This study is currently recruiting participants.
Number
18-H-0003
Sponsoring Institute
National Heart, Lung and Blood Institute (NHLBI)
Recruitment Detail
Type: Participants currently recruited/enrolled Gender: Female Min Age: 18 Years Max Age: 90 Years
Referral Letter Required
Yes
Population Exclusion(s)
Fetuses;Pregnant Women;Children;Male
Keywords
mTORC1 Inhibition; VEGF-D; MicroRNA; Natural History
Recruitment Keyword(s)
None
Condition(s)
Lymphangioleiomyomatosis
Investigational Drug(s)
Investigational Device(s)
Intervention(s)
Drug: Sirolimus 2mg
Supporting Site
National Heart, Lung, and Blood Institute
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.
Objective:
To determine if blood and urine markers after 1 dose and again after 9 months can be used to evaluate the correct dose of sirolimus for people with LAM.
Eligibility:
Women ages 18-90 with LAM whose doctors have decided they should start taking sirolimus to treat it.
Design:
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.
Visit 3 will be 9 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.
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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
- 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
Principal Investigator
Referral Contact
For more information: