Trial · NCT00790400
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
NCT00790400, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2012-08-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM). Trials, record NCT00790400. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00790400
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
- NCT number
- NCT00790400
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 118 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
- Interventions
- Everolimus (RAD001), Everolimus Placebo
- First posted
- 2008-11-13
- Last updated
- 2017-02-17
- Other registries' IDs
- eudract:2008-002113-48
- Results first posted
- 2012-08-28
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Angiomyolipoma Response Rate as Per Central Radiology Review | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years | Angiomyolipoma response defined as the combination of the following criteria: reduction in angiomyolipoma volume of ≥ 50% relative to baseline, where angiomyolipoma volume was sum of volumes of all target lesions identified at baseline, and with a confirmatory scan performed approximately 12 weeks later (no sooner than 8 weeks later); no new angiomyolipoma lesions ≥ 1.0 cm in longest diameter were identified; there were no kidney increases in volume \> 20% from nadir. The patient did not have any angiomyolipoma-related bleeding of ≥ grade 2. For the everolimus (core/extension periods) treatment group, the baseline means the latest value on or before starting everolimus. |
| Secondary | Time to Angiomyolipoma Progression as Per Central Radiology Review | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years | Time to angiomyolipoma progression (TTAP) is defined as time from date of randomization to date of first documented angiomyolipoma progression. Angiomyolipoma progression was defined as one or more of the following: Increase from nadir of ≥ 25% in angiomyolipoma volume to value greater than baseline; the appearance of a new angiomyolipoma ≥ 1.0 cm in longest diameter; an increase from nadir of 20% or more in the volume of either kidney to a value greater than baseline; angiomyolipoma-related bleeding grade ≥ 2. For the everolimus (core/extension periods) treatment group, the time to angiomyolipoma progression is defined starting from the start of everolimus. The baseline means the latest value on or before starting everolimus. |
| Secondary | Skin Lesion Response Rate as Per Investigator (Only Patients With at Least One Skin Lesion at Baseline) | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years | Skin lesion response rate in the double-blind period was determined only among patients with at least one skin lesion at baseline, and is the percentage of this group of patients with a best overall skin lesion response on the Physician's Global Assessment of Clinical Condition (PGA) of either complete clinical response (CCR) or partial response (PR). A complete clinical response (CCR) requires a grading of 0 indicating the absence of disease (histological confirmation is not required). Grades 1, 2, and 3 constitute partial response, indicating improvement of at least 50 percent, but less than 100 percent improvement. For the everolimus (core/extension periods) treatment group, the baseline means the latest value on or before starting everolimus. |
| Secondary | Percentage of Participants With Renal Impairment | Day 1 up to 28 days after end of treatment | Renal Impairment was measured by glomerular filtration rate which was calculated using the Modification of Diet in Renal Disease formula. Percentage of participants with renal impairment was reported. Severe renal impairment was defined as a GFR of \<30ml/min/1.73m2. |
| Secondary | Change From Baseline in Plasma Angiogenic Molecules - Vascular Endothelial Growth Factor (VEGF) Marker | 4 weeks, 12 weeks, 24 weeks, 36 weeks 48 weeks, 60 weeks, 72 weeks | Blood samples for biomarker assessment were collected immediately prior to study administration. On-treatment samples was compared to baseline samples with the change from baseline. |
| Secondary | Everolimus Trough Concentrations (Cmin) | Prior to dosing at weeks 2, 4, 12, 24, 48 | Cmin values collected prior to dose administration on the same study day and at 20-28 hours after previous dose, at steady state, and patient did not vomit within 4 hours of previous dose. Samples collected during the first 4 days of dosing were excluded from all analyses. |
| Secondary | Everolimus Blood Concentrations (C2h) at 2 Hours Post-dose | 2 hours post-dose administration at Weeks 2, 4, 12, 24, 48 | C2h values collected 1-3 hours after dose administration on the same study day, at steady state, and patient did not vomit between taking previous dose and blood collection. Samples collected during the first 4 days of dosing will be excluded from all analyses. |
| Secondary | Time to Angiomyolipoma Response - Only Everolimus Patients With Angiomyolipoma Response | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years | Time to angiomyolipoma response was defined as the time from the date of randomization until the date of the first documented angiomyolipoma response. Angiomyolipoma response defined as the combination of the following criteria: reduction in angiomyolipoma volume of ≥ 50% relative to baseline, where angiomyolipoma volume was sum of volumes of all target lesions identified at baseline, and with a confirmatory scan performed approximately 12 weeks later (no sooner than 8 weeks later); no new angiomyolipoma lesions ≥ 1.0 cm in longest diameter were identified; no kidney increases in volume \> 20% from nadir; no angiomyolipoma-related bleeding of ≥ grade 2. For the everolimus (core/extension periods) treatment group, the time to angiomyolipoma response is from the start of everolimus. The baseline in the response definition means the latest value on or before starting everolimus. |
| Secondary | Duration of Angiomyolipoma Response - Only Everolimus Patients With Angiomyolipoma Response | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years | Duration of angiomyolipoma response was defined as the time from the date of the first documented angiomyolipoma response until the date of the first documented angiomyolipoma progression . Angiomyolipoma response was defined as the combination of the following criteria: reduction in angiomyolipoma volume of ≥ 50% relative to baseline, where angiomyolipoma volume was sum of volumes of all target lesions identified at baseline, and with a confirmatory scan performed approximately 12 weeks later (no sooner than 8 weeks later); no new angiomyolipoma lesions ≥ 1.0 cm in longest diameter were identified; there were no kidney increases in volume \> 20% from nadir. The patient did not have any angiomyolipoma-related bleeding of ≥ grade 2. |
| Secondary | Duration of Skin Lesion Response - Only Everolimus Patients With Best Overall Skin Lesion Response of Complete Clinical Response (CCR) or Partial Response (PR) | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years | Duration of skin lesion response is defined as the time from the date of the first skin lesion response until the date of the first skin lesion progression, according to the PGA (physician's global assessment of clinical condition). A progression is when the disease is worse than at baseline evaluation by \>=25% or more. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Angiomyolipoma Response Rate as Per Central Radiology Review | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years | The Full Analysis Set (FAS) is defined according to the Intention-to-Treat principle and consists of all randomized patients. | Percentage of Participants | NUMBER |
| SECONDARY | Time to Angiomyolipoma Progression as Per Central Radiology Review | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years | The Full Analysis Set (FAS) is defined according to the Intention-to-Treat principle and consists of all randomized patients. | months | MEDIAN |
| SECONDARY | Skin Lesion Response Rate as Per Investigator (Only Patients With at Least One Skin Lesion at Baseline) | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years | The Full Analysis Set (FAS) is defined according to the Intention-to-Treat principle and consists of all randomized patients. Only patients with at least one skin lesion at baseline are included in the analysis. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Renal Impairment | Day 1 up to 28 days after end of treatment | Safety set consists of all patients who received at least 1 dose of the double-blind study drug with a valid post-baseline assessment. For the everolimus (core/extension) treatment arm, patients received at least 1 dose of everolimus with a valid post-baseline assessment, where baseline was defined as the assessment just before start of everolimus. | Percentage of Participants | NUMBER |
| SECONDARY | Change From Baseline in Plasma Angiogenic Molecules - Vascular Endothelial Growth Factor (VEGF) Marker | 4 weeks, 12 weeks, 24 weeks, 36 weeks 48 weeks, 60 weeks, 72 weeks | The Full Analysis Set (FAS) is defined according to the Intention-to-Treat principle and consists of all randomized patients. The 'n' signifies those participants evaluable for this measure at specified time points for each group, respectively. | pg/mL | MEAN |
| SECONDARY | Everolimus Trough Concentrations (Cmin) | Prior to dosing at weeks 2, 4, 12, 24, 48 | The analysis was performed in the Safety Set population for patients treated only with Everolimus and with a confirmed PK sample. | ng/mL | MEAN |
| SECONDARY | Everolimus Blood Concentrations (C2h) at 2 Hours Post-dose | 2 hours post-dose administration at Weeks 2, 4, 12, 24, 48 | The analysis was performed in the Safety Set population for patients treated only with Everolimus and with a confirmed PK sample. | ng/mL | MEAN |
| SECONDARY | Time to Angiomyolipoma Response - Only Everolimus Patients With Angiomyolipoma Response | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years | The analysis was performed on the Full analysis Set for patients in Everolimus arm and who experienced an angiomyolipoma response. | months | MEDIAN |
| SECONDARY | Duration of Angiomyolipoma Response - Only Everolimus Patients With Angiomyolipoma Response | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years | The analysis was performed on the Full analysis Set for patients in Everolimus arm and who experienced an angiomyolipoma response. | months | MEDIAN |
| SECONDARY | Duration of Skin Lesion Response - Only Everolimus Patients With Best Overall Skin Lesion Response of Complete Clinical Response (CCR) or Partial Response (PR) | From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years | The analysis was performed on the Full analysis Set for patients in Everolimus arm and who experienced a best overall skin lesion response CCR or PR. | months | MEDIAN |
Source ClinicalTrials.gov
Results by group45
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Angiomyolipoma Response Rate as Per Central Radiology Review | Everolimus Randomized (Core Period) | 41.8 | 95% Confidence Interval 30.8 to 53.4 | Percentage of Participants | |
| Angiomyolipoma Response Rate as Per Central Radiology Review | Placebo Randomized (Core Period) | 0 | 95% Confidence Interval 0.0 to 9.0 | Percentage of Participants | |
| Angiomyolipoma Response Rate as Per Central Radiology Review | Everolimus (Core and/or Extension Period) | 58.0 | 95% Confidence Interval 48.3 to 67.3 | Percentage of Participants | |
| Time to Angiomyolipoma Progression as Per Central Radiology Review | Everolimus Randomized (Core Period) | NA | 95% Confidence Interval NA to NA | months | |
| Time to Angiomyolipoma Progression as Per Central Radiology Review | Placebo Randomized (Core Period) | 11.37 | 95% Confidence Interval 11.07 to NA | months | |
| Time to Angiomyolipoma Progression as Per Central Radiology Review | Everolimus (Core and/or Extension Period) | NA | 95% Confidence Interval NA to NA | months | |
| Skin Lesion Response Rate as Per Investigator (Only Patients With at Least One Skin Lesion at Baseline) | Everolimus Randomized (Core Period) | 26 | 95% Confidence Interval 16.6 to 37.2 | Percentage of participants | |
| Skin Lesion Response Rate as Per Investigator (Only Patients With at Least One Skin Lesion at Baseline) | Placebo Randomized (Core Period) | 0 | 95% Confidence Interval 0.0 to 9.5 | Percentage of participants | |
| Skin Lesion Response Rate as Per Investigator (Only Patients With at Least One Skin Lesion at Baseline) | Everolimus (Core and/or Extension Period) | 68.2 | 95% Confidence Interval 58.5 to 76.9 | Percentage of participants | |
| Percentage of Participants With Renal Impairment | Everolimus Randomized (Core Period) | Glomerular filtration rate <30 ml/min/1.73m^2 | 2.5 | Percentage of Participants |
1 to 10 of 45Source ClinicalTrials.gov
See all 45 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-blind Period (Core Phase) | STARTED | Everolimus | 79 | |
| Double-blind Period (Core Phase) | STARTED | Placebo | 39 | |
| Double-blind Period (Core Phase) | COMPLETED | Everolimus | 72 | |
| Double-blind Period (Core Phase) | COMPLETED | Placebo | 26 | |
| Double-blind Period (Core Phase) | NOT COMPLETED | Everolimus | 7 | |
| Double-blind Period (Core Phase) | NOT COMPLETED | Placebo | 13 | |
| Everolimus Period (Core or Extension) | STARTED | Everolimus | 112 | |
| Everolimus Period (Core or Extension) | STARTED | Placebo | 0 | |
| Everolimus Period (Core or Extension) | COMPLETED | Everolimus | 83 | |
| Everolimus Period (Core or Extension) | COMPLETED | Placebo | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events726
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 | Anaemia | Blood and lymphatic system disorders | Everolimus Randomized (Core & Ext) | 9 | 79 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo Randomized/Crossed Over to Everolimus | 6 | 33 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo Randomized/Never Crossed-over | 0 | 6 | |
| other | Leukopenia | Blood and lymphatic system disorders | Everolimus Randomized (Core & Ext) | 9 | 79 | |
| other | Leukopenia | Blood and lymphatic system disorders | Placebo Randomized/Crossed Over to Everolimus | 7 | 33 | |
| other | Leukopenia | Blood and lymphatic system disorders | Placebo Randomized/Never Crossed-over | 0 | 6 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Everolimus Randomized (Core & Ext) | 5 | 79 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Placebo Randomized/Crossed Over to Everolimus | 5 | 33 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Placebo Randomized/Never Crossed-over | 0 | 6 | |
| other | Neutropenia | Blood and lymphatic system disorders | Everolimus Randomized (Core & Ext) | 7 | 79 |
1 to 10 of 726Source ClinicalTrials.gov
See all 726 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Everolimus Randomized (Core & Ext) | ||
| Total, all-cause mortality | Placebo Randomized/Crossed Over to Everolimus | ||
| Total, all-cause mortality | Placebo Randomized/Never Crossed-over | ||
| Total, other adverse events | Everolimus Randomized (Core & Ext) | 79 | 79 |
| Total, other adverse events | Placebo Randomized/Crossed Over to Everolimus | 33 | 33 |
| Total, other adverse events | Placebo Randomized/Never Crossed-over | 5 | 6 |
| Total, serious adverse events | Everolimus Randomized (Core & Ext) | 28 | 79 |
| Total, serious adverse events | Placebo Randomized/Crossed Over to Everolimus | 17 | 33 |
| Total, serious adverse events | Placebo Randomized/Never Crossed-over | 3 | 6 |
Source ClinicalTrials.gov
Papers5
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/NCT00790400
GET https://conductscience.org/api/trials/v1/NCT00790400/results?page=2