Trial · NCT01595009
An Open-label, Multi-center, Expanded Access Study of Everolimus in Participants With Advanced Neuroendocrine Tumors (NETs) (Core Study) and an Extension Study to the Open-label, Multi-center, Expanded Access Study of Everolimus in Patients With Advanced NETs (E1)
NCT01595009, Phase 4, Completed, Novartis Pharmaceuticals; results first posted 2018-11-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open-label, Multi-center, Expanded Access Study of Everolimus in Participants With Advanced Neuroendocrine Tumors (NETs) (Core Study) and an Extension Study to the Open-label, Multi-center, Expanded Access Study of Everolimus in Patients With Advanced NETs (E1). Trials, record NCT01595009. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01595009
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
An Open-label, Multi-center, Expanded Access Study of Everolimus in Participants With Advanced Neuroendocrine Tumors (NETs) (Core Study) and an Extension Study to the Open-label, Multi-center, Expanded Access Study of Everolimus in Patients With Advanced NETs (E1)
- NCT number
- NCT01595009
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 246 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Neuroendocrine Tumors
- Interventions
- Everolimus (RAD001)
- First posted
- 2012-05-09
- Last updated
- 2018-11-29
- Results first posted
- 2018-11-29
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core) | from the day of first treatment up to 19 months | The number of participants with AEs, SAEs and deaths were assessed. |
| Primary | Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths During the Extension Phase (E1) | from the first day of treatment in the extension up to 4 years | The number of participants with AEs, SAEs and deaths were assessed. |
| Secondary | Investigator-assessed Progression Free Survival (PFS) (Core) | from the day of first treatment up to 19 months | PFS was defined as the time from the date of the first dose to the date of the first radiologically documented disease progression or death due to any cause. If a participant had not progressed or died at the study end date or when he/she received any further anti-neoplastic therapy, PFS was censored at the time of the last tumor assessment before the end of study date. Progression was defined,using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. |
| Secondary | Mean European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30) Score (Core) | Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82 | The EORTC QLQ-C30 contains 30 questions assessed by the participant. There are 9 multiple-item scales: 5 scales that assess aspects of functioning (physical, role functioning, cognitive, emotional, and social); 3 symptom scales (Fatigue, Pain, and Nausea and Vomiting); and a global health status/Quality of Life (QOL) scale. There are 5 single-item measures assessing additional symptoms (i.e., dyspnea, loss of appetite, insomnia, constipation, and diarrhea) and a single item concerning perceived financial impact of the disease. All but two questions have 4-point scales ranging from "Not at all" to "Very much." The two questions concerning global health status/ QOL have 7 point scales with ratings ranging from "Very poor" to "Excellent." For each of the 14 domains, final scores are transformed such that they range from 0-100, where higher scores indicate improvement. |
| Secondary | Mean EORTC QLQ-G.I. NET21 Score (Core) | Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82 | The EORTC QLQ-G.I. NET21 contains 21 questions and has three defined multi-item symptom scales (endocrine - 3 questions, gastrointestinal - 5 questions, and treatment related side effects - 3 questions), two single item symptoms (bone/muscle pain and concern about weight loss), two psychosocial scales (social function - 3 questions and disease-related worries - 3 questions) and two other single items (sexuality and communication). For each of the 9 domains, final scores are transformed such that they range from 0-100, where higher scores indicate worsening. |
| Secondary | Number and Percentage of Participants With Ratings of 'no Problem, 'Some Problem' and 'Extreme Problem' in the EuroQol Five Dimensions Questionnaire (EQ-5D) (Core) | Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82 | The EQ-5D is divided into two distinct sections. The first section includes one item addressing each of five dimensions (mobility, self-care, usual activity, pain/discomfort, and anxiety/depression). Patients rate each of these items from "no problem," "some problem," or "extreme problem." A composite health index is then defined by combining the levels for each dimension. The second section of the questionnaire measures self-rated (global) health status utilizing a vertically oriented visual analogue scale where 100 represents the "best possible health state" and 0 represents the "worst possible health state." Respondents are asked to rate their current health by placing a mark along this continuum. The scores from each section are then transformed into a single health utility score. Overall scores range from 0 to 1 with lower scores representing a higher level of dysfunction. |
| Secondary | Mean EQ-5D Visual Analogue Scale (VAS) Score (Core) | Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82 | The EQ-5D is divided into two distinct sections. The first section includes one item addressing each of five dimensions (mobility, self-care, usual activity, pain/discomfort, and anxiety/depression). Patients rate each of these items from "no problem," "some problem," or "extreme problem." A composite health index is then defined by combining the levels for each dimension. The second section of the questionnaire measures self-rated (global) health status utilizing a vertically oriented visual analogue scale where 100 represents the "best possible health state" and 0 represents the "worst possible health state." Respondents are asked to rate their current health by placing a mark along this continuum. The scores from each section are then transformed into a single health utility score. Overall scores range from 0 to 1 with lower scores representing a higher level of dysfunction. |
| Secondary | Investigator-assessed Best Overall Response (Core) | from the start of treatment, every 12 weeks for the first year and then every 6 months up to 19 months | Best overall response was determined from the sequence of investigator overall lesions responses according to Response Evaluation Criteria in Solid Tumors (RECIST). Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR; Progression, a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. |
| Secondary | Investigator-assessed Progression Free Survival (PFS) (E1) | from first date of treatment in the extension up to 4 years | PFS was defined as the time from the date of the start of therapy in the extension study to the date of the first radiologically documented disease progression or death due to any cause. If a participant had not progressed or died at the analysis cut-off date or when he/she received any further anti-neoplastic therapy, PFS was censored at the time of the last adequate tumor evaluation before the cut-off date or the anti-neoplastic therapy start date. |
| Secondary | Change in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30) Score at the End of Treatment From Baseline (Baseline = First Day of Treatment in the Extension) (E1) | baseline, every 12 weeks and up to 4 years | The EORTC QLQ-C30 contains 30 questions assessed by the participant. There are 9 multiple-item scales: 5 scales that assess aspects of functioning (physical, role, cognitive, emotional, and social); 3 symptom scales (Fatigue, Pain, and Nausea and Vomiting); and a global health status and QOL scale. There are 5 single-item measures assessing additional symptoms (i.e., dyspnea, loss of appetite, insomnia, constipation, and diarrhea) and a single item concerning perceived financial impact of the disease. All but two questions have 4-point scales ranging from "Not at all" to "Very much." The two questions concerning global health status and QOL have 7 point scales with ratings ranging from "Very poor" to "Excellent." For each of the 14 domains, changes are calculated as value at later time point minus value at baseline, and final scores are transformed such that they range from 0-100, where higher scores indicate better outcomes. A positive change from baseline indicates improvement. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core) | from the day of first treatment up to 19 months | The safety population, which consisted of all patients who received at least one dose of everolimus and had at least one post-baseline safety assessment, was analyzed. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths During the Extension Phase (E1) | from the first day of treatment in the extension up to 4 years | The safety set, which consisted of all participants who received at least one dose of everolimus during the extension and had at least one post-baseline safety assessment during the extension, was analyzed. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Investigator-assessed Progression Free Survival (PFS) (Core) | from the day of first treatment up to 19 months | The full analysis set, which consisted of all participants who received at least one dose, was analyzed. | Months | MEDIAN |
| SECONDARY | Mean European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30) Score (Core) | Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82 | The full analysis set, which consisted of all participants who received at least one dose of everolimus, was considered for the analysis. Only participants with measurement at each given time point were analyzed for that time point. | units on a scale | MEAN |
| SECONDARY | Mean EORTC QLQ-G.I. NET21 Score (Core) | Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82 | The full analysis set, which consisted of all participants who received at least one dose of everolimus, was considered for the analysis. Only participants with measurement at each given time point were analyzed for that time point. | units on a scale | MEAN |
| SECONDARY | Number and Percentage of Participants With Ratings of 'no Problem, 'Some Problem' and 'Extreme Problem' in the EuroQol Five Dimensions Questionnaire (EQ-5D) (Core) | Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82 | The full analysis set, which consisted of all participants who received at least one dose of everolimus, was analyzed. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean EQ-5D Visual Analogue Scale (VAS) Score (Core) | Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82 | The full analysis set, which consisted of all participants who received at least one dose of everolimus, was considered for the analysis. Only participants with measurement at each given time point were analyzed for that time point. | units on a scale | MEAN |
| SECONDARY | Investigator-assessed Best Overall Response (Core) | from the start of treatment, every 12 weeks for the first year and then every 6 months up to 19 months | The full analysis set, which consisted of all participants who received at least one dose, was analyzed. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Investigator-assessed Progression Free Survival (PFS) (E1) | from first date of treatment in the extension up to 4 years | The full analysis set, which consisted of all participants who received at least one dose of everolimus during the extension, was analyzed | Days | MEDIAN |
| SECONDARY | Change in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30) Score at the End of Treatment From Baseline (Baseline = First Day of Treatment in the Extension) (E1) | baseline, every 12 weeks and up to 4 years | Only participants from the full analysis set, who had both baseline and end of treatment measurements, were included in the analysis. The full analysis set, which consisted of all participants who received at least one dose of everolimus during the extension, was analyzed. | score on a scale | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group624
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core) | pNET (Core) | Adverse events | 90 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core) | Non-pNET (Core) | Adverse events | 109 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core) | pNET (Core) | Serious adverse events | 25 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core) | Non-pNET (Core) | Serious adverse events | 59 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core) | pNET (Core) | Deaths | 5 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core) | Non-pNET (Core) | Deaths | 10 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths During the Extension Phase (E1) | Lung NET (E1) | AEs | 4 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths During the Extension Phase (E1) | GI NET | AEs | 11 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths During the Extension Phase (E1) | Lung NET (E1) | SAEs | 1 | Participants | |
| Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths During the Extension Phase (E1) | GI NET | SAEs | 4 | Participants |
1 to 10 of 624Source ClinicalTrials.gov
See all 624 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Core | STARTED | pNET (Core) | 126 | |
| Core | STARTED | Non-pNET (Core) | 120 | |
| Core | STARTED | Lung NET (E1) | 0 | |
| Core | STARTED | GI NET (E1) | 0 | |
| Core | COMPLETED | pNET (Core) | 0 | |
| Core | COMPLETED | Non-pNET (Core) | 0 | |
| Core | COMPLETED | Lung NET (E1) | 0 | |
| Core | COMPLETED | GI NET (E1) | 0 | |
| Core | NOT COMPLETED | pNET (Core) | 126 | |
| Core | NOT COMPLETED | Non-pNET (Core) | 120 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events876
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 | pNET (Core) | 7 | 123 | |
| other | Anaemia | Blood and lymphatic system disorders | Non-pNET (Core) | 10 | 117 | |
| other | Anaemia | Blood and lymphatic system disorders | Lung NET (E1) | 1 | 4 | |
| other | Anaemia | Blood and lymphatic system disorders | GI NET (E1) | 3 | 11 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | pNET (Core) | 12 | 123 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Non-pNET (Core) | 5 | 117 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Lung NET (E1) | 1 | 4 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | GI NET (E1) | 1 | 11 | |
| other | Tachyarrhythmia | Cardiac disorders | pNET (Core) | 0 | 123 | |
| other | Tachyarrhythmia | Cardiac disorders | Non-pNET (Core) | 0 | 117 |
1 to 10 of 876Source ClinicalTrials.gov
See all 876 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | pNET (Core) | ||
| Total, all-cause mortality | Non-pNET (Core) | ||
| Total, all-cause mortality | Lung NET (E1) | ||
| Total, all-cause mortality | GI NET (E1) | ||
| Total, other adverse events | pNET (Core) | 73 | 123 |
| Total, other adverse events | Non-pNET (Core) | 89 | 117 |
| Total, other adverse events | Lung NET (E1) | 4 | 4 |
| Total, other adverse events | GI NET (E1) | 11 | 11 |
| Total, serious adverse events | pNET (Core) | 25 | 123 |
| Total, serious adverse events | Non-pNET (Core) | 59 | 117 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT01595009
GET https://conductscience.org/api/trials/v1/NCT01595009/results?page=2