knee Arthritis | Joint; Arthrosis, PrimaryPoland
-
Andrew D. Luster, M.D.,Ph.D.National Institute of Allergy and Infectious Diseases (NIAID)Completed
-
National Cancer Institute (NCI)RecruitingStage IIB Mycosis Fungoides and Sezary Syndrome AJCC v8 | Stage III Mycosis Fungoides and Sezary Syndrome AJCC v8 | Stage IV Mycosis Fungoides and Sezary Syndrome AJCC v8United States
-
National Cancer Institute (NCI)RecruitingMalignant Solid Neoplasm | Stage IV Pancreatic Cancer AJCC v8 | Pancreatic Carcinoma | Stage IV Ovarian Cancer AJCC v8 | Ovarian Low Grade Serous AdenocarcinomaUnited States