Discovery of Sirolimus Sensitive Biomarkers in Blood
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|ClinicalTrials.gov Identifier: NCT03304678|
Recruitment Status : Recruiting
First Posted : October 9, 2017
Last Update Posted : December 16, 2021
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 drug called sirolimus stabilizes lung function in people with LAM. But researchers do not know what drug dose and blood serum levels are needed to reach this stability. Researchers want to learn more about the right dose of sirolimus for people with LAM.
To determine if blood and urine markers after 1 dose and again after 3 months can be used to evaluate the correct dose of sirolimus for people with LAM.
Women ages 18-90 with LAM whose doctors have decided they should start taking sirolimus to treat it.
At visit 1, participants will take their first dose of sirolimus by mouth at the clinic. They will have blood and urine collected.
Participants will take 1 tablet of the study drug each day.
Visit 2 will be 3 months after visit 1. Participants will have blood and urine collected.
Participant samples will be stored in a secure place. No personal data will be connected to them.
|Condition or disease|
|Study Type :||Observational|
|Estimated Enrollment :||26 participants|
|Official Title:||Discovery of Sirolimus Sensitive Biomarkers in Blood|
|Actual Study Start Date :||December 4, 2017|
|Estimated Primary Completion Date :||September 30, 2022|
|Estimated Study Completion Date :||September 30, 2022|
Subjects will come to the NIH and begin taking sirolimus 2mg
- to identify if miRNA are responsive to sirolimus in patients with LAM [ Time Frame: 9 months ]identify if miRNA are responsive to sirolimus in patients with LAM
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03304678
|Contact: Tatyana Worthy, R.N.||(301) firstname.lastname@example.org|
|United States, Maryland|
|National Institutes of Health Clinical Center||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 email@example.com|
|Principal Investigator:||Joel Moss, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|