Study:
Lymphangioleiomyomatosis Efficacy and Safety Trial
Rationale:
n/a
Purpose:
Lymphangioleiomyomatosis (LAM) is a rare lung disease that is caused by genetic mutations.
It results in the uncontrolled growth and proliferation of an unusual type of smooth muscle
cell. These cells invade lung tissue, including the airways, blood vessels, and lymph
vessels, and restrict the flow of air, blood, and lymph, respectively. Respiratory failure,
lung collapse (pneumothorax), and pleural effusions (chylothorax) are hallmarks of the
disease. This study will evaluate the safety and effectiveness of sirolimus, an
immunosuppressive medication, in stabilizing or improving lung function in people with LAM.
Study Status: Active, not recruiting
Recruiting:
n/a
| Condition |
Intervention |
Phase |
|
Lymphangioleiomyomatosis |
Drug: Sirolimus
Drug: Placebo sirolimus |
Phase 3 |
Verified by
Office of Rare Diseases (ORD)
August, 2009
Sponsored by: Office of Rare Diseases (ORD)
Information provided by: Office of Rare Diseases (ORD)
ClinicalTrials.gov identifier: NCT00414648
Study Type: Interventional
Study Design: Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Outcomes Assessor), Primary Purpose: Treatment
Cleveland Clinic Foundation
Cleveland, Ohio 44195
United States
Frank McCormack, MD., Principal Investigator
Bruce Trapnell, MD., Principal Investigator