Ignite Creation Date:
2024-05-05 @ 5:15 PM
Last Modification Date:
2024-10-26 @ 9:29 AM
Study NCT ID:
NCT00414648
Status:
COMPLETED
Last Update Posted:
2023-11-02
First Post:
2006-12-20
Brief Title:
Efficacy and Safety of Sirolimus in LAM
Sponsor:
University of Cincinnati
Organization:
University of Cincinnati
Study Overview
Official Title:
Multicenter International Lymphangioleiomyomatosis Efficacy and Safety of Sirolimus Trial
Status:
COMPLETED
Status Verified Date:
2023-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
MILES
Brief Summary:
Lymphangioleiomyomatosis LAM is a rare lung disease of women that is caused by genetic mutations It results in the uncontrolled growth of an unusual type of smooth muscle cell in the lung 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 inhibitor of the mTOR pathway in stabilizing or improving lung function in people with LAM
Detailed Description:
LAM is an uncommon progressive cystic lung disease that predominantly affects young women The disease is caused by mutations in tuberous sclerosis complex TSC genes which regulate cellular pathways that control nutrient sensing cell size cell migration and cell proliferation Individuals with LAM often experience pneumothorax and chylothorax as well progressive loss of lung function Sirolimus is drug that was approved for the prevention of kidney transplant rejection It directly affects the cellular pathway that causes LAM This study will evaluate the safety and effectiveness of sirolimus in stabilizing or improving lung function in people with LAM
Individuals interested in participating in this 2-year double-blind study will first report to the study sites for pulmonary function testing to determine their eligibility for participation Participants deemed eligible will be randomly assigned to receive either sirolimus or placebo for 1 year Sirolimus or placebo will be administered in 2 tablet doses 2 mg for sirolimus for the duration of the study Study visits will occur at baseline Week 3 every 3 months for 12 months and months 18 and 24 Study visits will include a physical exam questionnaires a pregnancy test blood and urine collection and functional lung tests A 6-minute walk test will occur at most study visits a chest x-ray will be taken at baseline and month 24 and a volumetric computed tomography scan will occur at baseline month 12 and month 24 Adverse events medication side effects and lung function will be assessed at each visit
Individuals interested in participating in this 2-year double-blind study will first report to the study sites for pulmonary function testing to determine their eligibility for participation Participants deemed eligible will be randomly assigned to receive either sirolimus or placebo for 1 year Sirolimus or placebo will be administered in 2 tablet doses 2 mg for sirolimus for the duration of the study Study visits will occur at baseline Week 3 every 3 months for 12 months and months 18 and 24 Study visits will include a physical exam questionnaires a pregnancy test blood and urine collection and functional lung tests A 6-minute walk test will occur at most study visits a chest x-ray will be taken at baseline and month 24 and a volumetric computed tomography scan will occur at baseline month 12 and month 24 Adverse events medication side effects and lung function will be assessed at each visit
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| FD-003362-01 | OTHER_GRANT | FDA Office of Orphan Product Development | httpsreporternihgovquickSearchU54RR019498-01 |
| U54RR019498-01 | NIH | None | None |