Trial Outcomes & Findings for A Study of LEE011 With Everolimus in Patients With Advanced Neuroendocrine Tumors (NCT NCT03070301)

NCT ID: NCT03070301

Last Updated: 2024-10-10

Results Overview

using RECIST v1.1

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 21 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2024-10-10

Participant Flow

Participant milestones

Measure	LEE011 and Everolimus Subjects will receive LEE011 200 mg daily, in combination with everolimus 5 mg daily; in the setting of toxicity, everolimus dosing will be changed to 2.5 mg daily or 2.5 mg every other day. Subjects will continue treatment until meeting one of the criteria for removal from study. LEE011: LEE011 200 mg daily everolimus: everolimus 5 mg daily or 2.5mg daily
Overall Study STARTED	21
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	20

Reasons for withdrawal

Measure	LEE011 and Everolimus Subjects will receive LEE011 200 mg daily, in combination with everolimus 5 mg daily; in the setting of toxicity, everolimus dosing will be changed to 2.5 mg daily or 2.5 mg every other day. Subjects will continue treatment until meeting one of the criteria for removal from study. LEE011: LEE011 200 mg daily everolimus: everolimus 5 mg daily or 2.5mg daily
Overall Study Progression of disease	16
Overall Study Physician Decision	3
Overall Study Withdrawal by Subject	1

Baseline Characteristics

A Study of LEE011 With Everolimus in Patients With Advanced Neuroendocrine Tumors

Baseline characteristics by cohort

Measure	LEE011 and Everolimus n=21 Participants Subjects will receive LEE011 200 mg daily, in combination with everolimus 5 mg daily; in the setting of toxicity, everolimus dosing will be changed to 2.5 mg daily or 2.5 mg every other day. Subjects will continue treatment until meeting one of the criteria for removal from study. LEE011: LEE011 200 mg daily everolimus: everolimus 5 mg daily or 2.5mg daily
Age, Continuous	56 years n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	21 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 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	18 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	21 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

using RECIST v1.1

Outcome measures

Measure	LEE011 and Everolimus n=21 Participants Subjects will receive LEE011 200 mg daily, in combination with everolimus 5 mg daily; in the setting of toxicity, everolimus dosing will be changed to 2.5 mg daily or 2.5 mg every other day. Subjects will continue treatment until meeting one of the criteria for removal from study. LEE011: LEE011 200 mg daily everolimus: everolimus 5 mg daily or 2.5mg daily
Progression Free Survival (PFS)	34 % of participants with response Interval 17.0 to 70.0

Adverse Events

LEE011 and Everolimus

Serious events: 8 serious events

Other events: 21 other events

Deaths: 13 deaths

Serious adverse events

Measure	LEE011 and Everolimus n=21 participants at risk Subjects will receive LEE011 200 mg daily, in combination with everolimus 5 mg daily; in the setting of toxicity, everolimus dosing will be changed to 2.5 mg daily or 2.5 mg every other day. Subjects will continue treatment until meeting one of the criteria for removal from study. LEE011: LEE011 200 mg daily everolimus: everolimus 5 mg daily or 2.5mg daily
General disorders Fever	14.3% 3/21 • 2 yeras
General disorders Flu like symptoms	4.8% 1/21 • 2 yeras
Infections and infestations Upper respiratory infection	9.5% 2/21 • 2 yeras
Respiratory, thoracic and mediastinal disorders Dyspnea	4.8% 1/21 • 2 yeras
Respiratory, thoracic and mediastinal disorders Pneumonitis	4.8% 1/21 • 2 yeras
Metabolism and nutrition disorders Hyperglycemia	4.8% 1/21 • 2 yeras
Injury, poisoning and procedural complications Fall	4.8% 1/21 • 2 yeras
Nervous system disorders Seizure	4.8% 1/21 • 2 yeras
Blood and lymphatic system disorders Febrile neutropenia	4.8% 1/21 • 2 yeras
Gastrointestinal disorders Abdominal pain	9.5% 2/21 • 2 yeras
Respiratory, thoracic and mediastinal disorders Cough	4.8% 1/21 • 2 yeras
General disorders Fatigue	4.8% 1/21 • 2 yeras
General disorders Death NOS	4.8% 1/21 • 2 yeras
Gastrointestinal disorders Small intestinal obstruction	4.8% 1/21 • 2 yeras
Gastrointestinal disorders Vomiting	4.8% 1/21 • 2 yeras
Blood and lymphatic system disorders Anemia	4.8% 1/21 • 2 yeras
Metabolism and nutrition disorders Hypoglycemia	9.5% 2/21 • 2 yeras
Gastrointestinal disorders Diarrhea	4.8% 1/21 • 2 yeras

Other adverse events

Measure	LEE011 and Everolimus n=21 participants at risk Subjects will receive LEE011 200 mg daily, in combination with everolimus 5 mg daily; in the setting of toxicity, everolimus dosing will be changed to 2.5 mg daily or 2.5 mg every other day. Subjects will continue treatment until meeting one of the criteria for removal from study. LEE011: LEE011 200 mg daily everolimus: everolimus 5 mg daily or 2.5mg daily
Blood and lymphatic system disorders Anemia	71.4% 15/21 • 2 yeras
Metabolism and nutrition disorders Anorexia	9.5% 2/21 • 2 yeras
Musculoskeletal and connective tissue disorders Arthralgia	9.5% 2/21 • 2 yeras
Gastrointestinal disorders Diarrhea	23.8% 5/21 • 2 yeras
Skin and subcutaneous tissue disorders Dry Skin	9.5% 2/21 • 2 yeras
General disorders Edema Limbs	19.0% 4/21 • 2 yeras
Respiratory, thoracic and mediastinal disorders Epistaxis	19.0% 4/21 • 2 yeras
General disorders Fatigue	66.7% 14/21 • 2 yeras
Blood and lymphatic system disorders Febrile Neutropenia	4.8% 1/21 • 2 yeras
General disorders Fever	23.8% 5/21 • 2 yeras
Gastrointestinal disorders Flatulence	9.5% 2/21 • 2 yeras
Nervous system disorders Headache	14.3% 3/21 • 2 yeras
Metabolism and nutrition disorders Hyperglycemia	95.2% 20/21 • 2 yeras
Metabolism and nutrition disorders Hypertriglyceridemia	9.5% 2/21 • 2 yeras
Metabolism and nutrition disorders Hypocalcemia	14.3% 3/21 • 2 yeras
Metabolism and nutrition disorders Hypokalemia	19.0% 4/21 • 2 yeras
Metabolism and nutrition disorders Hypomagnesemia	14.3% 3/21 • 2 yeras
Metabolism and nutrition disorders Hypophosphatemia	9.5% 2/21 • 2 yeras
Investigations Increased AST	14.3% 3/21 • 2 yeras
Investigations Increased cholesterol	14.3% 3/21 • 2 yeras
Investigations Increased creatinine	33.3% 7/21 • 2 yeras
Psychiatric disorders Insomnia	14.3% 3/21 • 2 yeras
Blood and lymphatic system disorders Leukopenia	76.2% 16/21 • 2 yeras
Investigations Lymphocytopenia	47.6% 10/21 • 2 yeras
Gastrointestinal disorders Mucositis oral	38.1% 8/21 • 2 yeras
Gastrointestinal disorders Nausea	9.5% 2/21 • 2 yeras
Respiratory, thoracic and mediastinal disorders Nasal congestion	9.5% 2/21 • 2 yeras
Blood and lymphatic system disorders Neutropenia	66.7% 14/21 • 2 yeras
General disorders Peripheral edema	19.0% 4/21 • 2 yeras
Skin and subcutaneous tissue disorders Pruritus	9.5% 2/21 • 2 yeras
Skin and subcutaneous tissue disorders Rash maculo-papular	33.3% 7/21 • 2 yeras
Blood and lymphatic system disorders Thrombocytopenia	76.2% 16/21 • 2 yeras
Infections and infestations Urinary tract infection	4.8% 1/21 • 2 yeras

Additional Information

Dr. Diane Reidy-Lagunes, MD

Memorial Sloan Kettering Cancer Center

Phone: 646-888-4185

Email: reidyd@mskcc.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place