Status:
COMPLETED
Discovery of Sirolimus Sensitive Biomarkers in Blood
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Lymphangioleiomyomatosis
Eligibility:
FEMALE
18-90 years
Phase:
PHASE2
Brief Summary
Background: Lymphangioleiomyomatosis (LAM) is a rare, progressive disease. It usually affects women in the prime of their lives. It typically results in lung destruction. Studies have shown that a dr...
Detailed Description
Sirolimus (rapamycin), which acts as a targeted inhibitor of the protein mechanistic target of rapamycin (mTOR), has been shown to be effective in patients with lymphangioleiomyomatosis (LAM). It stab...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA
- Female 18 to 90 years of age
- Diagnosis of LAM
- Initiation of sirolimus therapy (2mg daily) based on standard-of-care pulmonary indications and the advice of the patient s local physician
- EXCLUSION CRITERIA
- Unable to travel to the NIH
- Unable to provide informed consent
- 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
- Women who are pregnant or lactating
Exclusion
Key Trial Info
Start Date :
December 4 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 14 2025
Estimated Enrollment :
33 Patients enrolled
Trial Details
Trial ID
NCT03304678
Start Date
December 4 2017
End Date
March 14 2025
Last Update
April 2 2025
Active Locations (1)
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1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892