Lymphangioleiomyomatosis (LAM)
Conditions
Sponsors
Frank McCormack, M.D., University of Cincinnati Medical Center Director, Division of Pulmonary and Critical Care Medicine
a.Alan F. Barker, M.D. - Oregon Health & Sciences University
b.Kevin Brown, M.D., - National Jewish Medical & Research Center
c.Edwin K. Silverman, M.D., Ph.D. - Harvard/Brigham & Women's Hospital
d.James M. Stocks, M.D. - University of Texas Health Center
e.James K. Stoller, M.D. - Cleveland Clinic Foundation
f.Charlie Strange, M.D. - Medical University of South Carolina
g.Bruce Trapnell, M.D.-Cincinnati Children's Medical Center
h.Mark Brantly, M.D.-University of Florida, Gainesville
i.Y
Eligibility
Sex/Gender
Female
Inclusion criteria
Inclusion criteria: 1 Age 18 or over 2 Signed and dated informed consent 3 Diagnosis of LAM as determined by biopsy; or chest CT scan findings compatible with LAM in the setting of tuberous sclerosis, angiomyolipoma or chylous pleural effusion 4 Abnormal lung function i.e., postbronchodilator FEV1 less than or equal to 70% of predicted
Exclusion criteria
Exclusion criteria: 1 History of myocardial infarction or stroke related to atherosclerosis 2 Pregnancy or breast feeding 3 Inadequate contraception 4 Significant hematologic or hepatic abnormality 5 Intercurrent infection at initiation of sirolimus 6 Recent surgery (involving entry into a body cavity) within 2 months of initiation of sirolimus 7 Use of an investigational drug within the last 30 days 8 Uncontrolled hyperlipidemia 9 Previous lung transplantation 10 Inability to attend scheduled clinic visits 11 Inability to give informed consent 12 Inability to perform pulmonary function testing 13 Creatinine > 2.5 mg/dl 14 Chylous ascites sufficient to affect diaphragmatic function 15 Pleural effusion sufficient to affect pulmonary function 16 Acute pneumothorax within the past 2 months 17 Malignancy other than uncomplicated skin cancer in the past 2 years 18 Previous or current use of estrogen therapy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| FEV1 response | — |
Secondary
| Measure | Time frame |
|---|---|
| FVC response, six minute walk, diffusing capacity, lung volumes, dyspnea (shortness of breath) scores by questionnaire, mortality, 3 dimensional CT, adverse events | — |
Countries
Canada, Japan, United States
Contacts
Public ContactKoh Nakata, M.D., Ph.D.
Bioscience Medical Research Center (BMRC), Niigata University Medical & Dental Hospital
Outcome results
None listed