Trial Outcomes & Findings for Ribociclib in Treating Patients With Advanced Neuroendocrine Tumors of Foregut Origin (NCT NCT02420691)
NCT ID: NCT02420691
Last Updated: 2020-10-30
Results Overview
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) 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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
3 years 10 months
Results posted on
2020-10-30
Participant Flow
September 2015-August 2016. All patients enrolled at MD Anderson.
21 participants consented, 1 participant was not eligible due to screen failure.
Participant milestones
| Measure |
Study Participants
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
14
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Study Participants
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Withdrawal by Subject
|
3
|
Baseline Characteristics
Ribociclib in Treating Patients With Advanced Neuroendocrine Tumors of Foregut Origin
Baseline characteristics by cohort
| Measure |
Study Participants
n=20 Participants
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=5 Participants
|
|
Age, Continuous
|
57 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3 years 10 monthsPer Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) 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.
Outcome measures
| Measure |
Study Participants
n=19 Participants
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Number of Participants With Response Rate Per Response Evaluation Criteria in Solid Tumors Version 1.1
|
19 participants
|
SECONDARY outcome
Timeframe: At 6 monthsNumber of Participants that did not have progressive disease at 6 months.
Outcome measures
| Measure |
Study Participants
n=19 Participants
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Number of Participants With Clinical Benefit Rate
|
19 participants
|
SECONDARY outcome
Timeframe: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, up to 24 monthsPFS is the length of time during and after the treatment that a participant lives with the disease but it does not get worse.The Kaplan-Meier (KM) method will be used to estimate the PFS.
Outcome measures
| Measure |
Study Participants
n=20 Participants
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Progression Free Survival (PFS)
|
10.44 months
Interval 7.37 to 13.49
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline (Pre-Treatment) and Cycle 2 Day 1, each Cycle is 28 daysDecrease in pRB as Measured by Immunohistochemistry (IHC) in Biopsies From Baseline and From cycle 2 day 1. H-scores were calculated as the sum of the products of the percentage of positive staining areas and the staining intensity (0, 1, 2 or 3), and ranged from 0 to 300. A score of 0 represents the absence of expression, and an H-score of 300 represents maximum expression.
Outcome measures
| Measure |
Study Participants
n=19 Participants
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Change in pRB With Treatment
H- Score Pre-Treatment
|
236.47 score on a scale
Standard Deviation 67.25
|
|
Change in pRB With Treatment
H-Score Post-Treatment
|
238.32 score on a scale
Standard Deviation 39.56
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline (Pre-Treatment) and Cycle 2 Day 1, each Cycle is 28 daysKi-67 was calculated as a percentage cells staining positive by Immunohistochemistry (IHC).
Outcome measures
| Measure |
Study Participants
n=19 Participants
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Change in Ki-67 With Treatment
Pre-Treatment
|
13.61 percentage of cells
Standard Deviation 10.18
|
|
Change in Ki-67 With Treatment
Post-Treatment
|
19.11 percentage of cells
Standard Deviation 17.83
|
Adverse Events
Study Participants
Serious events: 7 serious events
Other events: 17 other events
Deaths: 11 deaths
Serious adverse events
| Measure |
Study Participants
n=20 participants at risk
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
5.0%
1/20 • 3 years and 5 months
|
|
Gastrointestinal disorders
Upper Gastrointestinal Hemorrhage
|
10.0%
2/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
5.0%
1/20 • 3 years and 5 months
|
|
Vascular disorders
Thromboembolic Event
|
5.0%
1/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
5.0%
1/20 • 3 years and 5 months
|
|
Renal and urinary disorders
Acute Kidney Injury
|
10.0%
2/20 • 3 years and 5 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothroax
|
5.0%
1/20 • 3 years and 5 months
|
|
General disorders
Non-cardiac Chest Pain
|
5.0%
1/20 • 3 years and 5 months
|
|
Nervous system disorders
Encephalopathy
|
5.0%
1/20 • 3 years and 5 months
|
Other adverse events
| Measure |
Study Participants
n=20 participants at risk
LEE011 will be taken orally, once a day for 21 consecutive days followed by a 7 day planned break. LEE011 will be dosed on a flat dosing scale of 600 mg/day, irrespective of size and weight.
|
|---|---|
|
Investigations
Neutrophil Count Increased
|
85.0%
17/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Nausea
|
60.0%
12/20 • 3 years and 5 months
|
|
Blood and lymphatic system disorders
Anemia
|
60.0%
12/20 • 3 years and 5 months
|
|
General disorders
Fatigue
|
55.0%
11/20 • 3 years and 5 months
|
|
Investigations
Platelet Count Decreased
|
45.0%
9/20 • 3 years and 5 months
|
|
Investigations
Creatinine Increased
|
40.0%
8/20 • 3 years and 5 months
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
5/20 • 3 years and 5 months
|
|
Investigations
Alanine Aminotransferase Increased
|
20.0%
4/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Anorexia
|
20.0%
4/20 • 3 years and 5 months
|
|
Gastrointestinal disorders
Diarrhea
|
20.0%
4/20 • 3 years and 5 months
|
|
Investigations
Aspartate Aminotransferase Increased
|
15.0%
3/20 • 3 years and 5 months
|
|
Investigations
Weight Loss
|
15.0%
3/20 • 3 years and 5 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
15.0%
3/20 • 3 years and 5 months
|
|
Skin and subcutaneous tissue disorders
Rash Maculo-papular
|
15.0%
3/20 • 3 years and 5 months
|
|
General disorders
Edema Limbs
|
15.0%
3/20 • 3 years and 5 months
|
|
Nervous system disorders
Dysguesia
|
15.0%
3/20 • 3 years and 5 months
|
|
General disorders
Fever
|
10.0%
2/20 • 3 years and 5 months
|
|
Investigations
Alkaline Phosphatase Increased
|
10.0%
2/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
10.0%
2/20 • 3 years and 5 months
|
|
Investigations
Blood Bilirubin Increased
|
10.0%
2/20 • 3 years and 5 months
|
|
Nervous system disorders
Headache
|
10.0%
2/20 • 3 years and 5 months
|
|
Nervous system disorders
Peripheral Sensory Neuropathy
|
10.0%
2/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
10.0%
2/20 • 3 years and 5 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
10.0%
2/20 • 3 years and 5 months
|
|
Gastrointestinal disorders
Constipation
|
10.0%
2/20 • 3 years and 5 months
|
|
Infections and infestations
Urinary Tract Infection
|
5.0%
1/20 • 3 years and 5 months
|
|
Investigations
Investigations: Other- Hyperphosphatemia
|
5.0%
1/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Hyperphosphatemia
|
5.0%
1/20 • 3 years and 5 months
|
|
Renal and urinary disorders
Acute Kidney Injury
|
5.0%
1/20 • 3 years and 5 months
|
|
Infections and infestations
Upper Respiratory Infection
|
5.0%
1/20 • 3 years and 5 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
5.0%
1/20 • 3 years and 5 months
|
|
Infections and infestations
Skin infection
|
5.0%
1/20 • 3 years and 5 months
|
|
General disorders
Malaise
|
5.0%
1/20 • 3 years and 5 months
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
5.0%
1/20 • 3 years and 5 months
|
|
Gastrointestinal disorders
Gastroparesis
|
5.0%
1/20 • 3 years and 5 months
|
|
General disorders
Edema Face
|
5.0%
1/20 • 3 years and 5 months
|
|
Vascular disorders
Hematoma
|
5.0%
1/20 • 3 years and 5 months
|
|
Gastrointestinal disorders
Abdominal Distention
|
5.0%
1/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
5.0%
1/20 • 3 years and 5 months
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
5.0%
1/20 • 3 years and 5 months
|
|
Gastrointestinal disorders
Gastroesophageal Reflux Disease
|
5.0%
1/20 • 3 years and 5 months
|
|
Gastrointestinal disorders
Mucositis Oral
|
5.0%
1/20 • 3 years and 5 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.0%
1/20 • 3 years and 5 months
|
|
Cardiac disorders
Palpitations
|
5.0%
1/20 • 3 years and 5 months
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
5.0%
1/20 • 3 years and 5 months
|
|
Metabolism and nutrition disorders
Dehydration
|
5.0%
1/20 • 3 years and 5 months
|
|
Eye disorders
Eye Disorders: Other- Periorbital Swelling and Crystal Discharge
|
5.0%
1/20 • 3 years and 5 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place