Trial Outcomes & Findings for Discovery of Sirolimus Sensitive Biomarkers in Blood (NCT NCT03304678)
NCT ID: NCT03304678
Last Updated: 2026-02-18
Results Overview
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.
COMPLETED
PHASE2
33 participants
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)
2026-02-18
Participant Flow
Participant milestones
| Measure |
Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus
Participants diagnosed with Lymphangioleiomyomatosis (LAM) will be administered Sirolimus 2 mg by mouth daily as prescribed by treating physician will be referred to the NIH Clinical Center for these studies.
|
|---|---|
|
Overall Study
STARTED
|
33
|
|
Overall Study
COMPLETED
|
24
|
|
Overall Study
NOT COMPLETED
|
9
|
Reasons for withdrawal
| Measure |
Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus
Participants diagnosed with Lymphangioleiomyomatosis (LAM) will be administered Sirolimus 2 mg by mouth daily as prescribed by treating physician will be referred to the NIH Clinical Center for these studies.
|
|---|---|
|
Overall Study
Physician Decision
|
5
|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Non-compliance
|
3
|
Baseline Characteristics
Discovery of Sirolimus Sensitive Biomarkers in Blood
Baseline characteristics by cohort
| Measure |
Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus
n=33 Participants
Participants diagnosed with Lymphangioleiomyomatosis (LAM) will be administered Sirolimus 2 mg by mouth daily as prescribed by treating physician will be referred to the NIH Clinical Center for these studies.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
33 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
33 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
32 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
25 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
33 participants
n=4 Participants
|
PRIMARY outcome
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)Population: Data from 20 participants were analyzed, as these individuals had plasma collections completed at all required timepoints.
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.
Outcome measures
| Measure |
Participant With LAM Receiving Sirolimus
n=10000 plasma proteins
Participants with Lymphangioleiomyomatosis (LAM) will receive Sirolimus 2 mg by mouth daily prescribed by treating physicians.
|
|---|---|
|
Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants
|
662 plasma proteins
|
Adverse Events
Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus
n=32 participants at risk
Participants diagnosed with Lymphangioleiomyomatosis (LAM) will be administered Sirolimus 2 mg by mouth daily as prescribed by treating physician will be referred to the NIH Clinical Center for these studies.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
3.1%
1/32 • Number of events 1 • Up to 9 months
|
Additional Information
Joel Moss, M.D., Ph.D.
National Heart, Lung, and Blood Institute (NHLBI)
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place