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 : July 29, 2022
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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.
Condition or disease |
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Lymphangioleiomyomatosis |
Study Type : | Observational |
Estimated Enrollment : | 26 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | Discovery of Sirolimus Sensitive Biomarkers in Blood |
Actual Study Start Date : | December 4, 2017 |
Estimated Primary Completion Date : | September 30, 2023 |
Estimated Study Completion Date : | September 30, 2023 |

Group/Cohort |
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taking sirolimus
Subjects will come to the NIH and begin taking sirolimus 2mg
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- 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

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Ages Eligible for Study: | 18 Years to 90 Years (Adult, Older Adult) |
Sexes Eligible for Study: | Female |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
- 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

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) 827-1376 | worthyt@mail.nih.gov | |
Contact: Joel Moss, M.D. | (301) 496-1597 | mossj@nhlbi.nih.gov |
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 prpl@cc.nih.gov |
Principal Investigator: | Joel Moss, M.D. | National Heart, Lung, and Blood Institute (NHLBI) |
Responsible Party: | National Heart, Lung, and Blood Institute (NHLBI) |
ClinicalTrials.gov Identifier: | NCT03304678 |
Other Study ID Numbers: |
180003 18-H-0003 |
First Posted: | October 9, 2017 Key Record Dates |
Last Update Posted: | July 29, 2022 |
Last Verified: | July 26, 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
mTORC1 Inhibition VEGF-D MicroRNA Natural History |
Lymphangioleiomyomatosis Immune System Diseases Lymphangiomyoma Lymphatic Vessel Tumors Neoplasms by Histologic Type Neoplasms |
Perivascular Epithelioid Cell Neoplasms Neoplasms, Connective and Soft Tissue Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders |