DRUG
Everolimus
Everolimus 7.5 mg administered daily for 28 days per cycle until disease progression or unacceptable toxicity.
Status
Completed
Phase
Phase 2
Enrollment
24
Locations
3
Results
Posted
Publications
0
Study summary
The purpose of this study is to estimate the time to disease progression when everolimus and pasireotide are given together in patients with advanced or metastatic HCC who have not had any prior systemic therapy.
This open label, single-arm Phase II study will assess time to progression (TTP) and safety of everolimus and pasireotide in patients with advanced or metastatic hepatocellular carcinoma (HCC) and limited prior systemic therapy. Should this regimen demonstrate efficacy, this will support a Phase III randomized clinical trial of this combination therapy. At least 30 patients will be enrolled into this Phase II study. Additionally, given the potential importance of the RAS/RAF/MEK/ERK and RAS/pAKT pathways, we propose to correlate outcomes with baseline pAKT, p-S6, somatostatin receptor tumor expression, and serum VEGF expression. We anticipate these exploratory analyses will increase understanding of the molecular pathways and their inhibition in this disease. The study will be performed as a University of North Carolina-coordinated, multicenter study.
Interventions
DRUG
Everolimus 7.5 mg administered daily for 28 days per cycle until disease progression or unacceptable toxicity.
DRUG
Monthly (every 28 days) intramuscular injection of long-acting pasireotide (pasireotide LAR 60 mg) repeated on day 1 of every 28 day cycle until disease progression or unacceptable toxicity.
Timeline
First posted
Dec 8, 2011
Study start
Dec 2011
Primary completion
Mar 2015
Study completion
Mar 2015
Results posted
Apr 7, 2016
Registry updated
Apr 7, 2016
Outcomes
Time to Progression (TTP)
Time frame · 3.5 years
Time to progression is defined as the time from study enrollment until radiological progression in a previously embolized lobe, development of new lesions in an untreated lobe, or evidence of extrahepatic progression (based on modified Hepatocellular Carcinoma (HCC) Response Evaluation Criteria In Solid Tumors (RECIST) criteria). Patients will be followed until death. Patients that die of causes unrelated to the study drug without evidence of progression will be censored.
Number of Individuals Experiencing Toxicity
Time frame · 3.5 years
Safety determinations are based on the rate of drug-related adverse events (AEs) reported based upon the toxicity as measured by the NCI Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Overall Survival (OS)
Time frame · 3.5 years
Overall survival is defined as the time from study enrollment until death.
Objective Response Rate (ORR)
Time frame · 3.5 years
ORR is the rate of complete responses (CRs) + partial responses (PRs) as determined by RECIST (v1.1) and modified HCC RECIST criteria. Responses defined as follows: CR: disappearance of all clinical/radiological evidence of tumor including any intratumoral arterial enhancement in all target lesions. Partial Response (PR): at least a 30% decrease in the sum of diameters of viable (contrast enhancement in the arterial phase) target lesions, referencing the baseline sum. Stable Disease (SD): any cases that do not qualify for either partial response or progressive disease. Progressive Disease (PD): an increase of at least 20% in the sum of the diameters of viable target lesions, referencing the nadir sum, and/or the appearance of one or more new lesions. A new hepatic nodule signals PD when the longest diameter is at least 10 mm and the nodule shows the typical vascular pattern of HCC on dynamic imaging or if at least 1-cm interval growth is seen in subsequent scans.
Eligibility
Inclusion Criteria: * Each subject must meet all of the following inclusion criteria to participate in this study: 1. Advanced or metastatic hepatocellular carcinoma (stage C per the BCLC criteria, see Appendix A). HCC may be diagnosed by tissue diagnosis or Alpha-fetoprotein (AFP) \>400 ng/mL with compatible mass on Magnetic Resonance Imaging Scan (MRI). Cat Scan (CT) abdomen with 3-phase contrast with arterial phase enhancement is acceptable, although MRI is preferred (imaging should be done within 4 weeks of study initiation). Recurrences of previously resected HCC will not require tissue confirmation if there is clear radiographic recurrence in the judgment of the investigator. Disease must not otherwise be amenable to local therapy. 2. Maximum Childs-Pugh score 6 (see Appendix A) with no active encephalopathy 3. Prior systemic therapy limited to sorafenib that was discontinued due to intolerance. Patients must undergo at least a 4-week washout prior to enrollment. 4. Eastern Cooperative Oncology Group (ECOG) PS of 0-2 5. Life expectancy of \>12 weeks 6. Age ≥18 years 7. Patients who have received previous local therapy, such as surgery, radiotherapy, hepatic arterial embolization, chemoembolization, radiofrequency ablation, percutaneous injection, or cryoablation, will be eligible for enrollment in the study provided that there is documented progression and disease is not amenable to further local therapies. Therapy must be completed \>4 weeks prior to study initiation (Day 1 of everolimus and pasireotide administration). 8. Minimum of 4 weeks since any major surgery 9. No active serious infection or other comorbid illness which would impair ability to participate in the trial. 10. International Normalized Ratio (INR) ≤1.5. (Anticoagulation is allowed if target INR ≤2.0 on a stable dose of warfarin or on a stable dose of low molecular weight heparin (LMWH) for \>2 weeks at time of enrollment). 11. Fasting serum cholesterol ≤300 mg/dL OR ≤7.75 mmol/L AND fasting triglycerides (TGs) ≤2.5 x upper limit of normal (ULN). NOTE: In case one or both of these thresholds are exceeded, the patient can only be included after initiation of appropriate lipid lowering medication. 12. Patients must have adequate organ function as evidenced by: * Absolute neutrophil count (ANC) ≥1.5 x 109/L * Platelet count ≥50 x 109/L * Hemoglobin (Hg) \>9 g/dL * Bilirubin ≤2 x ULN * Aspartate transaminase (AST) or Alanine transaminase (ALT) ≤5 x ULN * Serum creatinine ≤1.5 x ULN OR creatinine clearance ≥50 mL/min (estimated by Cockcroft Gault or measured) 13. Serum magnesium and serum potassium within institutional normal limits (patients may be on replacement) 14. Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test performed within 7 days prior to Day 1 of everolimus and pasireotide administration. 15. WOCBP and men must agree to use adequate contraception (barrier method of birth control) prior to study entry and for the duration of study participation. Men and women should use adequate birth control for at least 8 weeks after the last administration of study drugs. (Oral, implantable, or injectable contraceptives may be affected by cytochrome P450 interactions and are therefore not considered effective for this study.) 16. Signed, Institutional Review Board (IRB) approved written informed consent Exclusion Criteria: * Patients meeting any of the following exclusion criteria at baseline will be excluded from study participation: 1. Patients who have received prior treatment with an mTOR inhibitor (e.g., sirolimus, temsirolimus, everolimus) or somatostatin analog (e.g. octeotride) 2. Chronic treatment with systemic steroids (except for intermittent topical, local injection, or eye drops) or another immunosuppressive agent. NOTE: This restriction regarding systemic steroids does not apply should patient need course of glucocorticoid for treatment of non-infectious pneumonitis during study (see Section 4.5.2). 3. Patients with a known hypersensitivity to everolimus or other rapamycins (e.g., sirolimus, temsirolimus) or to its excipients 4. Patients with a known hypersensitivity to somatostatin or to its excipients 5. Concurrent or planned radiation, hormonal, chemotherapeutic, experimental, or targeted biologic therapy 6. Prior treatment with any investigational drug within the preceding 4 weeks 7. Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as: * Symptomatic congestive heart failure (New York Heart Association \[NYHA\] Class III or IV) * Unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction within 6 months of start of study drug, serious uncontrolled cardiac arrhythmia, or any other clinically significant cardiac disease * Severely impaired lung function as defined as spirometry and diffusing capacity for carbon monoxide (DLCO) that is 50% of the normal predicted value and/or 02 saturation that is 88% or less at rest on room air * Uncontrolled diabetes as defined by fasting serum glucose \>1.5 x ULN or Glycated hemoglobin (HbA1c) \>8.0% (Note: at the principle investigator's discretion, ineligible patients can be re-screened after adequate medical therapy has been instituted.) * Active (acute or chronic) or uncontrolled severe infections. NOTE: A detailed assessment of Hepatitis B/C medical history and risk factors must be done at screening for all patients. Hepatitis B viral deoxyribonucleic acid (HBV DNA) and Hepatitis C viral ribonucleic acid (HCV RNA) polymerase chain reaction (PCR) testing are required at screening for all patients with a positive medical history based on risk factors and/or confirmation of prior HBV/HCV infection. See Section 4.2 for further information. 8. Clinically significant third space fluid accumulation (i.e., ascites requiring paracentesis despite use of diuretics) or pleural effusion that either requires thoracentesis or is associated with shortness of breath 9. Risk factors for prolongation of Corrected QT Interval (QTc)\* including: * QTc at screening \>450 msec * History of syncope or family history of idiopathic sudden death * Sustained or clinically significant cardiac arrhythmias * Risk factors for Torsades de Pointes such as hypokalemia, hypomagnesemia, cardiac failure, clinically significant/symptomatic bradycardia, or high-grade AV block * Concomitant disease(s) that could prolong QT such as autonomic neuropathy (caused by diabetes or Parkinson's disease), human immunodeficiency virus (HIV), uncontrolled hypothyroidism, or cardiac failure * Concomitant medication(s) known to increase QT interval (See Appendix B) * University of North Carolina at Chapel Hill (UNC) uses GE electrocardiogram (ECG) carts which use the Bazett formula for QTc. 10. Patients should not receive immunization with attenuated live vaccines within 1 week of study entry or during study period. Close contact with those who have received attenuated live vaccines should be avoided during treatment with everolimus. Examples of live vaccines include intranasal influenza, measles, mumps, rubella, oral polio, Bacillus Calmette-Guérin (BCG), yellow fever, varicella, and TY21a typhoid vaccines. 11. Uncontrolled brain or leptomeningeal metastases, including patients who continue to require glucocorticoids for brain or leptomeningeal metastases 12. Symptomatic cholelithiasis 13. Other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin 14. A known history of HIV seropositivity (HIV testing is not mandatory) 15. Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of everolimus (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection.) 16. Patients with an active, bleeding diathesis or on oral anti-vitamin K medication (except warfarin as long as the goal INR is ≤1.5). Low-molecular-weight heparin (LMWH) is permitted (see Section 3.1.10.) 17. Unable or unwilling to discontinue use of prohibited fruit (or its juices) and/or prohibited medications listed in Appendix B for at least 14 days or five half-lives of a drug (whichever is longer) prior to the first dose of study drug and for the duration of the study 18. Male patient whose sexual partner(s) are WOCBP who are not willing to use adequate contraception during the study and for 8 weeks after the end of treatment 19. Active alcohol intake of 80 grams or more per day. For reference, one portion of alcohol (one glass of wine, one can or bottle of beer, or one ounce of hard liquor) contains approximately 15 grams of ethanol. 20. Inability to comply with study and/or follow-up procedures 21. History of noncompliance to medical regimens
Study locations
United States. Showing up to 24 locations stored in the fast local snapshot.
Moffitt Cancer Center
Tampa, Florida, United States
University of North Carolina at Chapel Hill Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, United States
Comprehensive Cancer Center of Wake Forest University
Winston-Salem, North Carolina, United States
Publications
No PMID-linked publications were present in this registry snapshot.
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