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NCT03304678 · ClinicalTrials.gov registry record · Phase 2
Discovery of Sirolimus Sensitive Biomarkers in Blood
A Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 2
- Development phase
- 33
- Enrollment target
NCT03304678: Completed Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT03304678 is a Phase 2 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 33 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (75% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03304678, a Phase 2 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 33 participants
- Enrollment target
Study 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.
Primary Outcome
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.
Interventions
- DRUG Sirolimus 2mg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 33 participants |
| Start Date | 2017-12-04 |
| Est. Completion | 2025-03-14 |
| Phase | Phase 2 |
What the finished NCT03304678 record still lists
NCT03304678 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 33 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (75% lower).
The record links to 0 conditions, and to 1 intervention - of which Sirolimus 2mg is the first listed.
NCT03304678 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03304678 about?
NCT03304678 is a clinical study titled "Discovery of Sirolimus Sensitive Biomarkers in Blood". 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 dr...
What is the current status of trial NCT03304678?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 33 participants. The study started on 2017-12-04. Estimated completion is 2025-03-14.
What interventions are being tested in trial NCT03304678?
The interventions under investigation include: Sirolimus 2mg (DRUG).
Who is sponsoring clinical trial NCT03304678?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03304678's enrollment target sits among peer trials
33 1553rd of 2000 higher than 438 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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