Trial · NCT01524783
Everolimus Plus Best Supportive Care vs Placebo Plus Best Supportive Care in the Treatment of Patients With Advanced Neuroendocrine Tumors (GI or Lung Origin)
NCT01524783, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2016-12-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Everolimus Plus Best Supportive Care vs Placebo Plus Best Supportive Care in the Treatment of Patients With Advanced Neuroendocrine Tumors (GI or Lung Origin). Trials, record NCT01524783. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01524783
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Everolimus Plus Best Supportive Care vs Placebo Plus Best Supportive Care in the Treatment of Patients With Advanced Neuroendocrine Tumors (GI or Lung Origin)
- NCT number
- NCT01524783
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 302 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Advanced NET of GI Origin, Advanced NET of Lung Origin, Neuroendocrine Tumors
- Interventions
- Everolimus, Placebo, Best suportive care (BSC)
- First posted
- 2012-02-02
- Last updated
- 2021-08-05
- Other registries' IDs
- eudract:2011-002887-26
- Results first posted
- 2016-12-28
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | From date of randomization to progression or death, whichever comes first, assessed up to 27 months | PFS is defined as the time from randomization to the date of the first documented tumor progression or death from any cause, whichever comes first. Progression was defined using modified RECIST 1.0 and as per central radiology assessment as at least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline. Progression was assessed by cat scan (CT) and/or magnetic resonance imaging (MRI). For participants who had not progressed or died at the analysis cut-off date, PFS was censored at the date of the last adequate tumor evaluation date. An adequate tumour assessment is a tumour assessment with an overall response other than unknown. The percentage event-free probability estimate is the estimated probability that a patient will remain event-free in PFS up to the specified time point. |
| Secondary | Overall Survival (OS) | From date of randomization to date of death, assessed up to approximately 8 years | OS is defined as the time from the date of randomization to date of death due to any cause. If a death had not been observed by the date of analysis cut-off, then OS was censored at the date of last contact. All participants randomized to placebo arm who crossed over to everolimus were censored. |
| Secondary | Overall Response Rate (ORR) as Per Modified RECIST 1.0 According to Central Evaluation | From randomization until end of treatment, assessed up to approximately 2.5 years | ORR is defined as the proportion of patients with best overall response (BOR) of complete response (CR) or partial response (PR), according to central evaluation and as per modified RECIST 1.0. CR: disappearance of all target lesions. PR: At least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the baseline sum of the longest diameters. |
| Secondary | Disease Control Rate (DCR) Based on Modified RECIST 1.0 and as Per Central Radiology Assessment | From randomization until end of treatment, assessed up to approximately 2.5 years | DCR is defined as the proportion of subjects with best overall response of CR or PR or stable disease based on modified RECIST 1.0 and as per central radiology assessment. CR: disappearance of all target lesions. PR: At least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the baseline sum of the longest diameters. Stable disease: Neither sufficient shrinkage to qualify for PR or CR nor an increase in lesions which would qualify for progression. |
| Secondary | Time to Definitive Deterioration in Functional Assessment of Cancer Therapy - General (FACT-G) Questionnaire Total Score | From randomization to definitive deterioration of FACT-G total score, assessed up to approximately 3 years | FACT-G is a self-assessed health-related quality of life questionnaire. The questionnaire is comprised of 27 questions examining physical, social/family, emotional, and functional well-being. Participants responded to the items on a five-point scale, ranging from 0: "Not at all" to 4: "Very much." The total score ranges from 0 to 108, with higher scores indicating a better patient-reported outcome/quality of life. Definitive deterioration is defined as a decrease in the total score by at least 7 points compared to baseline with no further improvement. Death was considered as worsening of the FACT-G total score if it occurred close to the last available assessment, where "close" was defined as twice the planned period between two assessments. Patients without definitive worsening prior to analysis cut-off or prior to start of another anticancer therapy were censored at the date of their last assessment. |
| Secondary | Change From Baseline in Chromogranin A (CgA) Levels | From baseline (every 4 weeks) up to 116 weeks | CgA is a potential biomarker for tumor response. Blood samples were collected for assessment of CgA levels. Change from Baseline at a particular visit was calculated as the CgA level at that visit minus Baseline. |
| Secondary | Change From Baseline in Neuron Specific Enolase (NSE) Levels | From baseline (every 4 weeks) up to Week 116 | NSE is a potential biomarker for tumor response. Blood samples were collected for assessment of NSE levels. Change from Baseline at a particular visit was calculated as the NSE level at that visit minus Baseline. |
| Secondary | Time to Definitive Deterioration in World Health Organization (WHO) Performance Status (PS) Change | From randomization to definitive deterioration of WHO performance status, assessed up to approximately 3 years | WHO PS is a scale rated from 0 (fully active) to 5 (death) by a healthcare professional to assess the overall status of a patient: a lower score represents a higher ability to perform daily tasks. Deterioration is defined as an increase of at least one point compared to baseline. Deterioration is considered definitive if no improvements in the WHO PS status is observed at a subsequent time of measurement during the treatment period following the time point where the deterioration is observed. Death was considered as worsening of the WHO PS if it occurred close to the last available assessment, where "close" was defined as twice the planned period between two assessments. Patients without definitive worsening prior to analysis cut-off or prior to start of another anticancer therapy were censored at the date of their last assessment. |
| Secondary | Pharmacokinetics (PK): Predose Concentration (Cmin) of Everolimus at Day 29 | Pre-dose at Day 29. | A pre-dose blood sample at day 29 was collected to determine the exposure of everolimus at the steady-state pre-dose concentration (Cmin). Cmin is provided for participants randomized to everolimus+BSC who received 10mg of everolimus daily and also for participants randomized to everolimus+BSC who received 5mg of everolimus daily which was required for a number of participants in the study experiencing adverse events requiring dose modifications |
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 | Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | From date of randomization to progression or death, whichever comes first, assessed up to 27 months | The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization. | Percent event-free probability in PFS | NUMBER |
| SECONDARY | Overall Survival (OS) | From date of randomization to date of death, assessed up to approximately 8 years | The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization. | Months | MEDIAN |
| SECONDARY | Overall Response Rate (ORR) as Per Modified RECIST 1.0 According to Central Evaluation | From randomization until end of treatment, assessed up to approximately 2.5 years | The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization. | Percentage of participants | NUMBER |
| SECONDARY | Disease Control Rate (DCR) Based on Modified RECIST 1.0 and as Per Central Radiology Assessment | From randomization until end of treatment, assessed up to approximately 2.5 years | The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization. | Percentage of participants | NUMBER |
| SECONDARY | Time to Definitive Deterioration in Functional Assessment of Cancer Therapy - General (FACT-G) Questionnaire Total Score | From randomization to definitive deterioration of FACT-G total score, assessed up to approximately 3 years | The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization. | Months | MEDIAN |
| SECONDARY | Change From Baseline in Chromogranin A (CgA) Levels | From baseline (every 4 weeks) up to 116 weeks | The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization. Number analyzed signified number of participants with available data for this outcome measure at specified timepoints. | microgram/liter (ug/L) | MEAN |
| SECONDARY | Change From Baseline in Neuron Specific Enolase (NSE) Levels | From baseline (every 4 weeks) up to Week 116 | The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization. Number analyzed signified number of participants with available data for this outcome measure at specified timepoints. | microgram/liter (ug/L) | MEAN |
| SECONDARY | Time to Definitive Deterioration in World Health Organization (WHO) Performance Status (PS) Change | From randomization to definitive deterioration of WHO performance status, assessed up to approximately 3 years | The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization. | Months | MEDIAN |
| SECONDARY | Pharmacokinetics (PK): Predose Concentration (Cmin) of Everolimus at Day 29 | Pre-dose at Day 29. | All patients who received at least one dose of the study drug with evaluable blood samples for this endpoint. | nanogram/milliliter (ng/mL) | MEAN |
| POST_HOC | All Collected Deaths | On-treatment deaths: up to approximately 8 years. All deaths: up to approximately 8 years | The safety set consists of all participants who received at least one dose of the study drug and had at least one post-baseline safety evaluation. Participants were analyzed according to treatment actually received: One participant randomized to everolimus arm received only placebo and therefore, appears in the placebo arm in the safety set | Participants | NUMBER |
Source ClinicalTrials.gov
Results by group158
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Everolimus + BSC | 2 months | 90.1 | 95% Confidence Interval 84.8 to 93.5 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Placebo + BSC | 2 months | 74.6 | 95% Confidence Interval 64.3 to 82.4 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Everolimus + BSC | 4 months | 81.2 | 95% Confidence Interval 74.9 to 86.2 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Placebo + BSC | 4 months | 49.1 | 95% Confidence Interval 38.1 to 59.2 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Everolimus + BSC | 6 months | 72.1 | 95% Confidence Interval 65.0 to 78.0 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Placebo + BSC | 6 months | 40.1 | 95% Confidence Interval 29.5 to 50.5 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Everolimus + BSC | 8 months | 62.4 | 95% Confidence Interval 54.8 to 69.1 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Placebo + BSC | 8 months | 35.8 | 95% Confidence Interval 25.4 to 46.2 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Everolimus + BSC | 10 months | 51.7 | 95% Confidence Interval 44.0 to 59.0 | Percent event-free probability in PFS |
| Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment | Placebo + BSC | 10 months | 31.3 | 95% Confidence Interval 21.3 to 41.7 | Percent event-free probability in PFS |
1 to 10 of 158Source ClinicalTrials.gov
See all 158 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Blinded Period | STARTED | Everolimus + BSC (Throughout Study) | 205 | |
| Blinded Period | STARTED | Placebo+BSC (Blinded Period) | 97 | |
| Blinded Period | STARTED | Everolimus+BSC (Crossover) | 0 | |
| Blinded Period | COMPLETED | Everolimus + BSC (Throughout Study) | 26 | |
| Blinded Period | COMPLETED | Placebo+BSC (Blinded Period) | 6 | |
| Blinded Period | COMPLETED | Everolimus+BSC (Crossover) | 0 | |
| Blinded Period | NOT COMPLETED | Everolimus + BSC (Throughout Study) | 179 | |
| Blinded Period | NOT COMPLETED | Placebo+BSC (Blinded Period) | 91 | |
| Blinded Period | NOT COMPLETED | Everolimus+BSC (Crossover) | 0 | |
| Open-label Period | STARTED | Everolimus + BSC (Throughout Study) | 26 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events968
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+BSC (Throughout Study) | 47 | 202 | |
| other | Anaemia | Blood and lymphatic system disorders | Everolimus +BSC (Crossover) | 0 | 6 | |
| other | Anaemia | Blood and lymphatic system disorders | Everolimus+BSC (All) | 47 | 208 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo+BSC (Blinded Period) | 9 | 98 | |
| other | Deafness | Ear and labyrinth disorders | Everolimus+BSC (Throughout Study) | 1 | 202 | |
| other | Deafness | Ear and labyrinth disorders | Everolimus +BSC (Crossover) | 1 | 6 | |
| other | Deafness | Ear and labyrinth disorders | Everolimus+BSC (All) | 2 | 208 | |
| other | Deafness | Ear and labyrinth disorders | Placebo+BSC (Blinded Period) | 0 | 98 | |
| other | Vertigo | Ear and labyrinth disorders | Everolimus+BSC (Throughout Study) | 3 | 202 | |
| other | Vertigo | Ear and labyrinth disorders | Everolimus +BSC (Crossover) | 2 | 6 |
1 to 10 of 968Source ClinicalTrials.gov
See all 968 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Everolimus+BSC (Throughout Study) | 10 | 202 |
| Total, all-cause mortality | Everolimus +BSC (Crossover) | 0 | 6 |
| Total, all-cause mortality | Everolimus+BSC (All) | 10 | 208 |
| Total, all-cause mortality | Placebo+BSC (Blinded Period) | 5 | 98 |
| Total, other adverse events | Everolimus+BSC (Throughout Study) | 197 | 202 |
| Total, other adverse events | Everolimus +BSC (Crossover) | 6 | 6 |
| Total, other adverse events | Everolimus+BSC (All) | 203 | 208 |
| Total, other adverse events | Placebo+BSC (Blinded Period) | 81 | 98 |
| Total, serious adverse events | Everolimus+BSC (Throughout Study) | 93 | 202 |
| Total, serious adverse events | Everolimus +BSC (Crossover) | 1 | 6 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers5
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.
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