Phase 2 N=33 Other

Discovery of Sirolimus Sensitive Biomarkers in Blood

Lymphangioleiomyomatosis

Bottom Line

View on ClinicalTrials.gov: NCT03304678 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Feb 2026

Primary outcome: Primary: Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants — 662 plasma proteins

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Sirolimus 2mg (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: Female
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Primary completion: Mar 2025

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants	662	—

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 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.

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03304678). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.