Discovery of Sirolimus Sensitive Biomarkers in Blood
| Status: | Recruiting |
|---|---|
| Conditions: | Lymphoma, Endocrine |
| Therapuetic Areas: | Endocrinology, Oncology |
| Healthy: | No |
| Age Range: | 18 - 90 |
| Updated: | 4/6/2019 |
| Start Date: | December 4, 2017 |
| End Date: | September 30, 2020 |
| Contact: | Tania R Machado |
| Email: | tania.machado@nih.gov |
| Phone: | (301) 496-3632 |
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
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 3 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.
Participant samples will be stored in a secure place. No personal data will be connected to
them.
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 3 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.
Participant samples will be stored in a secure place. No personal data will be connected to
them.
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 stabilizes lung function, resolves chylous effusions and lymphangioleiomas and
shrinks angiomyolipomas. The current study is to understand better the short-term action of
the drug by following the effects on potential biomarkers in blood and urine. Patients with
LAM will have samples taken prior to administration of first dose of the drug, at 1 hr and
then at 23 hours after the drug (trough level). At 3 and 9 months, samples will be obtained
at trough and 1 hour after the dose. Molecular and cellular analyses will be performed to
look for potential biomarkers.
of rapamycin (mTOR), has been shown to be effective in patients with lymphangioleiomyomatosis
(LAM). It stabilizes lung function, resolves chylous effusions and lymphangioleiomas and
shrinks angiomyolipomas. The current study is to understand better the short-term action of
the drug by following the effects on potential biomarkers in blood and urine. Patients with
LAM will have samples taken prior to administration of first dose of the drug, at 1 hr and
then at 23 hours after the drug (trough level). At 3 and 9 months, samples will be obtained
at trough and 1 hour after the dose. Molecular and cellular analyses will be performed to
look for potential biomarkers.
- 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
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
301-496-2563
Phone: 800-411-1222
National Institutes of Health Clinical Center The National Institutes of Health (NIH) Clinical Center in...
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