Neuroendocrine TumorsPhase 2NCT03070301
A Study of LEE011 With Everolimus in Patients With Advanced Neuroendocrine Tumors
A Phase 2 study testing LEE011 and everolimus in people with Neuroendocrine Tumors, run by Memorial Sloan Kettering Cancer Center at 5 sites in United States.
Trial summary
The purpose of this study is to test any good and bad effects of the combination of LEE011 with everolimus on the participant and the cancer. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuroendocrine Tumors
- What is being tested?
- LEE011 and everolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 5 sitesUnited States
- Study dates
- 27 Feb 2017to 31 Jan 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient has signed the Informed Consent prior to any screening procedures being performed and is able to comply with the protocol requirements.
- Adults ≥ 18 years old
- Histologic or cytologic diagnosis of a WDNET, Ki67 ≤ 30%, unresectable, of foregut origin (thymic, bronchopulmonary, gastric, duodenal, and pancreatic) confirmed by the enrolling institution
- Note: If patients have a functional NET, they are permitted to continue on a somatostatin analog for hormonal symptom control
- MSK patient has tissue available from a previous biopsy for the evaluation of potential predictive biomarkers. If tissue is not available for MSK patient, a new tumor specimen will need to be obtained prior to the start of study treatment If archived tissue is available, participating site patient will provide for the evaluation of potential predictive biomarkers. If tissue is not available for participating site patient, a new tumor specimen is optional prior to the start of study treatment.
- Documented radiological evidence for disease progression (measurable or nonmeasurable) ≤12 months prior to enrollment
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- Disease that is currently not amenable to surgical resection with curative intent as determined by the treating investigator
- Measurable disease as defined by RECIST v1.1
- ECOG performance status 0 or 1 or KPS performance status 100 to 70
- Patient has adequate bone marrow and organ function as defined by the following laboratory values at screening:
- Absolute neutrophil count ≥1.5 x 10^9/L
- Platelets ≥ 100 x 10^9/L
- Hemoglobin ≥ 9.0 g/dL
- INR ≤ 1.5
- Serum creatinine <1.5mg/dL or creatinine clearance ≥ 50 mL/min
- In the absence of liver metastases, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <2.5 x ULN. If a patient has liver metastases, ALT and AST <5 x ULN
- Total bilirubin < ULN; or total bilirubin ≤3.0 x ULN or direct bilirubin ≤1.5 x ULN in patients with well-documented Gilbert"s Syndrome
- Negative serum pregnancy test done ≤14 days prior to registration, for women of childbearing potential only A serum pregnancy test will be conducted ≤ 72 hours prior to treatment start as a pre-treatment parameter. All women of reproductive potential and their partners must agree to use adequate methods of birth control (e.g. latex condoms) throughout the study and for 30 days after the last dose of study drug.
- † A female of reproductive potential is a sexually mature female who: has not undergone a hysterectomy or bilateral oophorectomy; or has not been naturally postmenopausal for at least 24 consecutive months (i.e. has had menses at any time in the preceding 24 consecutive months).
- Patient with standard 12-lead ECG with the following parameters at screening (defined as the mean of the triplicate ECGs)
- °QTcF interval at screening <450msec (using Fridericia"s correction)
- Must be able to swallow LEE011 and everolimus capsules/tablets
- Recovered from adverse events (to grade 1 or less toxicity according to CTCAE 4.0) due to agents administered previously *NOTE: Chemotherapy-induced alopecia and grade 2 neuropathy are acceptable
Who cannot join
- Patient has a known hypersensitivity to any of the excipients of LEE011 or everolimus
- Previous treatment with a CDK 4/6 inhibitor or an mTOR inhibitor
- Has had prior chemotherapy, targeted small molecule therapy within 2 weeks prior to study Day 1 or who has not recovered (i.e. ≤ Grade 1 or at baseline) from adverse events due to a previously administered agent
- Note: Subjects with < Grade 2 neuropathy or chemotherapy-induced alopecia are an exception to this criterion and may qualify for the study
- Patient has a concurrent malignancy or malignancy within 3 years prior to starting study drug, with the exception of adequately treated, basal or squamous cell carcinoma, non-melanomatous skin cancer or curatively resected cervical cancer
- Patients with central nervous system (CNS) involvement unless they meet ALL of the following criteria:
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- At least 4 weeks from prior therapy completion (including radiation and/or surgery) to starting the study treatment
- Clinically stable CNS tumor at the time of screening and not receiving steroids and/or enzyme-inducing anti-epileptic medications for brain metastases
- Patient has impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of the study drugs (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection)
- Patient has a known history of HIV infection (testing not mandatory)
- Patient has any other concurrent severe and/or uncontrolled medical condition that would, in the investigator"s judgment, cause unacceptable safety risks, contraindicate patient participation in the clinical study or compromise compliance with the protocol (e.g. chronic pancreatitis, active untreated or uncontrolled fungal, bacterial or viral infections, etc.).
- Uncontrolled diabetes mellitus as defined by HbA1c >8% despite adequate therapy. Patients with a known history of impaired fasting glucose or diabetes mellitus (DM) may be included, however blood glucose and antidiabetic treatment must be monitored closely throughout the trial and adjusted as necessary
- Clinically significant, uncontrolled heart disease and/or cardiac repolarization abnormality, including any of the following:
- History of myocardial infarction (MI), angina pectoris, symptomatic pericarditis, or coronary artery bypass graft (CABG) within 6 months prior to study entry
- Documented cardiomyopathy
- Left Ventricular Ejection Fraction (LVEF) < 50% as determined by Multiple Gated acquisition (MUGA) scan or echocardiogram (ECHO)
- Long QT syndrome or family history of idiopathic sudden death or congenital long QT syndrome, or any of the following:
- Risk factors for Torsades de Pointe (TdP) including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure, or history of clinically significant/symptomatic bradycardia
- Concomitant medication(s) with a known risk to prolong the QT interval and/or known to cause Torsades de Pointe that cannot be discontinued or replaced by safe alternative medication (e.g. within 5 half-lives or 7 days prior to starting study drug)
- Inability to determine the QTcF interval
- Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade AV block (e.g., bifascicular block, Mobitz type II and third degree AV block)
- Patient is currently receiving any of the following medications and cannot be discontinued 7 days prior to starting study drug
- Known strong inducers or inhibitors of CYP3A4/5, including grapefruit, grapefruit hybrids, pummelos, star-fruit, and Seville oranges, that have a narrow therapeutic window and are predominantly metabolized through CYP3A4/5
- Herbal preparations/medications, dietary supplements
- Patient is currently receiving or has received systemic corticosteroids ≤2 weeks prior to starting study drug, or who have not fully recovered from side effects of such treatment
- °The following uses of corticosteroids are permitted: single doses, topical applications (e.g., for rash), inhaled sprays (e.g., for obstructive airways diseases), eye drops or local injections (e.g., intra-articular)
- Participation in a prior investigational study within 30 days prior to enrollment or within 5 half-lives of the investigational product, whichever is longer
- Patient who has received radiotherapy ≤4 weeks or limited field radiation for palliation ≤2 weeks prior to starting study drug, and who has not recovered to grade 1 or better from related side effects of such therapy (exceptions include alopecia) or in whom ≥25% of the bone marrow (Ellis, 1961) was irradiated
- Patient has had major surgery within 14 days prior to starting study drug or has not recovered from major side effects (tumor biopsy is not considered as major surgery)
- Patient with a Child-Pugh score B or C
- Patient has a history of non-compliance to medical regimen or inability to grant consent
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception until the termination of gestation, confirmed by a positive hCG laboratory test.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Md Anderson Cancer Center
Houston, Texas, 77030
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: A Phase II Trial of LEE011 in Combination With Everolimus in the Treatment of Advanced Well Differentiated Neuroendocrine Tumors of Foregut Origin
The purpose of this study is to test any good and bad effects of the combination of LEE011 with everolimus on the participant and the cancer.
- Conditions
- Neuroendocrine Tumors
- Being tested
- LEE011, everolimus
- Keywords
- LEE011, Everolimus, 16-1535
- Medical subject headings
- Neuroendocrine Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LEE011Drug
LEE011 200 mg daily
everolimusDrug
everolimus 5 mg daily or 2.5mg daily
| Study group | Role | What they receive |
|---|---|---|
| LEE011 and everolimus | Experimental | 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. Receives: Drug: LEE011; Drug: everolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression Free Survival (PFS)
using RECIST v1.1
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2017
- Study start27 Feb 2017
- Primary completion31 Jan 2024
- Study completion31 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Neuroendocrine TumorsStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This will be a multicenter, non-randomized, open-label phase II clinical trial.
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis, Dana-Farber Cancer Institute, M.D. Anderson Cancer Center
- Responsible party
- Sponsor
- Investigators
- Diane Reidy-Lagunes, MD, MS, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03070301
- Sponsor's protocol ID
- 16-1535
- Status last verified
- Oct 2024
- First submitted
- 28 Feb 2017
- Last updated
- 10 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Raj N, Zheng Y, Hauser H, Chou J, Rafailov J, Bou-Ayache J, Sawan P, Chaft J, Chan J, Perez K, Rudin C, Tang L, Reidy-Lagunes D. Ribociclib and everolimus in well-differentiated foregut neuroendocrine tumors. Endocr Relat Cancer. 2021 Apr;28(4):237-246. doi: 10.1530/ERC-20-0446.
PubMed 33640871
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of LEE011 in Combination With Everolimus in the Treatment of Advanced Well Differentiated Neuroendocrine Tumors of Foregut Origin
- ClinicalTrials.gov ID
- NCT03070301
- Acronym / sponsor ID
- 16-1535
- Conditions
- Neuroendocrine Tumors
- Keywords
- LEE011, Everolimus, 16-1535
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Novartis, Dana-Farber Cancer Institute, M.D. Anderson Cancer Center
- Study start
- 27 Feb 2017 (actual)
- Primary completion
- 31 Jan 2024 (actual)
- Study completion
- 31 Jan 2024 (actual)
- First posted
- 3 Mar 2017 (actual)
- Results
- Posted 10 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to test any good and bad effects of the combination of LEE011 with everolimus on the participant and the cancer.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This will be a multicenter, non-randomized, open-label phase II clinical trial.
| Arm | Type | Receives |
|---|---|---|
| LEE011 and everolimus | Experimental | Drug: LEE011, Drug: everolimusSubjects 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Patient has signed the Informed Consent prior to any screening procedures being performed and is able to comply with the protocol requirements.
- Adults ≥ 18 years old
- Histologic or cytologic diagnosis of a WDNET, Ki67 ≤ 30%, unresectable, of foregut origin (thymic, bronchopulmonary, gastric, duodenal, and pancreatic) confirmed by the enrolling institution
- Note: If patients have a functional NET, they are permitted to continue on a somatostatin analog for hormonal symptom control
- MSK patient has tissue available from a previous biopsy for the evaluation of potential predictive biomarkers. If tissue is not available for MSK patient, a new tumor specimen will need to be obtained prior to the start of study treatment If archived tissue is available, participating site patient will provide for the evaluation of potential predictive biomarkers. If tissue is not available for participating site patient, a new tumor specimen is optional prior to the start of study treatment.
Key exclusion criteria (32 listed)
- Patient has a known hypersensitivity to any of the excipients of LEE011 or everolimus
- Previous treatment with a CDK 4/6 inhibitor or an mTOR inhibitor
- Has had prior chemotherapy, targeted small molecule therapy within 2 weeks prior to study Day 1 or who has not recovered (i.e. ≤ Grade 1 or at baseline) from adverse events due to a previously administered agent
- Note: Subjects with < Grade 2 neuropathy or chemotherapy-induced alopecia are an exception to this criterion and may qualify for the study
- Patient has a concurrent malignancy or malignancy within 3 years prior to starting study drug, with the exception of adequately treated, basal or squamous cell carcinoma, non-melanomatous skin cancer or curatively resected cervical cancer
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LEE011LEE011 200 mg daily | Drug | LEE011 and everolimus |
| everolimuseverolimus 5 mg daily or 2.5mg daily | Drug | LEE011 and everolimus |
Primary outcome
Progression Free Survival (PFS)
Time frame: 12 months
using RECIST v1.1
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33640871Raj N, Zheng Y, Hauser H, Chou J, Rafailov J, Bou-Ayache J, Sawan P, Chaft J, Chan J, Perez K, Rudin C, Tang L, Reidy-Lagunes D. Ribociclib and everolimus in well-differentiated foregut neuroendocrine tumors. Endocr Relat Cancer. 2021 Apr;28(4):237-246. doi: 10.1530/ERC-20-0446.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Diane Reidy-Lagunes, MD, MS principal investigator
Memorial Sloan Kettering Cancer Center
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LEE011 and everolimus in people with Neuroendocrine Tumors, run by Memorial Sloan Kettering Cancer Center at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 23 inclusion and 32 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression Free Survival (PFS) (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03070301, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Oct 2024.