Trial · NCT01059318
A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
NCT01059318, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2020-11-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis. Trials, record NCT01059318. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01059318
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
- NCT number
- NCT01059318
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 24 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Lymphangioleiomyomatosis
- Interventions
- Everolimus
- First posted
- 2010-01-29
- Last updated
- 2020-11-19
- Other registries' IDs
- eudract:2010-019825-32
- Results first posted
- 2020-11-19
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations | Baseline, 26 weeks | Blood samples (1 mL) for determination of VEGF-D were collected from a forearm vein (direct venipuncture or from an indwelling cannula) and 2 aliquots of serum were collected. VEGF-D levels were determined from only 1 of the 2 serum aliquots, with the second acting as a back-up. A serum VEGF-D \>800 pg/mL level supports a diagnosis of Lymphangioleiomyomatosis (LAM) |
| Primary | Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State | pre-dose and at 2 hour post dose at week 26 | Venous blood samples (2 mL) for pharmacokinetic evaluation were collected pre dose and 2 hours post dose at preselected visits. |
| Secondary | Change From Baseline in Forced Vital Capacity (FVC) | Baseline, 26 weeks | All spirometry evaluation followed recommendations of the Standardization of Lung Function Testing. All spirometry maneuvers were performed in sitting position whilst wearing nose clips. At least three acceptable maneuvers were performed for each time point, and results met within-test and between-test criteria for acceptability. The highest value was obtained of FVC from any of the three maneuvers that met acceptability criteria. Change from baseline in FVC was compared between everolimus and historical placebo data from multicenter trial of sirolimus in LAM. (MILES NCT00414648) due to absence of placebo group in this current study on a rare lung disease. These two studies had different study designs, so the change from baseline to 6 months (26 weeks) in MILES study was estimated from the publicly reported rate of change per month to provide a meaningful comparison. This historical control be can be viewed on the Novartis Clinical Trial Results website. |
| Secondary | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) | Baseline, 26 weeks | All spirometry evaluation followed the recommendations of the Standardization of Lung Function Testing. All spirometry maneuvers were performed in the sitting position whilst wearing nose clips. At least three acceptable maneuvers were performed for each time point, and the results met within-test and between-test criteria for acceptability. The highest value was obtained of FEV1 from any of the three maneuvers that met acceptability criteria. Change from baseline in FEV1 was compared between everolimus and historical placebo data from the MILES study (NCT00414648) due to the absence of a placebo group in this current study on a rare lung disease. These two studies had different study designs, so the change from baseline to 6 months (26 weeks) in MILES study was estimated from the publicly reported rate of change per month in order to provide a meaningful comparison. This historical control can be viewed on the Novartis Clinical Trial Results website. |
| Secondary | Change From Baseline in Extended Pulmonary Function Testing | Baseline, 26 weeks | Extended pulmonary function testing such as Total Lung Capacity (TLC), Thoracic Gas Volume (TGV), Residual Volume (RV) were measured using a body plethysmograph |
| Secondary | Change From Baseline in Carbon Monoxide Diffusing Capacity (DLCO) | Baseline, 26 weeks | Carbon Monoxide Diffusing Capacity (DLCO) one of the extended pulmonary function testing which was also measured using a body plethysmograph. |
| Secondary | Change From Baseline in 6-minute Walk Test Score to Measure Exercise Capacity | Baseline, 26 weeks | A standardized 6-minute walk test (6MWT) was performed in accordance with the guidelines of the American Thoracic Society 2002. The test was done about the same time of day to avoid diurnal variation in an environment that had an adequate temperature to avoid additional burden to the patient due to heat or cold air. The distance walked in six minutes (6MWD) was recorded. |
| Secondary | Change From Baseline in Oxygen Saturation | Baseline, 26 weeks | Oxygen saturation means the amount of oxygen in blood stream. Oxygen saturation was measured by using a Pulse Oximeter. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations | Baseline, 26 weeks | Pharmacodynamic analysis included patients who received study drug without any major protocol violation. Same patients in different dose groups were included in this analysis with available data. | pg/mL | MEAN |
| PRIMARY | Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State | pre-dose and at 2 hour post dose at week 26 | Pharmacokinetics analysis set. Patients with measurable data at particular time points were included in this analysis. | ng/mL | MEAN |
| SECONDARY | Change From Baseline in Forced Vital Capacity (FVC) | Baseline, 26 weeks | Pharmacodynamic analysis set for everolimus reporting arm. Patients with both baseline and 26 weeks assessment were included in this analysis. | mL | MEAN |
| SECONDARY | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) | Baseline, 26 weeks | Pharmacodynamic analysis set for everolimus reporting arm. Patients with both baseline and 26 weeks assessment were included in this analysis. | mL | MEAN |
| SECONDARY | Change From Baseline in Extended Pulmonary Function Testing | Baseline, 26 weeks | Pharmacodynamic analysis set. Patients with both baseline and 26 weeks assessment were included in the analysis. | Liters (L) | MEAN |
| SECONDARY | Change From Baseline in Carbon Monoxide Diffusing Capacity (DLCO) | Baseline, 26 weeks | Pharmacodynamic analysis set. Patients with both baseline and 26 weeks assessment were included in the analysis. | ml/min/mmHg | MEAN |
| SECONDARY | Change From Baseline in 6-minute Walk Test Score to Measure Exercise Capacity | Baseline, 26 weeks | Pharmacodynamic analysis set. Patients with both baseline and 26 weeks assessment were included in the analysis. | meters (m) | MEAN |
| SECONDARY | Change From Baseline in Oxygen Saturation | Baseline, 26 weeks | Pharmacodynamic analysis set. Patients with both baseline and 26 weeks assessment were included in the analysis. | percentage of oxygen | MEAN |
Source ClinicalTrials.gov
Results by group17
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations | Everolimus 2.5 mg/Day | -464.3 | Standard Deviation 745.23 | pg/mL | |
| Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations | Everolimus 5 mg/Day | -1113.2 | Standard Deviation 1468.9 | pg/mL | |
| Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations | Everolimus 10 mg/Day | -1771.7 | Standard Deviation 2091.6 | pg/mL | |
| Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State | Everolimus 2.5 mg/Day | Trough (C0, ss) | 3.1 | Standard Deviation 1.33 | ng/mL |
| Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State | Everolimus 5 mg/Day | Trough (C0, ss) | 5.8 | Standard Deviation 3.02 | ng/mL |
| Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State | Everolimus 10 mg/Day | Trough (C0, ss) | 11.0 | Standard Deviation 3.45 | ng/mL |
| Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State | Everolimus 2.5 mg/Day | Peak (C2, ss) | 12.1 | Standard Deviation 3.56 | ng/mL |
| Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State | Everolimus 5 mg/Day | Peak (C2, ss) | 22.6 | Standard Deviation 7.22 | ng/mL |
| Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State | Everolimus 10 mg/Day | Peak (C2, ss) | 45.7 | Standard Deviation 12.52 | ng/mL |
| Change From Baseline in Forced Vital Capacity (FVC) | Everolimus | 10 | 95% Confidence Interval -111 to 132 | mL |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 results by groupParticipant flow10
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Core Study (26 Week) | STARTED | Everolimus | 24 | |
| Core Study (26 Week) | Everolimus 2.5 mg for 4 Weeks | Everolimus | 24 | |
| Core Study (26 Week) | Everolimus 5 mg for 4 Weeks | Everolimus | 24 | |
| Core Study (26 Week) | Everolimus 10 mg for 18 Weeks | Everolimus | 24 | |
| Core Study (26 Week) | Pharmacokinetics (PK) Analysis Set | Everolimus | 16 | |
| Core Study (26 Week) | COMPLETED | Everolimus | 20 | |
| Core Study (26 Week) | NOT COMPLETED | Everolimus | 4 | |
| Extension (36 Week) | STARTED | Everolimus | 20 | |
| Extension (36 Week) | COMPLETED | Everolimus | 17 | |
| Extension (36 Week) | NOT COMPLETED | Everolimus | 3 |
Source ClinicalTrials.gov
Adverse events59
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 | Abdominal pain upper | Gastrointestinal disorders | Everolimus | 2 | 24 | |
| other | Aphthous stomatitis | Gastrointestinal disorders | Everolimus | 5 | 24 | |
| other | Constipation | Gastrointestinal disorders | Everolimus | 2 | 24 | |
| other | Diarrhoea | Gastrointestinal disorders | Everolimus | 7 | 24 | |
| other | Haemorrhoids | Gastrointestinal disorders | Everolimus | 3 | 24 | |
| other | Mouth ulceration | Gastrointestinal disorders | Everolimus | 11 | 24 | |
| other | Nausea | Gastrointestinal disorders | Everolimus | 7 | 24 | |
| other | Stomatitis | Gastrointestinal disorders | Everolimus | 7 | 24 | |
| other | Vomiting | Gastrointestinal disorders | Everolimus | 4 | 24 | |
| other | Fatigue | General disorders | Everolimus | 7 | 24 |
1 to 10 of 59Source ClinicalTrials.gov
See all 59 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Everolimus | ||
| Total, other adverse events | Everolimus | 24 | 24 |
| Total, serious adverse events | Everolimus | 8 | 24 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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/NCT01059318
GET https://conductscience.org/api/trials/v1/NCT01059318/events?page=2