Trial Outcomes & Findings for RAD001 and Erlotinib in Patients With Neuroendocrine Tumors (NCT NCT00843531)
NCT ID: NCT00843531
Last Updated: 2020-09-29
Results Overview
Objective response is defined as complete response (CR) or partial response (PR) assessed using Response Evaluation Criteria in Solid Tumors (RECIST) criteria. The primary analysis population is based on efficacy-evaluable patients, defined as those patients who receive at least one cycle of RAD001 plus erlotinib and undergo at least one post-baseline response assessment or die while on therapy.
TERMINATED
PHASE2
17 participants
Up to 2 years
2020-09-29
Participant Flow
Participant milestones
| Measure |
RAD001 and Erlotinib
RAD001 and erlotinib
Each 28 day cycle:
RAD001: 5 mg per day by mouth (self-administered) Erlotinib: 100 mg per day by mouth (self-administered)
|
|---|---|
|
Overall Study
STARTED
|
17
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
RAD001 and Erlotinib in Patients With Neuroendocrine Tumors
Baseline characteristics by cohort
| Measure |
RAD001 and Erlotinib
n=17 Participants
* everolimus 5 mg daily and continued this throughout the duration of treatment
* erlotinib at a dose of 100 mg daily with the option to dose reduce erlotinib to 100 mg daily in the setting of grade 3/4 rash or diarrhea
* cycles defined as every 28 days
|
|---|---|
|
Age, Customized
20-29 years
|
1 Participants
n=99 Participants
|
|
Age, Customized
30-39 years
|
0 Participants
n=99 Participants
|
|
Age, Customized
40-49 years
|
2 Participants
n=99 Participants
|
|
Age, Customized
50-59 years
|
2 Participants
n=99 Participants
|
|
Age, Customized
60-69 years
|
12 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
17 Participants
n=99 Participants
|
|
Neuroendocrine Tumor Type
Carcinoid Neuroendocrine Tumor
|
9 Participants
n=99 Participants
|
|
Neuroendocrine Tumor Type
Pancreatic neuroendocrine tumor (PNET)
|
8 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Up to 2 yearsObjective response is defined as complete response (CR) or partial response (PR) assessed using Response Evaluation Criteria in Solid Tumors (RECIST) criteria. The primary analysis population is based on efficacy-evaluable patients, defined as those patients who receive at least one cycle of RAD001 plus erlotinib and undergo at least one post-baseline response assessment or die while on therapy.
Outcome measures
| Measure |
RAD001 Plus Erlotinib
n=17 Participants
* everolimus 5 mg daily and continued this throughout the duration of treatment
* erlotinib at a dose of 100 mg daily with the option to dose reduce erlotinib to 100 mg daily in the setting of grade 3/4 rash or diarrhea
* cycles defined as every 28 days
|
|---|---|
|
Objective Response Rate (ORR)
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 9 monthsPrimary safety analysis will be conducted after the first 16 patients have had potential for completion of two cycles of protocol therapy. All patients receiving at least one dose of protocol therapy will be included in the analyses. All patients with treatment-related, Grade 3 or higher adverse events according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 3 resulting in a DLT will be reported.
Outcome measures
| Measure |
RAD001 Plus Erlotinib
n=17 Participants
* everolimus 5 mg daily and continued this throughout the duration of treatment
* erlotinib at a dose of 100 mg daily with the option to dose reduce erlotinib to 100 mg daily in the setting of grade 3/4 rash or diarrhea
* cycles defined as every 28 days
|
|---|---|
|
Number of Patients With Dose-limiting Toxicity (DLT)
|
8 Participants
|
SECONDARY outcome
Timeframe: Up to 2 yearsDuration of objective response will be defined as the time from the initial documentation of response to documented disease progression or death from any cause on study
Outcome measures
| Measure |
RAD001 Plus Erlotinib
n=17 Participants
* everolimus 5 mg daily and continued this throughout the duration of treatment
* erlotinib at a dose of 100 mg daily with the option to dose reduce erlotinib to 100 mg daily in the setting of grade 3/4 rash or diarrhea
* cycles defined as every 28 days
|
|---|---|
|
Duration of Objective Response
|
NA months
No participants obtained an objective response
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Data for overall survival not collected
Overall survival will be defined as the time from first day of treatment until death
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: No participants obtained an objective response (CR or PR) therefore data for long term progression-free survival data was not collected.
PFS will be defined as the time from the first day of treatment and documented objective response to time of progression or death from any cause, whichever occurs first
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Data for time to progression not collected
Time to disease progression will be defined as the time from baseline until documented disease progression or death (whichever occurs first).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Data for time to treatment failure not collected
Time to treatment failure will be defined as the time from first day of treatment until study discontinuation (for any cause).
Outcome measures
Outcome data not reported
Adverse Events
RAD001 Plus Erlotinib
Serious adverse events
| Measure |
RAD001 Plus Erlotinib
n=17 participants at risk
* everolimus 5 mg daily and continued this throughout the duration of treatment
* erlotinib at a dose of 100 mg daily with the option to dose reduce erlotinib to 100 mg daily in the setting of grade 3/4 rash or diarrhea
* cycles defined as every 28 days
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Vascular disorders
Edema: limb
|
5.9%
1/17 • Number of events 2 • Up to 3 years
|
|
Investigations
Creatinine
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain - Back
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain - Extremity-limb
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Renal and urinary disorders
Incontinence, urinary
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Renal and urinary disorders
Renal failure
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Investigations
Alkaline phosphatase
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Investigations
Pain - Other - Right upper quadrant pain
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Gastrointestinal disorders
Pain - Abdomen NOS
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
General disorders
Fever
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Gastrointestinal disorders
Distension/bloating, abdominal
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
Other adverse events
| Measure |
RAD001 Plus Erlotinib
n=17 participants at risk
* everolimus 5 mg daily and continued this throughout the duration of treatment
* erlotinib at a dose of 100 mg daily with the option to dose reduce erlotinib to 100 mg daily in the setting of grade 3/4 rash or diarrhea
* cycles defined as every 28 days
|
|---|---|
|
Gastrointestinal disorders
Stomatitis
|
76.5%
13/17 • Number of events 41 • Up to 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
88.2%
15/17 • Number of events 36 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Rash: acne/acneiform
|
64.7%
11/17 • Number of events 24 • Up to 3 years
|
|
Metabolism and nutrition disorders
Anorexia
|
64.7%
11/17 • Number of events 14 • Up to 3 years
|
|
General disorders
Fatigue
|
76.5%
13/17 • Number of events 21 • Up to 3 years
|
|
Blood and lymphatic system disorders
Lymphopenia
|
11.8%
2/17 • Number of events 4 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
29.4%
5/17 • Number of events 8 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
64.7%
11/17 • Number of events 13 • Up to 3 years
|
|
Investigations
Weight Loss
|
52.9%
9/17 • Number of events 16 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary/upper respiratory - Nose
|
52.9%
9/17 • Number of events 13 • Up to 3 years
|
|
Nervous system disorders
Dysgeusia
|
52.9%
9/17 • Number of events 11 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
52.9%
9/17 • Number of events 10 • Up to 3 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
41.2%
7/17 • Number of events 20 • Up to 3 years
|
|
Renal and urinary disorders
Creatinine
|
35.3%
6/17 • Number of events 15 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Nail changes
|
35.3%
6/17 • Number of events 11 • Up to 3 years
|
|
Metabolism and nutrition disorders
Dehydration
|
35.3%
6/17 • Number of events 10 • Up to 3 years
|
|
Investigations
Hypercholesteremia
|
35.3%
6/17 • Number of events 8 • Up to 3 years
|
|
Gastrointestinal disorders
Dry mouth
|
35.3%
6/17 • Number of events 8 • Up to 3 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
29.4%
5/17 • Number of events 11 • Up to 3 years
|
|
Gastrointestinal disorders
Flatulence
|
29.4%
5/17 • Number of events 7 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
29.4%
5/17 • Number of events 7 • Up to 3 years
|
|
Investigations
AST, SGOT(serum glutamic oxaloacetic transaminase)
|
23.5%
4/17 • Number of events 11 • Up to 3 years
|
|
Vascular disorders
Edema: limb
|
23.5%
4/17 • Number of events 11 • Up to 3 years
|
|
Gastrointestinal disorders
Vomiting
|
23.5%
4/17 • Number of events 10 • Up to 3 years
|
|
Gastrointestinal disorders
Nausea
|
23.5%
4/17 • Number of events 9 • Up to 3 years
|
|
General disorders
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)
|
23.5%
4/17 • Number of events 7 • Up to 3 years
|
|
Nervous system disorders
Pain - Head/headache
|
23.5%
4/17 • Number of events 4 • Up to 3 years
|
|
Investigations
Alkaline phosphatase
|
17.6%
3/17 • Number of events 7 • Up to 3 years
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
17.6%
3/17 • Number of events 7 • Up to 3 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
17.6%
3/17 • Number of events 5 • Up to 3 years
|
|
Gastrointestinal disorders
Pain - Abdomen NOS
|
17.6%
3/17 • Number of events 4 • Up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain - Back
|
17.6%
3/17 • Number of events 4 • Up to 3 years
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
17.6%
3/17 • Number of events 3 • Up to 3 years
|
|
Gastrointestinal disorders
Constipation
|
17.6%
3/17 • Number of events 3 • Up to 3 years
|
|
Gastrointestinal disorders
Dysphagia
|
17.6%
3/17 • Number of events 3 • Up to 3 years
|
|
Cardiac disorders
Dyspnea
|
17.6%
3/17 • Number of events 3 • Up to 3 years
|
|
Investigations
Hemoglobin
|
17.6%
3/17 • Number of events 3 • Up to 3 years
|
|
General disorders
Chills
|
17.6%
3/17 • Number of events 3 • Up to 3 years
|
|
Blood and lymphatic system disorders
Neutrophils/granulocytes
|
11.8%
2/17 • Number of events 12 • Up to 3 years
|
|
Reproductive system and breast disorders
Cough
|
11.8%
2/17 • Number of events 2 • Up to 3 years
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
5.9%
1/17 • Number of events 2 • Up to 3 years
|
|
Blood and lymphatic system disorders
Platelets
|
11.8%
2/17 • Number of events 4 • Up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain - Joint
|
5.9%
1/17 • Number of events 2 • Up to 3 years
|
|
Vascular disorders
Hypotension
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Nervous system disorders
Syncope
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Vascular disorders
Thrombosis/embolism
|
5.9%
1/17 • Number of events 1 • Up to 3 years
|
|
Gastrointestinal disorders
Distension/bloating, abdominal
|
5.9%
1/17 • Number of events 3 • Up to 3 years
|
Additional Information
Emily Bergsland, MD
University of California, San Francisco
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place