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Trial record 76 of 2733 for:    Neuroendocrine Tumors | Neuroendocrine Tumors

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)

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ClinicalTrials.gov Identifier: NCT01595009
Recruitment Status : Completed
First Posted : May 9, 2012
Results First Posted : November 29, 2018
Last Update Posted : November 29, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Neuroendocrine Tumors
Intervention Drug: Everolimus (RAD001)
Enrollment 246
Recruitment Details  
Pre-assignment Details  
Arm/Group Title pNET (Core) Non-pNET (Core) Lung NET (E1) GI NET (E1)
Hide Arm/Group Description Participants with NETs of a pancreatic origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason. Participants with NETs of a GI or lung origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason. Participants with NETs of a lung origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason. Participants with NETs of a GI origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Period Title: Core
Started 126 120 0 0
Completed 0 0 0 0
Not Completed 126 120 0 0
Reason Not Completed
Patients completed per protocol             85             44             0             0
Adverse Event             19             23             0             0
Disease progression             14             26             0             0
Withdrawal by Subject             4             17             0             0
Death             3             6             0             0
Lost to Follow-up             1             2             0             0
Protocol deviation             0             1             0             0
Administrative problems             0             1             0             0
Period Title: Extension
Started 0 0 4 11
Completed 0 0 0 0
Not Completed 0 0 4 11
Reason Not Completed
Administrative problems             0             0             0             1
Patients completed per protocol             0             0             0             2
Disease progression             0             0             4             7
Adverse Event             0             0             0             1
Arm/Group Title pNET (Core) Non-pNET (Core) Total
Hide Arm/Group Description Participants with NETs of a pancreatic origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason. Participants with NETs of a GI or lung origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason. Total of all reporting groups
Overall Number of Baseline Participants 126 120 246
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 126 participants 120 participants 246 participants
59.3  (13.04) 64.4  (9.28) 61.8  (11.62)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 126 participants 120 participants 246 participants
Female
58
  46.0%
61
  50.8%
119
  48.4%
Male
68
  54.0%
59
  49.2%
127
  51.6%
1.Primary Outcome
Title Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core)
Hide Description The number of participants with AEs, SAEs and deaths were assessed.
Time Frame from the day of first treatment up to 19 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title pNET (Core) Non-pNET (Core)
Hide Arm/Group Description:
Participants with NETs of a pancreatic origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants with NETs of a GI or lung origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 123 117
Measure Type: Count of Participants
Unit of Measure: Participants
Adverse events
90
  73.2%
109
  93.2%
Serious adverse events
25
  20.3%
59
  50.4%
Deaths
5
   4.1%
10
   8.5%
2.Primary Outcome
Title Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths During the Extension Phase (E1)
Hide Description The number of participants with AEs, SAEs and deaths were assessed.
Time Frame from the first day of treatment in the extension up to 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lung NET (E1) GI NET
Hide Arm/Group Description:
Participants with NETs of a lung origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants with NETs of a GI origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 4 11
Measure Type: Count of Participants
Unit of Measure: Participants
AEs
4
 100.0%
11
 100.0%
SAEs
1
  25.0%
4
  36.4%
Deaths
0
   0.0%
1
   9.1%
3.Secondary Outcome
Title Investigator-assessed Progression Free Survival (PFS) (Core)
Hide Description 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.
Time Frame from the day of first treatment up to 19 months
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set, which consisted of all participants who received at least one dose, was analyzed.
Arm/Group Title pNET (Core) Non-pNET (Core)
Hide Arm/Group Description:
Participants with NETs of a pancreatic origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants with NETs of a GI or lung origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 126 120
Median (95% Confidence Interval)
Unit of Measure: Months
7.62
(5.52 to 7.62)
10.78 [1] 
(8.77 to NA)
[1]
Upper limit of the 95% confidence interval was not estimable because there were too few participants at this time point.
4.Secondary Outcome
Title Mean European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30) Score (Core)
Hide Description 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.
Time Frame Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title pNET (Core) Non-pNET (Core)
Hide Arm/Group Description:
Participants with NETs of a pancreatic origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants with NETs of a GI or lung origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 126 120
Mean (Standard Deviation)
Unit of Measure: units on a scale
Global health status/QOL, Baseline Number Analyzed 120 participants 110 participants
64.93  (20.946) 60.53  (21.497)
Global health status/QOL, Week 4 Number Analyzed 102 participants 102 participants
63.15  (19.673) 58.91  (20.622)
Global health status/QOL, Week 8 Number Analyzed 72 participants 86 participants
59.72  (21.303) 56.59  (20.417)
Global health status/QOL,Week 20 Number Analyzed 28 participants 57 participants
63.10  (22.387) 59.65  (17.663)
Global health status/QOL, Week 32 Number Analyzed 5 participants 39 participants
50.0  (26.352) 56.20  (20.297)
Global health status/QOL, Week 44 Number Analyzed 3 participants 14 participants
61.11  (9.623) 57.74  (25.205)
Global health status/QOL, EOT up to Week 82 Number Analyzed 87 participants 73 participants
61.40  (20.847) 48.86  (27.823)
Physical functioning, Baseline Number Analyzed 120 participants 111 participants
79.89  (20.850) 72.97  (22.938)
Physical functioning, week 4 Number Analyzed 103 participants 103 participants
77.02  (22.380) 68.62  (23.648)
Physical functioning, week 8 Number Analyzed 74 participants 88 participants
76.22  (25.810) 67.48  (24.973)
Physical functioning, week 20 Number Analyzed 29 participants 57 participants
79.77  (18.212) 73.71  (21.351)
Physical functioning, week 32 Number Analyzed 5 participants 39 participants
68.00  (26.415) 71.62  (23.729)
Physical functioning, week 44 Number Analyzed 3 participants 14 participants
68.89  (13.878) 76.19  (22.602)
Physical functioning, EOT up to week 82 Number Analyzed 86 participants 76 participants
75.70  (24.509) 60.94  (28.037)
Role functioning, Baseline Number Analyzed 120 participants 110 participants
75.28  (28.663) 67.27  (32.397)
Role functioning, week 4 Number Analyzed 103 participants 102 participants
72.49  (28.364) 62.25  (30.483)
Role functioning, week 8 Number Analyzed 71 participants 88 participants
71.83  (30.025) 58.14  (31.460)
Role functioning, week 20 Number Analyzed 28 participants 56 participants
79.76  (25.803) 58.33  (29.129)
Role functioning, week 32 Number Analyzed 5 participants 38 participants
66.67  (26.352) 55.26  (35.325)
Role functioning, week 44 Number Analyzed 3 participants 14 participants
61.11  (9.623) 66.67  (26.954)
Role functioning, EOT up to week 82 Number Analyzed 85 participants 77 participants
67.84  (31.788) 49.13  (33.212)
Emotional functioning, Baseline Number Analyzed 120 participants 110 participants
74.79  (22.650) 67.20  (22.988)
Emotional functioning, week 4 Number Analyzed 102 participants 103 participants
76.31  (20.991) 66.69  (25.380)
Emotional functioning, week 8 Number Analyzed 72 participants 88 participants
73.38  (24.730) 64.02  (26.640)
Emotional functioning, week 20 Number Analyzed 28 participants 57 participants
76.19  (19.072) 63.16  (27.456)
Emotional functioning, week 32 Number Analyzed 5 participants 39 participants
81.67  (14.907) 61.11  (28.184)
Emotional functioning, week 44 Number Analyzed 3 participants 14 participants
75.00  (8.333) 65.67  (31.059)
Emotional functioning, EOT up to week 82 Number Analyzed 86 participants 75 participants
74.61  (23.602) 57.63  (25.841)
Cognitive functioning, Baseline Number Analyzed 119 participants 110 participants
84.17  (19.630) 80.61  (23.842)
Cognitive functioning, week 4 Number Analyzed 102 participants 103 participants
86.11  (20.050) 78.80  (23.708)
Cognitive functioning, week 8 Number Analyzed 72 participants 88 participants
81.94  (21.803) 76.33  (27.418)
Cognitive functioning, week 20 Number Analyzed 28 participants 57 participants
83.93  (19.501) 78.65  (23.307)
Cognitive functioning, week 32 Number Analyzed 5 participants 39 participants
86.67  (18.257) 75.21  (24.445)
Cognitive functioning, week 44 Number Analyzed 3 participants 14 participants
94.44  (9.623) 84.52  (20.111)
Cognitive functioning, EOT up to week 82 Number Analyzed 84 participants 76 participants
81.35  (21.241) 73.46  (24.522)
Social functioning, Baseline Number Analyzed 120 participants 111 participants
75.56  (28.249) 67.42  (28.989)
Social functioning, week 4 Number Analyzed 101 participants 103 participants
78.71  (23.347) 67.80  (30.093)
Social functioning, week 8 Number Analyzed 72 participants 86 participants
75.69  (24.695) 65.31  (29.396)
Social functioning, week 20 Number Analyzed 28 participants 56 participants
73.21  (24.148) 66.07  (29.977)
Social functioning, week 32 Number Analyzed 5 participants 39 participants
60.00  (27.889) 66.24  (32.329)
Social functioning, week 44 Number Analyzed 3 participants 14 participants
66.67  (0.000) 72.62  (31.082)
Social functioning, EOT up to week 82 Number Analyzed 85 participants 75 participants
75.10  (27.533) 52.89  (33.933)
Fatigue, baseline Number Analyzed 120 participants 111 participants
31.30  (22.361) 42.39  (28.534)
Fatigue, week 4 Number Analyzed 103 participants 103 participants
35.22  (22.887) 43.58  (28.035)
Fatigue, week 8 Number Analyzed 73 participants 88 participants
36.91  (25.515) 48.86  (29.878)
Fatigue, week 20 Number Analyzed 29 participants 57 participants
34.29  (18.430) 44.44  (27.698)
Fatigue, week 32 Number Analyzed 5 participants 39 participants
42.22  (18.257) 48.72  (29.575)
Fatigue, week 44 Number Analyzed 3 participants 14 participants
37.04  (6.415) 38.89  (26.777)
Fatigue, EOT up to week 82 Number Analyzed 87 participants 77 participants
38.83  (24.896) 56.49  (30.474)
Nausea and vomiting, Baseline Number Analyzed 120 participants 111 participants
11.25  (17.442) 9.46  (17.353)
Nausea and vomiting, week 4 Number Analyzed 103 participants 101 participants
10.52  (15.998) 7.43  (14.995)
Nausea and vomiting, week 8 Number Analyzed 73 participants 87 participants
9.59  (17.984) 10.34  (18.015)
Nausea and vomiting, week 20 Number Analyzed 29 participants 57 participants
8.05  (15.185) 6.14  (12.048)
Nausea and vomiting, week 32 Number Analyzed 5 participants 39 participants
6.67  (9.129) 5.98  (11.139)
Nausea and vomiting, week 44 Number Analyzed 3 participants 14 participants
11.11  (9.623) 4.76  (10.187)
Nausea and vomiting, EOT up to week 82 Number Analyzed 86 participants 77 participants
9.69  (17.048) 15.80  (27.023)
Pain, Baseline Number Analyzed 119 participants 110 participants
21.57  (24.582) 31.21  (31.535)
Pain, week 4 Number Analyzed 101 participants 102 participants
25.91  (26.716) 30.88  (29.796)
Pain, week 8 Number Analyzed 72 participants 87 participants
25.23  (27.972) 37.55  (32.372)
Pain, week 20 Number Analyzed 28 participants 57 participants
27.98  (22.247) 33.33  (32.581)
Pain, week 32 Number Analyzed 5 participants 39 participants
43.33  (27.889) 34.62  (32.977)
Pain, week 44 Number Analyzed 3 participants 14 participants
38.89  (9.623) 26.19  (30.462)
Pain, EOT up to week 82 Number Analyzed 86 participants 71 participants
27.33  (28.343) 43.90  (33.362)
Dyspnea, Baseline Number Analyzed 120 participants 111 participants
16.11  (22.448) 24.32  (29.110)
Dyspnea, week 4 Number Analyzed 103 participants 102 participants
17.80  (24.171) 26.14  (28.388)
Dyspnea, week 8 Number Analyzed 73 participants 87 participants
21.00  (27.502) 29.12  (31.665)
Dyspnea, week 20 Number Analyzed 29 participants 57 participants
11.49  (18.422) 28.07  (30.072)
Dyspnea, week 32 Number Analyzed 5 participants 39 participants
13.33  (18.257) 35.90  (31.885)
Dyspnea, week 44 Number Analyzed 3 participants 14 participants
22.22  (19.245) 21.43  (28.063)
Dyspnea, EOT up to week 82 Number Analyzed 86 participants 77 participants
25.19  (30.222) 33.77  (30.824)
Insomnia, Baseline Number Analyzed 120 participants 110 participants
24.72  (30.093) 28.18  (32.929)
Insomnia, week 4 Number Analyzed 102 participants 103 participants
27.78  (29.324) 32.36  (30.769)
Insomnia, week 8 Number Analyzed 73 participants 85 participants
32.88  (30.171) 29.02  (28.540)
Insomnia, week 24 Number Analyzed 29 participants 57 participants
24.14  (26.572) 35.09  (32.380)
Insomnia, week 32 Number Analyzed 5 participants 38 participants
26.67  (27.889) 33.33  (32.880)
Insomnia, week 48 Number Analyzed 3 participants 14 participants
22.22  (19.245) 28.57  (36.648)
Insomnia, EOT up o week 82 Number Analyzed 86 participants 77 participants
32.17  (31.289) 34.63  (32.643)
Appetite loss, Baseline Number Analyzed 120 participants 111 participants
20.56  (30.613) 18.62  (29.365)
Appetitie loss, week 4 Number Analyzed 102 participants 102 participants
25.16  (31.618) 21.90  (29.849)
Appetitie loss, week 8 Number Analyzed 73 participants 87 participants
24.66  (30.946) 25.67  (31.623)
Appetitie loss, week 24 Number Analyzed 29 participants 57 participants
18.39  (27.583) 20.47  (27.998)
Appetitie loss, week 36 Number Analyzed 5 participants 39 participants
20.00  (18.257) 23.93  (30.540)
Appetitie loss, week 44 Number Analyzed 3 participants 14 participants
11.11  (19.245) 26.19  (29.753)
Appetitie loss, EOT up to week 82 Number Analyzed 87 participants 76 participants
24.14  (29.505) 33.77  (37.906)
Constipation, Baseline Number Analyzed 120 participants 111 participants
13.06  (24.175) 13.21  (27.072)
Constipation, week 4 Number Analyzed 103 participants 103 participants
11.00  (24.427) 11.33  (24.501)
Constipation, week 8 Number Analyzed 73 participants 88 participants
10.50  (24.137) 7.58  (22.447)
Constipation, week 20 Number Analyzed 29 participants 57 participants
6.90  (16.377) 8.77  (21.387)
Constipation, week 32 Number Analyzed 5 participants 39 participants
0.00  (0.000) 4.27  (13.636)
Constipation, week 44 Number Analyzed 3 participants 14 participants
0.00  (0.000) 2.38  (8.909)
Constipation, EOT up to week 82 Number Analyzed 86 participants 77 participants
12.79  (24.611) 10.39  (23.107)
Diarrhea, Baseline Number Analyzed 119 participants 110 participants
19.89  (29.535) 36.67  (38.828)
Diarrhea, week 4 Number Analyzed 102 participants 103 participants
20.92  (25.658) 38.19  (36.577)
Diarrhea, week 8 Number Analyzed 73 participants 88 participants
27.40  (29.576) 42.42  (39.708)
Diarrhea, week 20 Number Analyzed 28 participants 57 participants
16.67  (21.276) 31.58  (29.828)
Diarrhea, week 32 Number Analyzed 5 participants 39 participants
13.33  (18.257) 40.17  (36.015)
Diarrhea, week 44 Number Analyzed 3 participants 14 participants
22.22  (19.245) 40.48  (37.390)
Diarrhea, EOT up to week 82 Number Analyzed 86 participants 76 participants
25.97  (32.095) 39.04  (38.639)
Financial difficulties, Baseline Number Analyzed 119 participants 111 participants
18.77  (26.975) 16.52  (25.770)
Financial difficulties, week 4 Number Analyzed 100 participants 100 participants
16.67  (24.845) 16.67  (27.010)
Financial difficulties, week 8 Number Analyzed 72 participants 87 participants
19.44  (26.091) 16.86  (24.837)
Financial difficulties, week 20 Number Analyzed 28 participants 57 participants
21.43  (22.616) 19.88  (27.357)
Financial difficulties, week 32 Number Analyzed 5 participants 39 participants
40.00  (27.889) 21.37  (28.084)
Financial difficulties, week 44 Number Analyzed 3 participants 14 participants
22.22  (19.245) 19.05  (25.198)
Financial difficulties, EOT up to week 82 Number Analyzed 86 participants 76 participants
21.71  (27.897) 27.63  (30.496)
5.Secondary Outcome
Title Mean EORTC QLQ-G.I. NET21 Score (Core)
Hide Description 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.
Time Frame Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title pNET (Core) Non-pNET (Core)
Hide Arm/Group Description:
Participants with NETs of a pancreatic origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants with NETs of a GI or lung origin received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 126 120
Mean (Standard Deviation)
Unit of Measure: units on a scale
Endocrine, Baseline Number Analyzed 65 participants 111 participants
12.99  (20.372) 23.52  (23.771)
Endocrine, week 4 Number Analyzed 53 participants 99 participants
13.21  (17.440) 17.85  (21.403)
Endocrine, week 8 Number Analyzed 33 participants 88 participants
13.47  (17.293) 16.79  (20.216)
Endocrine, week 20 Number Analyzed 8 participants 56 participants
12.50  (12.511) 15.87  (21.067)
Endocrine, week 32 Number Analyzed 4 participants 38 participants
16.67  (14.344) 13.74  (17.978)
Endocrine, week 44 Number Analyzed 1 participants 14 participants
0.00 [1]   (NA) 7.14  (11.202)
Endocrine, EOT up to week 82 Number Analyzed 41 participants 76 participants
9.49  (15.627) 15.20  (18.755)
G.I., Baseline Number Analyzed 65 participants 111 participants
23.69  (20.183) 26.19  (21.328)
G.I., week 4 Number Analyzed 53 participants 99 participants
23.02  (20.488) 23.39  (18.958)
G.I., week 8 Number Analyzed 34 participants 89 participants
22.34  (18.398) 27.53  (21.341)
G.I., week 20 Number Analyzed 8 participants 56 participants
25.63  (14.056) 22.31  (18.837)
G.I., week 32 Number Analyzed 4 participants 38 participants
25.00  (11.386) 26.84  (19.879)
G.I., week 44 Number Analyzed 1 participants 14 participants
0.00 [1]   (NA) 20.95  (16.714)
G.I., EOT up to week 82 Number Analyzed 41 participants 76 participants
25.85  (19.899) 30.48  (24.484)
Treatment, baseline Number Analyzed 33 participants 69 participants
9.26  (11.675) 18.44  (19.374)
Treatment, week 4 Number Analyzed 42 participants 84 participants
17.72  (15.337) 22.22  (20.077)
Treatment, week 8 Number Analyzed 23 participants 77 participants
16.18  (19.528) 20.27  (17.283)
Treatment, week 20 Number Analyzed 6 participants 47 participants
28.70  (21.493) 19.74  (19.755)
Treatment, week 32 Number Analyzed 2 participants 27 participants
27.78  (7.857) 24.49  (23.684)
Treatment, week 44 Number Analyzed 1 participants 11 participants
16.67 [1]   (NA) 18.18  (18.936)
Treatment, EOT up to week 82 Number Analyzed 35 participants 58 participants
22.06  (21.705) 24.81  (21.735)
Social function, Baseline Number Analyzed 66 participants 110 participants
39.56  (23.721) 47.42  (25.726)
Social function, week 4 Number Analyzed 54 participants 100 participants
36.52  (26.294) 45.28  (27.483)
Social function, week 8 Number Analyzed 31 participants 87 participants
38.35  (23.629) 46.49  (26.265)
Social function, week 20 Number Analyzed 8 participants 56 participants
40.28  (14.472) 42.66  (25.648)
Social function, week 32 Number Analyzed 4 participants 38 participants
38.89  (14.344) 48.54  (23.735)
Social function, week 44 Number Analyzed 1 participants 14 participants
33.33 [1]   (NA) 40.08  (26.165)
Social function, EOT up to week 82 Number Analyzed 43 participants 75 participants
38.89  (25.051) 54.22  (28.428)
Disease-related worries, Baseline Number Analyzed 66 participants 110 participants
43.10  (25.599) 53.94  (27.664)
Disease-related worries, week 4 Number Analyzed 54 participants 100 participants
39.20  (28.562) 46.33  (28.165)
Disease-related worries, week 8 Number Analyzed 30 participants 87 participants
40.56  (28.183) 47.32  (28.541)
Disease-related worries, week 20 Number Analyzed 8 participants 56 participants
37.50  (8.267) 46.83  (32.096)
Disease-related worries, week 32 Number Analyzed 4 participants 38 participants
38.89  (11.111) 53.51  (27.137)
Disease-related worries, week 44 Number Analyzed 1 participants 14 participants
33.33 [2]   (NA) 44.05  (32.647)
Disease-related worries, EOT up to week 82 Number Analyzed 43 participants 75 participants
42.89  (28.876) 55.04  (31.667)
Muscle/bone pain, Baseline Number Analyzed 65 participants 109 participants
26.67  (33.347) 29.66  (31.210)
Muscle/bone pain, week 4 Number Analyzed 54 participants 100 participants
24.07  (29.966) 33.00  (31.956)
Muscle/bone pain, week 8 Number Analyzed 31 participants 88 participants
31.18  (34.357) 33.33  (33.524)
Muscle/bone pain, week 20 Number Analyzed 8 participants 56 participants
41.67  (15.430) 34.52  (33.614)
Muscle/bone pain, week 32 Number Analyzed 4 participants 38 participants
50.00  (19.245) 40.35  (34.795)
Muscle/bone pain, week 44 Number Analyzed 1 participants 14 participants
33.33 [2]   (NA) 26.19  (26.726)
Muscle/bone pain, EOT up to week 82 Number Analyzed 42 participants 75 participants
34.13  (28.973) 40.89  (33.141)
Sexual function, Baseline Number Analyzed 47 participants 78 participants
35.46  (32.899) 39.32  (40.468)
Sexual function, week 4 Number Analyzed 38 participants 75 participants
21.05  (26.191) 40.89  (40.483)
Sexual function, week 8 Number Analyzed 22 participants 63 participants
28.79  (33.007) 43.92  (40.083)
Sexual function, week 20 Number Analyzed 3 participants 35 participants
33.33  (33.333) 38.10  (39.724)
Sexual function, week 32 Number Analyzed 3 participants 24 participants
11.11  (19.245) 29.17  (35.864)
Sexual function, week 44 Number Analyzed 0 participants 7 participants
47.62  (46.576)
Sexual function, EOT up to week 82 Number Analyzed 31 participants 47 participants
25.81  (28.166) 48.94  (43.323)
Communication function, Baseline Number Analyzed 66 participants 108 participants
7.58  (16.325) 9.88  (22.904)
Communication function, week 4 Number Analyzed 53 participants 98 participants
8.18  (17.179) 6.46  (15.626)
Communication function, week 8 Number Analyzed 30 participants 86 participants
12.22  (18.535) 9.69  (19.714)
Communication function, week 20 Number Analyzed 7 participants 55 participants
14.29  (17.817) 10.30  (19.108)
Communication function, week 32 Number Analyzed 4 participants 38 participants
8.33  (16.667) 7.89  (19.658)
Communication function, week 44 Number Analyzed 1 participants 14 participants
0.00 [2]   (NA) 4.76  (12.105)
Communication function, EOT up to week 82 Number Analyzed 42 participants 74 participants
12.70  (22.028) 12.61  (23.214)
Body image, Baseline Number Analyzed 65 participants 107 participants
15.90  (28.932) 22.43  (34.195)
Body image, week 4 Number Analyzed 53 participants 98 participants
15.09  (27.399) 22.79  (31.239)
Body image, week 8 Number Analyzed 31 participants 83 participants
21.51  (27.953) 26.10  (31.256)
Body image, week 20 Number Analyzed 8 participants 55 participants
16.67  (17.817) 30.30  (34.708)
Body image, week 32 Number Analyzed 4 participants 37 participants
0.00  (0.000) 28.83  (33.483)
Body image, week 44 Number Analyzed 1 participants 14 participants
0.00 [2]   (NA) 30.95  (27.625)
Body image, EOT up to week 82 Number Analyzed 42 participants 73 participants
23.02  (32.500) 34.70  (37.449)
[1]
Standard deviation does not apply when n = 1.
[2]
Standard deviation does not apply when n=1.
6.Secondary Outcome
Title Number and Percentage of Participants With Ratings of 'no Problem, 'Some Problem' and 'Extreme Problem' in the EuroQol Five Dimensions Questionnaire (EQ-5D) (Core)
Hide Description 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.
Time Frame Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set, which consisted of all participants who received at least one dose of everolimus, was analyzed.
Arm/Group Title pNET (Core) Non-pNET (Core)
Hide Arm/Group Description:
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 126 120
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline, Mobility, No problem Number Analyzed 126 participants 120 participants
84
  66.7%
76
  63.3%
Baseline, Mobility, Some problem Number Analyzed 126 participants 120 participants
31
  24.6%
35
  29.2%
Baseline, Mobility, Extreme problem Number Analyzed 126 participants 120 participants
2
   1.6%
1
   0.8%
Baseline, Self-care, No problem Number Analyzed 126 participants 120 participants
100
  79.4%
102
  85.0%
Baseline, Self-care, Some problem Number Analyzed 126 participants 120 participants
15
  11.9%
10
   8.3%
Baseline, Self-care, Extreme problem Number Analyzed 126 participants 0 participants
2
   1.6%
Baseline, Usual activities, No problem Number Analyzed 126 participants 120 participants
71
  56.3%
60
  50.0%
Baseline, Usual activities, Some problem Number Analyzed 126 participants 120 participants
43
  34.1%
46
  38.3%
Baseline, Usual actvities, Extreme problem Number Analyzed 126 participants 120 participants
4
   3.2%
6
   5.0%
Baseline, Pain/Discomfort, No problem Number Analyzed 126 participants 120 participants
59
  46.8%
40
  33.3%
Baseline, Pain/discomfort, Some problem Number Analyzed 126 participants 120 participants
52
  41.3%
66
  55.0%
Baseline, Pain/discomfort, Extreme problem Number Analyzed 126 participants 120 participants
6
   4.8%
7
   5.8%
Baseline, Anxiety/depression, No problem Number Analyzed 126 participants 120 participants
65
  51.6%
53
  44.2%
Baseline, Anxiety/depression, Some problem Number Analyzed 126 participants 120 participants
47
  37.3%
54
  45.0%
Baseline, Anxiety/depression, Extreme problem Number Analyzed 126 participants 120 participants
5
   4.0%
6
   5.0%
Week 4, Mobility, No problem Number Analyzed 126 participants 120 participants
72
  57.1%
66
  55.0%
Week 4, Mobility, Some problem Number Analyzed 126 participants 120 participants
27
  21.4%
37
  30.8%
Week 4, Mobility, Extreme problem Number Analyzed 126 participants 120 participants
2
   1.6%
1
   0.8%
Week 4, Self-care, No problem Number Analyzed 126 participants 120 participants
89
  70.6%
86
  71.7%
Week 4, Self-care, Some problem Number Analyzed 126 participants 120 participants
10
   7.9%
16
  13.3%
Week 4, Self-care, Extreme problem Number Analyzed 126 participants 120 participants
3
   2.4%
2
   1.7%
Week 4, Usual activities, No problem Number Analyzed 126 participants 120 participants
66
  52.4%
55
  45.8%
Week 4, Usual activities, Some problem Number Analyzed 126 participants 120 participants
31
  24.6%
42
  35.0%
Week 4, Usual activities, Extreme problem Number Analyzed 126 participants 120 participants
4
   3.2%
7
   5.8%
Week 4, Pain/discomfort, No problem Number Analyzed 126 participants 120 participants
39
  31.0%
30
  25.0%
Week 4, Pain/discomfort, Some problem Number Analyzed 126 participants 120 participants
58
  46.0%
64
  53.3%
Week 4, Pain/discomfort, Extreme problem Number Analyzed 126 participants 120 participants
3
   2.4%
8
   6.7%
Week 4, Anxiety/depression, No problem Number Analyzed 126 participants 120 participants
54
  42.9%
57
  47.5%
Week 4, Anxiety depression, Some problem Number Analyzed 126 participants 120 participants
46
  36.5%
43
  35.8%
Week 4, Anxiety/depression, Extreme problem Number Analyzed 126 participants 120 participants
1
   0.8%
3
   2.5%
Week 8, Mobility, No problem Number Analyzed 126 participants 120 participants
48
  38.1%
45
  37.5%
Week 8, Mobility, Some problem Number Analyzed 126 participants 120 participants
24
  19.0%
37
  30.8%
Week 8, Mobility, Extreme problem Number Analyzed 126 participants 120 participants
2
   1.6%
3
   2.5%
Week 8, Self-care, No problem Number Analyzed 126 participants 120 participants
61
  48.4%
70
  58.3%
Week 8, Self-care, Some problem Number Analyzed 126 participants 120 participants
7
   5.6%
15
  12.5%
Week 8, Self-care, Extreme problem Number Analyzed 126 participants 120 participants
6
   4.8%
1
   0.8%
Week 8, Usual activities, No problem Number Analyzed 126 participants 120 participants
46
  36.5%
40
  33.3%
Week 8, Usual activities, Some problem Number Analyzed 126 participants 120 participants
23
  18.3%
41
  34.2%
Week 8, Usual activties, Extreme problem Number Analyzed 126 participants 120 participants
4
   3.2%
5
   4.2%
Week 8, Pain/discomfort, No problem Number Analyzed 126 participants 120 participants
34
  27.0%
25
  20.8%
Week 8, Pain/discomfort, Some problem Number Analyzed 126 participants 120 participants
32
  25.4%
52
  43.3%
Week 8, Pain/discomfort, Extreme problem Number Analyzed 126 participants 120 participants
7
   5.6%
10
   8.3%
Week 8, Anxiety/depression, No problem Number Analyzed 126 participants 120 participants
43
  34.1%
38
  31.7%
Week 8, Anxiety/depression, Some problem Number Analyzed 126 participants 120 participants
30
  23.8%
41
  34.2%
Week 8, Anxiety/depression, Extreme problem Number Analyzed 126 participants 120 participants
1
   0.8%
8
   6.7%
Week 20, Mobility, No problem Number Analyzed 126 participants 120 participants
26
  20.6%
37
  30.8%
Week 20, Mobility, Some problem Number Analyzed 126 participants 120 participants
3
   2.4%
17
  14.2%
Week 20, Mobility, Extreme problem Number Analyzed 0 participants 120 participants
1
   0.8%
Week 20, Self-care, No problem Number Analyzed 126 participants 120 participants
25
  19.8%
49
  40.8%
Week 20, Self-care, Some problem Number Analyzed 126 participants 120 participants
4
   3.2%
6
   5.0%
Week 20, Self-care, Extreme problem Number Analyzed 0 participants 0 participants
Week 20, Usual activities, No problem Number Analyzed 126 participants 120 participants
18
  14.3%
30
  25.0%
Week 20 Usual activities, Some problem Number Analyzed 126 participants 120 participants
10
   7.9%
22
  18.3%
Week 20, Usual activities, Extreme problem Number Analyzed 126 participants 120 participants
1
   0.8%
2
   1.7%
Week 20, Pain/discomfort, No problem Number Analyzed 126 participants 120 participants
12
   9.5%
19
  15.8%
Week 20, Pain/discomfort, Some problem Number Analyzed 126 participants 120 participants
16
  12.7%
34
  28.3%
Week 20, Pain/discomfort, Extreme problem Number Analyzed 126 participants 120 participants
1
   0.8%
2
   1.7%
Week 20, Anxiety/depression, No problem Number Analyzed 126 participants 120 participants
16
  12.7%
27
  22.5%
Week 20, Anxiety/depression, Some problem Number Analyzed 126 participants 120 participants
12
   9.5%
23
  19.2%
Week 20, Anxiety/depression, Extreme problem Number Analyzed 126 participants 120 participants
1
   0.8%
5
   4.2%
Week 32, Mobility, No problem Number Analyzed 126 participants 120 participants
3
   2.4%
25
  20.8%
Week 32, Mobility, Some problem Number Analyzed 126 participants 120 participants
2
   1.6%
12
  10.0%
Week 32, Mobility, Extreme problem Number Analyzed 0 participants 120 participants
2
   1.7%
Week 32, Self-care, No problem Number Analyzed 126 participants 120 participants
5
   4.0%
32
  26.7%
Week 32, Self-care, Some problem Number Analyzed 0 participants 120 participants
3
   2.5%
Week 32, Self-care, Extreme problem Number Analyzed 0 participants 120 participants
2
   1.7%
Week 32, Usual activities, No problem Number Analyzed 126 participants 120 participants
4
   3.2%
22
  18.3%
Week 32, Usual activities, Some problem Number Analyzed 126 participants 120 participants
1
   0.8%
14
  11.7%
Week 32, Usual activities, Extreme problem Number Analyzed 0 participants 120 participants
3
   2.5%
Week 32, Pain/discomfort, No problem Number Analyzed 126 participants 120 participants
1
   0.8%
10
   8.3%
Week 32, Pain/discomfort, Some problem Number Analyzed 126 participants 120 participants
4
   3.2%
27
  22.5%
Week 32, Pain/discomfort, Extreme problem Number Analyzed 0 participants 120 participants
2
   1.7%
Week 32, Anxiety/depression, No problem Number Analyzed 126 participants 120 participants
4
   3.2%
20
  16.7%
Week 32, Anxiety/depression, Some problem Number Analyzed 126 participants 120 participants
1
   0.8%
14
  11.7%
Week 32, Anxiety/depression, Extreme problem Number Analyzed 0 participants 120 participants
5
   4.2%
Week 44, Mobility, No problem Number Analyzed 126 participants 120 participants
2
   1.6%
9
   7.5%
Week 44, Mobility, Some problem Number Analyzed 126 participants 120 participants
1
   0.8%
4
   3.3%
Week 44, Mobility, Extreme problem Number Analyzed 0 participants 120 participants
1
   0.8%
Week 44, Self-care, No problem Number Analyzed 126 participants 120 participants
3
   2.4%
12
  10.0%
Week 44, Self-care, Some problem Number Analyzed 0 participants 120 participants
2
   1.7%
Week 44, Self-care, Extreme problem Number Analyzed 0 participants 0 participants
Week 44, Usual activities, No problem Number Analyzed 0 participants 120 participants
6
   5.0%
Week 44, usual activities, Some problem Number Analyzed 126 participants 120 participants
3
   2.4%
7
   5.8%
Week 44, usual activities, Extreme problem Number Analyzed 0 participants 0 participants
Week 44, Pain/discomfort, No problem Number Analyzed 0 participants 120 participants
6
   5.0%
Week 44, Pain/discomfort, Some problem Number Analyzed 126 participants 120 participants
3
   2.4%
8
   6.7%
Week 44, Pain/discomfort, Extreme problem Number Analyzed 0 participants 0 participants
Week 44, Anxiry/depression, No problem Number Analyzed 126 participants 120 participants
1
   0.8%
7
   5.8%
Week 44, Anxiety/depression, Some problem Number Analyzed 126 participants 120 participants
2
   1.6%
6
   5.0%
Week 44, Anxiety/depression, Extreme problem Number Analyzed 0 participants 120 participants
1
   0.8%
EOT up to Week 82, Mobility, No problem Number Analyzed 126 participants 120 participants
59
  46.8%
42
  35.0%
EOT up to Week 82, Mobility, Some problem Number Analyzed 126 participants 120 participants
23
  18.3%
31
  25.8%
EOT up to Week 82, Mobility, Extreme problem Number Analyzed 126 participants 120 participants
4
   3.2%
4
   3.3%
EOT up to Week 82, Self-care, No problem Number Analyzed 126 participants 120 participants
69
  54.8%
56
  46.7%
EOT up to Week 82, Self-care, Some problem Number Analyzed 126 participants 120 participants
12
   9.5%
19
  15.8%
EOT up to Week 82, Self-care, Extreme problem Number Analyzed 126 participants 120 participants
2
   1.6%
1
   0.8%
EOT up toWeek 82, Usual activities, No problem Number Analyzed 126 participants 120 participants
54
  42.9%
29
  24.2%
EOT up to Week 82, Usual activities, Some problem Number Analyzed 126 participants 120 participants
24
  19.0%
36
  30.0%
EOT up to Week 82, Usual activities, Extreme prob. Number Analyzed 126 participants 120 participants
5
   4.0%
11
   9.2%
EOT up to Week 82, Pain/discomfort, No problem Number Analyzed 126 participants 120 participants
43
  34.1%
18
  15.0%
EOT up to Week 82, Pain/discomfort, Some problem Number Analyzed 126 participants 120 participants
36
  28.6%
48
  40.0%
EOT up to Week 82, Pain/discomfort, Extreme prob. Number Analyzed 126 participants 120 participants
5
   4.0%
11
   9.2%
EOT up to Week 82, Anxiety/depression, No problem Number Analyzed 126 participants 120 participants
48
  38.1%
31
  25.8%
EOT up to Week 82, Anxiety/depression, Some prob. Number Analyzed 126 participants 120 participants
33
  26.2%
40
  33.3%
EOT up toWeek 82, Anxiety/depression, Extreme prob Number Analyzed 126 participants 120 participants
3
   2.4%
6
   5.0%
7.Secondary Outcome
Title Mean EQ-5D Visual Analogue Scale (VAS) Score (Core)
Hide Description 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.
Time Frame Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title pNET (Core) Non-pNET (Core)
Hide Arm/Group Description:
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 126 120
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 118 participants 111 participants
68.8  (19.90) 63.9  (19.09)
Week 4 Number Analyzed 100 participants 104 participants
69.3  (18.06) 63.8  (18.78)
Week 8 Number Analyzed 73 participants 88 participants
64.6  (21.77) 61.2  (18.65)
Week 20 Number Analyzed 28 participants 54 participants
67.4  (19.58) 64.6  (15.51)
Week 32 Number Analyzed 5 participants 37 participants
42.6  (24.67) 62.0  (17.65)
Week 44 Number Analyzed 3 participants 13 participants
66.7  (15.28) 68.6  (18.71)
EOT up to Week 82 Number Analyzed 86 participants 76 participants
66.5  (20.64) 55.3  (23.02)
8.Secondary Outcome
Title Investigator-assessed Best Overall Response (Core)
Hide Description 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.
Time Frame from the start of treatment, every 12 weeks for the first year and then every 6 months up to 19 months
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set, which consisted of all participants who received at least one dose, was analyzed.
Arm/Group Title pNET (Core) Non-pNET (Core)
Hide Arm/Group Description:
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 126 120
Measure Type: Count of Participants
Unit of Measure: Participants
Partial response (PR)
2
   1.6%
1
   0.8%
Stable disease (SD)
74
  58.7%
84
  70.0%
Progressive disease (PD)
9
   7.1%
8
   6.7%
Unknown
41
  32.5%
27
  22.5%
Objective response rate (complete response or PR)
2
   1.6%
1
   0.8%
9.Secondary Outcome
Title Investigator-assessed Progression Free Survival (PFS) (E1)
Hide Description 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.
Time Frame from first date of treatment in the extension up to 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set, which consisted of all participants who received at least one dose of everolimus during the extension, was analyzed
Arm/Group Title Lung NET (E1) GI NET (E1)
Hide Arm/Group Description:
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 4 11
Median (95% Confidence Interval)
Unit of Measure: Days
159
(10 to 1074)
655 [1] 
(9 to NA)
[1]
The upper limit of the 95% confidence interval was not estimable since there were too few number of participants at this time point.
10.Secondary Outcome
Title 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)
Hide Description 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.
Time Frame baseline, every 12 weeks and up to 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lung NET (E1) GI NET
Hide Arm/Group Description:
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 1 6
Mean (Standard Deviation)
Unit of Measure: score on a scale
Global health status/QoL 0.00 [1]   (NA) -0.00  (11.785)
Functional scale: Physical functioning 6.67 [1]   (NA) 0.00  (7.303)
Functional scale: Role functioning -16.67 [1]   (NA) 5.56  (22.771)
Functional scale: Emotional functioning -25.00 [1]   (NA) -1.85  (17.003)
Functional scale: Cognitive functioning -66.67 [1]   (NA) -16.67  (21.082)
Functional scale: Social functioning 0.00 [1]   (NA) -8.33  (9.129)
Symptom scale: Fatigue 0.00 [1]   (NA) -3.70  (22.951)
Symptom scale: Nausea and vomiting 0.00 [1]   (NA) 0.00  (18.257)
Symptom scale: Pain 0.00 [1]   (NA) 16.67  (27.889)
Symptom scale: Dyspnea 0.00 [1]   (NA) -5.56  (13.608)
Symptom scale: Insomnia 33.33 [1]   (NA) 11.11  (17.213)
Symptom scale: Appetite loss 0.00 [1]   (NA) 0.00  (21.082)
Symptom scale: Constipation 0.00 [1]   (NA) 5.56  (25.092)
Symptom scale: Diarrhea 0.00 [1]   (NA) -16.67  (45.947)
Symptom scale: Financial difficulties -33.33 [1]   (NA) 16.67  (18.257)
[1]
Standard deviation does not apply when n=1.
11.Secondary Outcome
Title Change in EORTC QLQ-G.I. NET21 Score at the End of Treatment From Baseline (Baseline = First Day of Treatment in the Extension) (E1)
Hide Description 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, 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 outcomes. A positive change from baseline indicates worsening.
Time Frame baseline, every 12 weeks and up to 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lung NET (E1) GI NET
Hide Arm/Group Description:
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 4 11
Mean (Standard Deviation)
Unit of Measure: units on a scale
Social function Number Analyzed 1 participants 6 participants
11.11 [1]   (NA) 7.41  (22.951)
Disease-related worries Number Analyzed 1 participants 6 participants
44.44 [1]   (NA) -0.93  (7.384)
Sexual function Number Analyzed 0 participants 1 participants
50.00  (43.033)
Communicative functioning Number Analyzed 1 participants 6 participants
0.00 [1]   (NA) 11.11  (17.213)
Endocrine scale Number Analyzed 1 participants 6 participants
11.11 [1]   (NA) 5.56  (18.257)
G.I. scale Number Analyzed 1 participants 6 participants
0.00 [1]   (NA) 14.44  (23.633)
Treatment scale Number Analyzed 1 participants 5 participants
-16.67 [1]   (NA) 5.56  (12.423)
Muscle/bone pain symptom Number Analyzed 0 participants 6 participants
33.33  (29.814)
Body image Number Analyzed 1 participants 6 participants
0.00 [1]   (NA) 0.00  (21.082)
[1]
Standard deviation does not apply when n=1.
12.Secondary Outcome
Title Change in EuroQol Five Dimensions Questionnaire (EQ-5D) Score at the End of Treatment From Baseline (Baseline = First Day of Treatment in the Extension) (E1)
Hide Description The EQ-5D contains 2 sections:1st section has 1 item addressing each of 5 dimensions (mobility, self-care, usual activity, pain/discomfort, anxiety/depression). Each dimension has 3 levels: no problems, some problems, extreme problems, 1–3, respectively. A health state is defined by combining 1 level from each dimension. 243 health states are possible. Each state is referred to in a 5 digit code. E.g., state 11111 indicates no problems on any dimension, while state 11223 indicates no problems with mobility and self-care, some problems with performing usual activities, moderate pain or discomfort and extreme anxiety or depression. The 2nd section measures self-rated health status using a visual analogue scale (VAS) where 100 represents best possible health and 0 represents worst possible health. Patients are asked to rate their current health by placing a mark on the VAS. Scores from each section are then transformed into an overall score of 0 or 1, with 0= higher level of dysfunction.
Time Frame baseline, every 12 weeks and up to 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lung NET (E1) GI NET
Hide Arm/Group Description:
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 1 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
Composite health index 7.26 [1]   (NA) -8.58  (13.302)
Self-rated health status -10.00 [1]   (NA) -4.17  (21.479)
[1]
Standard deviation does not apply when n=1.
13.Secondary Outcome
Title Investigator-assessed Best Overall Response During the Extension Phase (E1)
Hide Description 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.
Time Frame from the start of treatment, every 12 weeks for the first year and then every 6 months up to 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set, which consisted of all participants who received at least one dose of everolimus during the extension, was analyzed
Arm/Group Title Lung NET (E1) GI NET
Hide Arm/Group Description:
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
Overall Number of Participants Analyzed 4 11
Measure Type: Count of Participants
Unit of Measure: Participants
Complete response
0
   0.0%
0
   0.0%
Partial response
0
   0.0%
1
   9.1%
Stable disease
2
  50.0%
7
  63.6%
Progressive disease
2
  50.0%
3
  27.3%
Unknown
0
   0.0%
0
   0.0%
Time Frame up to 19 month for the core and up to 4 years for the extension
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title pNET (Core) Non-pNET (Core) Lung NET (E1) GI NET (E1)
Hide Arm/Group Description pNET (Core) Non-pNET (Core) Lung NET (E1) GI NET (E1)
All-Cause Mortality
pNET (Core) Non-pNET (Core) Lung NET (E1) GI NET (E1)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
pNET (Core) Non-pNET (Core) Lung NET (E1) GI NET (E1)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   25/123 (20.33%)   59/117 (50.43%)   1/4 (25.00%)   4/11 (36.36%) 
Blood and lymphatic system disorders         
Anaemia  1  1/123 (0.81%)  3/117 (2.56%)  1/4 (25.00%)  0/11 (0.00%) 
Haemoglobinaemia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Thrombocytopenia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Cardiac disorders         
Atrial fibrillation  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Cardiac failure  1  1/123 (0.81%)  2/117 (1.71%)  1/4 (25.00%)  0/11 (0.00%) 
Coronary artery disease  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Mitral valve incompetence  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Right ventricular failure  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Tricuspid valve incompetence  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Eye disorders         
Retinal vascular thrombosis  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Gastrointestinal disorders         
Abdominal hernia  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Abdominal pain  1  1/123 (0.81%)  5/117 (4.27%)  0/4 (0.00%)  1/11 (9.09%) 
Ascites  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Constipation  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Diarrhoea  1  3/123 (2.44%)  4/117 (3.42%)  0/4 (0.00%)  0/11 (0.00%) 
Dysphagia  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Faecal incontinence  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Faecaloma  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Gastric ulcer perforation  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Gastritis haemorrhagic  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Gastrointestinal haemorrhage  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Ileitis  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Inguinal hernia  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Intestinal obstruction  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Nausea  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  0/11 (0.00%) 
Pancreatitis  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Peptic ulcer  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Proctalgia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Stomatitis  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Subileus  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  0/11 (0.00%) 
Tongue oedema  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  0/11 (0.00%) 
Varices oesophageal  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Vomiting  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
General disorders         
Asthenia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Chills  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Death  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Device occlusion  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Euthanasia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Fatigue  1  0/123 (0.00%)  5/117 (4.27%)  0/4 (0.00%)  0/11 (0.00%) 
General physical health deterioration  1  2/123 (1.63%)  7/117 (5.98%)  0/4 (0.00%)  1/11 (9.09%) 
Impaired healing  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Multi-organ failure  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Oedema  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Oedema peripheral  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Pyrexia  1  2/123 (1.63%)  3/117 (2.56%)  0/4 (0.00%)  0/11 (0.00%) 
Hepatobiliary disorders         
Cholangitis  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Cholecystitis  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Cholecystocholangitis  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Cholestasis  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Hepatic failure  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Hyperbilirubinaemia  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  0/11 (0.00%) 
Infections and infestations         
Abscess limb  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Arthritis infective  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Cholecystitis infective  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Cystitis  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Gastrointestinal infection  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Herpes zoster  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Infection  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Peritonitis  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  0/11 (0.00%) 
Peritonitis bacterial  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Pneumonia  1  2/123 (1.63%)  6/117 (5.13%)  0/4 (0.00%)  0/11 (0.00%) 
Pyelonephritis acute  1  2/123 (1.63%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Sepsis  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Septic shock  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Sinusitis  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Upper respiratory tract infection  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Urinary tract infection  1  2/123 (1.63%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Urinary tract infection bacterial  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Injury, poisoning and procedural complications         
Femur fracture  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Limb traumatic amputation  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Ulna fracture  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Investigations         
Antineutrophil cytoplasmic antibody increased  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Blood cholesterol increased  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Blood uric acid increased  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
C-reactive protein increased  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Weight decreased  1  1/123 (0.81%)  2/117 (1.71%)  0/4 (0.00%)  1/11 (9.09%) 
White blood cell count increased  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Metabolism and nutrition disorders         
Decreased appetite  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Diabetic ketoacidosis  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Hypercalcaemia  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Hyperglycaemia  1  2/123 (1.63%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Hypertriglyceridaemia  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Hypocalcaemia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Hypoglycaemia  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Hyponatraemia  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Hypophosphataemia  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Musculoskeletal and connective tissue disorders         
Hypercreatinaemia  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Pain in extremity  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Breast cancer  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Metastases to liver  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Metastases to pancreas  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Neoplasm progression  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Neuroendocrine tumour  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  0/11 (0.00%) 
Prostate cancer  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Vipoma  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Nervous system disorders         
Hepatic encephalopathy  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Hypoglycaemic seizure  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Ischaemic stroke  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Loss of consciousness  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Peripheral sensory neuropathy  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Psychiatric disorders         
Depression  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Major depression  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Renal and urinary disorders         
Calculus urinary  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Cystitis haemorrhagic  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Renal failure acute  1  3/123 (2.44%)  2/117 (1.71%)  0/4 (0.00%)  0/11 (0.00%) 
Reproductive system and breast disorders         
Metrorrhagia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Acute respiratory distress syndrome  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Dyspnoea  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Epistaxis  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Lung infiltration  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Pleural effusion  1  0/123 (0.00%)  1/117 (0.85%)  1/4 (25.00%)  0/11 (0.00%) 
Pneumonitis  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  0/11 (0.00%) 
Pulmonary oedema  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Respiratory failure  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  0/11 (0.00%) 
Skin and subcutaneous tissue disorders         
Rash  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Vascular disorders         
Circulatory collapse  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  0/11 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (19.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
pNET (Core) Non-pNET (Core) Lung NET (E1) GI NET (E1)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   73/123 (59.35%)   89/117 (76.07%)   4/4 (100.00%)   11/11 (100.00%) 
Blood and lymphatic system disorders         
Anaemia  1  7/123 (5.69%)  10/117 (8.55%)  1/4 (25.00%)  3/11 (27.27%) 
Thrombocytopenia  1  12/123 (9.76%)  5/117 (4.27%)  1/4 (25.00%)  1/11 (9.09%) 
Cardiac disorders         
Tachyarrhythmia  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Ear and labyrinth disorders         
Vertigo  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Eye disorders         
Cataract  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Eyelid oedema  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Gastrointestinal disorders         
Abdominal hernia  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Abdominal pain  1  4/123 (3.25%)  10/117 (8.55%)  0/4 (0.00%)  5/11 (45.45%) 
Abdominal pain upper  1  3/123 (2.44%)  4/117 (3.42%)  0/4 (0.00%)  1/11 (9.09%) 
Anal haemorrhage  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Ascites  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Bowel movement irregularity  1  0/123 (0.00%)  0/117 (0.00%)  1/4 (25.00%)  0/11 (0.00%) 
Constipation  1  0/123 (0.00%)  4/117 (3.42%)  2/4 (50.00%)  1/11 (9.09%) 
Diarrhoea  1  10/123 (8.13%)  34/117 (29.06%)  0/4 (0.00%)  3/11 (27.27%) 
Dry mouth  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  2/11 (18.18%) 
Flatulence  1  0/123 (0.00%)  1/117 (0.85%)  2/4 (50.00%)  3/11 (27.27%) 
Frequent bowel movements  1  0/123 (0.00%)  0/117 (0.00%)  1/4 (25.00%)  0/11 (0.00%) 
Inguinal hernia  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Mouth ulceration  1  2/123 (1.63%)  2/117 (1.71%)  0/4 (0.00%)  1/11 (9.09%) 
Nausea  1  10/123 (8.13%)  10/117 (8.55%)  1/4 (25.00%)  0/11 (0.00%) 
Stomatitis  1  11/123 (8.94%)  13/117 (11.11%)  0/4 (0.00%)  2/11 (18.18%) 
Vomiting  1  2/123 (1.63%)  5/117 (4.27%)  1/4 (25.00%)  0/11 (0.00%) 
General disorders         
Asthenia  1  12/123 (9.76%)  6/117 (5.13%)  0/4 (0.00%)  1/11 (9.09%) 
Chest pain  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Fatigue  1  1/123 (0.81%)  13/117 (11.11%)  0/4 (0.00%)  3/11 (27.27%) 
Generalised oedema  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Inflammation  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Injection site reaction  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Mucosal inflammation  1  12/123 (9.76%)  5/117 (4.27%)  0/4 (0.00%)  0/11 (0.00%) 
Oedema  1  1/123 (0.81%)  3/117 (2.56%)  1/4 (25.00%)  0/11 (0.00%) 
Oedema peripheral  1  5/123 (4.07%)  15/117 (12.82%)  0/4 (0.00%)  4/11 (36.36%) 
Peripheral swelling  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Pyrexia  1  8/123 (6.50%)  5/117 (4.27%)  0/4 (0.00%)  2/11 (18.18%) 
Immune system disorders         
Hypersensitivity  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Infections and infestations         
Bronchitis  1  1/123 (0.81%)  2/117 (1.71%)  2/4 (50.00%)  0/11 (0.00%) 
Conjunctivitis  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Cystitis  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  2/11 (18.18%) 
Erysipelas  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  1/11 (9.09%) 
Gastroenteritis  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Gastrointestinal infection  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  2/11 (18.18%) 
Laryngitis  1  0/123 (0.00%)  1/117 (0.85%)  1/4 (25.00%)  0/11 (0.00%) 
Nasopharyngitis  1  2/123 (1.63%)  5/117 (4.27%)  2/4 (50.00%)  5/11 (45.45%) 
Otitis media acute  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Paronychia  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Pulpitis dental  1  0/123 (0.00%)  3/117 (2.56%)  0/4 (0.00%)  2/11 (18.18%) 
Pyelonephritis  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Pyelonephritis acute  1  0/123 (0.00%)  0/117 (0.00%)  1/4 (25.00%)  0/11 (0.00%) 
Respiratory tract infection  1  0/123 (0.00%)  4/117 (3.42%)  0/4 (0.00%)  1/11 (9.09%) 
Rhinitis  1  2/123 (1.63%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Sinusitis  1  2/123 (1.63%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Urinary tract infection  1  1/123 (0.81%)  5/117 (4.27%)  0/4 (0.00%)  2/11 (18.18%) 
Injury, poisoning and procedural complications         
Incisional hernia  1  0/123 (0.00%)  0/117 (0.00%)  1/4 (25.00%)  0/11 (0.00%) 
Ligament sprain  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  2/11 (18.18%) 
Investigations         
Blood creatine phosphokinase increased  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Blood creatinine increased  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  2/11 (18.18%) 
Blood glucose increased  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Blood lactate dehydrogenase increased  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Blood uric acid increased  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Weight decreased  1  0/123 (0.00%)  3/117 (2.56%)  0/4 (0.00%)  2/11 (18.18%) 
Metabolism and nutrition disorders         
Decreased appetite  1  6/123 (4.88%)  9/117 (7.69%)  0/4 (0.00%)  0/11 (0.00%) 
Diabetes mellitus  1  0/123 (0.00%)  2/117 (1.71%)  0/4 (0.00%)  1/11 (9.09%) 
Hypercholesterolaemia  1  3/123 (2.44%)  5/117 (4.27%)  1/4 (25.00%)  1/11 (9.09%) 
Hyperglycaemia  1  13/123 (10.57%)  6/117 (5.13%)  0/4 (0.00%)  0/11 (0.00%) 
Hyperkalaemia  1  0/123 (0.00%)  0/117 (0.00%)  1/4 (25.00%)  0/11 (0.00%) 
Hypertriglyceridaemia  1  3/123 (2.44%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Hypoglycaemia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Hypokalaemia  1  2/123 (1.63%)  6/117 (5.13%)  0/4 (0.00%)  0/11 (0.00%) 
Musculoskeletal and connective tissue disorders         
Arthralgia  1  2/123 (1.63%)  2/117 (1.71%)  0/4 (0.00%)  2/11 (18.18%) 
Arthritis  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Back pain  1  0/123 (0.00%)  6/117 (5.13%)  0/4 (0.00%)  4/11 (36.36%) 
Muscle spasms  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Muscle tightness  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Myalgia  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Osteitis  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Pain in extremity  1  0/123 (0.00%)  3/117 (2.56%)  0/4 (0.00%)  2/11 (18.18%) 
Nervous system disorders         
Cervicobrachial syndrome  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Headache  1  4/123 (3.25%)  9/117 (7.69%)  0/4 (0.00%)  0/11 (0.00%) 
Psychiatric disorders         
Depressed mood  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Depression  1  1/123 (0.81%)  3/117 (2.56%)  0/4 (0.00%)  1/11 (9.09%) 
Sleep disorder  1  0/123 (0.00%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Renal and urinary disorders         
Chronic kidney disease  1  0/123 (0.00%)  0/117 (0.00%)  1/4 (25.00%)  1/11 (9.09%) 
Haematuria  1  0/123 (0.00%)  0/117 (0.00%)  1/4 (25.00%)  0/11 (0.00%) 
Nephrolithiasis  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Renal cyst haemorrhage  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Respiratory, thoracic and mediastinal disorders         
Chronic obstructive pulmonary disease  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Cough  1  3/123 (2.44%)  6/117 (5.13%)  0/4 (0.00%)  2/11 (18.18%) 
Dyspnoea  1  5/123 (4.07%)  6/117 (5.13%)  1/4 (25.00%)  2/11 (18.18%) 
Dyspnoea exertional  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Epistaxis  1  2/123 (1.63%)  3/117 (2.56%)  0/4 (0.00%)  2/11 (18.18%) 
Pleural effusion  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Skin and subcutaneous tissue disorders         
Angioedema  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  2/11 (18.18%) 
Dermatitis  1  3/123 (2.44%)  0/117 (0.00%)  1/4 (25.00%)  1/11 (9.09%) 
Dry skin  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Hair growth abnormal  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Nail dystrophy  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  2/11 (18.18%) 
Onychoclasis  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  2/11 (18.18%) 
Panniculitis  1  0/123 (0.00%)  0/117 (0.00%)  1/4 (25.00%)  0/11 (0.00%) 
Pityriasis rosea  1  0/123 (0.00%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Pruritus  1  1/123 (0.81%)  4/117 (3.42%)  0/4 (0.00%)  1/11 (9.09%) 
Rash  1  9/123 (7.32%)  7/117 (5.98%)  0/4 (0.00%)  2/11 (18.18%) 
Vascular disorders         
Haematoma  1  1/123 (0.81%)  0/117 (0.00%)  0/4 (0.00%)  1/11 (9.09%) 
Hypertension  1  5/123 (4.07%)  6/117 (5.13%)  0/4 (0.00%)  5/11 (45.45%) 
Hypotension  1  1/123 (0.81%)  1/117 (0.85%)  0/4 (0.00%)  1/11 (9.09%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (19.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Results Point of Contact
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-1873
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01595009     History of Changes
Other Study ID Numbers: CRAD001K24133
First Submitted: May 7, 2012
First Posted: May 9, 2012
Results First Submitted: August 7, 2017
Results First Posted: November 29, 2018
Last Update Posted: November 29, 2018