Trial · NCT00414648
Efficacy and Safety of Sirolimus in LAM
NCT00414648, Phase 3, Completed, University of Cincinnati; results first posted 2023-11-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Sirolimus in LAM. Trials, record NCT00414648. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00414648
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Sirolimus in LAM
- NCT number
- NCT00414648
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 89 participants, actual
- Lead sponsor
- University of Cincinnati
- Conditions
- Lymphangioleiomyomatosis
- Interventions
- Sirolimus, Placebo
- First posted
- 2006-12-21
- Last updated
- 2023-11-02
- Results first posted
- 2023-11-02
- Limitations and caveats
The observation period of the trial was truncated for some subjects, driven by funding limitations, need for timely reporting and drug expiration factors. Please see supplement to McCormack FX, et al. Efficacy and safety of sirolimus in LAM. N Engl J Med. 2011, 28;364:1595-606. PMID: 21410393 for more detail.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Changes in Forced Expiratory Volume in One-second (FEV1) Slope in Milliliters Per Month | Baseline to Month 12 | FEV1 values reported are in liters or milliliters. There are no definite minimum and maximum values of FEV1 as it is a physiological measure of lung function and varies from individual to individual. Normative ranges are determined in populations stratified by gender, height, age and race/ethnicity. Higher FEV1 values indicate better lung function. |
| Secondary | Percent of Participants With Serious Adverse Events | Baseline to Month 12 | |
| Secondary | Changes in FVC Slope in ml/Month | Baseline to Month 12 | FVC values are reported in liters or milliliters. Normative ranges are determined in populations that are stratified by gender, height, age and race/ethnicity. There are no minimum or maximum values as it is a physiologic measure of lung function and varies from individual to individual. Higher FVC scores indicate better lung function. |
| Secondary | Rate of Change in Diffusing Capacity for Carbon Monoxide (DLCO) in ml/Min/mmHg | Baseline to Month 12 | Diffusing capacity for carbon monoxide is abbreviated DLCO. DLCO is measured in liters and milliliters. There are no definite minimum and maximum values of FEV1 as it is a physiological measure of lung function and varies from individual to individual. A lower DLCO means that there is lower lung function. |
| Secondary | Rate of Change in Total Lung Volume in ml Per Month | Baseline to Month 12 | There are two methods for measurement of total lung volume, nitrogen or helium wash out (total lung capacity or TLC), or plethysmography (Total gas volume or TGV). TLC or TGV is measured in milliliters or liters. Normative ranges for TLC or TGV are stratified by age, height, and sex in population based studies of normal subjects.. There are no definite minimum and maximum values of lung volume as it is a physiological measure of lung function and varies from individual to individual. A low TGV or TLC is suggestive of a restrictive lung disease, and a high TGV or TLC is consistent with obstructive lung disease with hyperinflation. |
| Secondary | Rate of Change in Six Minute Walk Distance in Meters/Month | Baseline to Month 12 | Number of meters walked in six minutes. There is no minimum or maximum number of feet walked as this varies from individual to individual. A higher number of feet walked indicates better exercise tolerance |
| Secondary | Rate of Change in Serum Vascular Endothelial Growth Factor-D (VEGF-D) Levels in pg/ml Per Month | Baseline to Month 12 | Serum VEGF-D is a protein produced by LAM cells that serves as a biomarker of mTOR activation and sirolimus response. Mean serum levels of VEGF-D in normal subjects are around 350 pg/ml. Higher levels of serum VEGF-D are correlated with greater degrees of mTOR activation, and a fall in VEGF-D levels suggest suppression of the mTOR axis. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Changes in Forced Expiratory Volume in One-second (FEV1) Slope in Milliliters Per Month | Baseline to Month 12 | Intention to treat | milliliters per month | MEAN |
| SECONDARY | Percent of Participants With Serious Adverse Events | Baseline to Month 12 | Intention to treat | percentage of participants with SAE | NUMBER |
| SECONDARY | Changes in FVC Slope in ml/Month | Baseline to Month 12 | Intention To Treat | milliliters/month | MEAN |
| SECONDARY | Rate of Change in Diffusing Capacity for Carbon Monoxide (DLCO) in ml/Min/mmHg | Baseline to Month 12 | Intention to treat | ml/min/mmHg | MEAN |
| SECONDARY | Rate of Change in Total Lung Volume in ml Per Month | Baseline to Month 12 | Intention to treat | milliliters/month | MEAN |
| SECONDARY | Rate of Change in Six Minute Walk Distance in Meters/Month | Baseline to Month 12 | Intention to treat | meters/month | MEAN |
| SECONDARY | Rate of Change in Serum Vascular Endothelial Growth Factor-D (VEGF-D) Levels in pg/ml Per Month | Baseline to Month 12 | pg/ml per month | MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Changes in Forced Expiratory Volume in One-second (FEV1) Slope in Milliliters Per Month | Sirolimus | 1 | Standard Deviation 2 | milliliters per month | |
| Changes in Forced Expiratory Volume in One-second (FEV1) Slope in Milliliters Per Month | Placebo | -12 | Standard Deviation 2 | milliliters per month | |
| Percent of Participants With Serious Adverse Events | Active Agent (Sirolimus) | 50 | percentage of participants with SAE | ||
| Percent of Participants With Serious Adverse Events | Placebo Arm | 41.86 | percentage of participants with SAE | ||
| Changes in FVC Slope in ml/Month | Sirolimus Treatment Arm | 8 | Standard Deviation 3 | milliliters/month | |
| Changes in FVC Slope in ml/Month | Placebo Treatment Arm | -11 | Standard Deviation 3 | milliliters/month | |
| Rate of Change in Diffusing Capacity for Carbon Monoxide (DLCO) in ml/Min/mmHg | Sirolimus Treatment Arm | -0.01 | Standard Deviation 0.02 | ml/min/mmHg | |
| Rate of Change in Diffusing Capacity for Carbon Monoxide (DLCO) in ml/Min/mmHg | Placebo Treatment Arm | -0.06 | Standard Deviation 0.03 | ml/min/mmHg | |
| Rate of Change in Total Lung Volume in ml Per Month | Sirolimus Treatment Arm | 8 | Standard Deviation 7 | milliliters/month | |
| Rate of Change in Total Lung Volume in ml Per Month | Placebo Treatment Arm | -2 | Standard Deviation 7 | milliliters/month |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sirolimus Treatment Arm | 46 | |
| Overall Study | STARTED | Placebo Treatment Arm | 43 | |
| Overall Study | COMPLETED | Sirolimus Treatment Arm | 41 | |
| Overall Study | COMPLETED | Placebo Treatment Arm | 34 | |
| Overall Study | NOT COMPLETED | Sirolimus Treatment Arm | 5 | |
| Overall Study | NOT COMPLETED | Placebo Treatment Arm | 9 |
Source ClinicalTrials.gov
Adverse events376
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 | Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | Sirolimus Treatment Arm | 2 | 46 | 2 |
| other | Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | Placebo Treatment Arm | 2 | 43 | 4 |
| other | Anemia | Blood and lymphatic system disorders | Sirolimus Treatment Arm | 2 | 46 | 2 |
| other | Anemia | Blood and lymphatic system disorders | Placebo Treatment Arm | 1 | 43 | 1 |
| other | Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | Sirolimus Treatment Arm | 1 | 46 | 1 |
| other | Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | Placebo Treatment Arm | 4 | 43 | 7 |
| other | Cardiac disorders - Other, specify | Cardiac disorders | Sirolimus Treatment Arm | 4 | 46 | 7 |
| other | Cardiac disorders - Other, specify | Cardiac disorders | Placebo Treatment Arm | 4 | 43 | 5 |
| other | Left ventricular systolic dysfunction | Cardiac disorders | Sirolimus Treatment Arm | 1 | 46 | 1 |
| other | Left ventricular systolic dysfunction | Cardiac disorders | Placebo Treatment Arm | 0 | 43 | 0 |
1 to 10 of 376Source ClinicalTrials.gov
See all 376 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sirolimus Treatment Arm | 0 | 46 |
| Total, all-cause mortality | Placebo Treatment Arm | 1 | 43 |
| Total, other adverse events | Sirolimus Treatment Arm | 46 | 46 |
| Total, other adverse events | Placebo Treatment Arm | 43 | 43 |
| Total, serious adverse events | Sirolimus Treatment Arm | 23 | 46 |
| Total, serious adverse events | Placebo Treatment Arm | 18 | 43 |
Source ClinicalTrials.gov
Papers12
The papers the trial lists on ClinicalTrials.gov.
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U54RR019498 | Rare Lung Disease Clinical Research Network | TRAPNELL, BRUCE C | CINCINNATI CHILDRENS HOSP MED CTR | NATIONAL CENTER FOR RESEARCH RESOURCES | 2003 to 2008 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Sirolimus Treatment Arm vs Placebo Treatment Arm: deaths from any cause | Trial comparisons |
| Sirolimus Treatment Arm vs Placebo Treatment Arm: serious adverse events | Trial comparisons |
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/NCT00414648
GET https://conductscience.org/api/trials/v1/NCT00414648/events?page=2