Clinical trial · Interventional
Discovery of Sirolimus Sensitive Biomarkers in Blood
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Lymphangioleiomyomatosis | Lymphangioleiomyomatosis | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Sirolimus 2mg | Drug | Sirolimus | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- OTHER
- label
- Participants diagnosed with Lymphangioleiomyomatosis receiving Sirolimus
- description
- Participants diagnosed with Lymphangioleiomyomatosis will be administered Sirolimus 2 mg by mouth daily to be prescribed by treating physician.
- interventionNames
- Drug: Sirolimus 2mg
Primary outcomes (1)
- measure
- Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants
- timeFrame
- Day 0 (pre-treatment, 1 hour post-Sirolimus, 23 hours post-Sirolimus), Month 3 (23 hours post-Sirolimus, 1 hour post next dose), Month 9 (23 hours post-Sirolimus, 1 hour post next dose)
- description
- Plasma from Lymphangioleiomyomatosis (LAM) participants was profiled using the aptamer-based Somascan 11k assay (Somalogic). The primary outcome is the number of plasma proteins that exhibit a statistically significant change from each participant's baseline following sirolimus administration, defined by pre-specified multiplicity-adjusted thresholds (e.g., FDR q\<0.05 with absolute log2 fold-change ≥0.5), representing candidate sirolimus-sensitive biomarkers.
Eligibility
Eligibility (as posted)
- Sex
- Female
- Minimum age
- 18 Years
- Maximum age
- 90 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available