Blood markers may guide LAM treatment dosing

NCT ID NCT03304678

Completed Knowledge-focused Sponsor: National Heart, Lung, and Blood Institute (NHLBI) Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study looked at 33 women with LAM, a rare lung disease, to find blood and urine markers that show how the drug sirolimus is working. Participants took sirolimus daily for 9 months and gave samples at the start, 3 months, and 9 months. The goal was to identify markers that could help doctors choose the right dose for each patient.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: sirolimus (Rapamune)
What this could lead to: If successful, this could help doctors determine the best dose of sirolimus for women with LAM, potentially improving treatment and reducing side effects.
What could go wrong: This is a small, early-phase study focused on finding biomarkers, not proving the drug works. The results may not lead to a clear dosing guideline or apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

lymphangioleiomyomatosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

LYMPHANGIOLEIOMYOMATOSIS

Contacts and locations

Locations

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

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