Trial · NCT03304678
Discovery of Sirolimus Sensitive Biomarkers in Blood
NCT03304678, Phase 2, Completed, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2026-02-18
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Discovery of Sirolimus Sensitive Biomarkers in Blood. Trials, record NCT03304678. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03304678
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Discovery of Sirolimus Sensitive Biomarkers in Blood
- NCT number
- NCT03304678
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 33 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Lymphangioleiomyomatosis
- Interventions
- Sirolimus 2mg
- First posted
- 2017-10-09
- Last updated
- 2026-02-18
- Results first posted
- 2026-02-18
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) 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) | 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. |
Source ClinicalTrials.gov
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) 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) | Data from 20 participants were analyzed, as these individuals had plasma collections completed at all required timepoints. | plasma proteins | NUMBER |
Source ClinicalTrials.gov
Results by group1
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants | Participant With LAM Receiving Sirolimus | 662 | plasma proteins |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus | 33 | |
| Overall Study | COMPLETED | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus | 24 | |
| Overall Study | NOT COMPLETED | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus | 9 |
Source ClinicalTrials.gov
Adverse events1
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Pneumothorax | Respiratory, thoracic and mediastinal disorders | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus | 1 | 32 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus | 0 | 32 |
| Total, other adverse events | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus | 1 | 32 |
| Total, serious adverse events | Participants Diagnosed With Lymphangioleiomyomatosis Receiving Sirolimus | 0 | 32 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03304678
GET https://conductscience.org/api/trials/v1/NCT03304678/registered