Fibrolamellar CarcinomaPhase 2NCT01642186
Study of (1) Everolimus, (2) Estrogen Deprivation Therapy (EDT) With Leuprolide + Letrozole and (3) Everolimus + EDT in Patients With Unresectable Fibrolamellar Hepatocellular Carcinoma (FLL-HCC)
A Phase 2 study testing everolimus, letrozole plus leuprolide and combination of everolimus, letrozole and leuprolide in people with Fibrolamellar Carcinoma and Fibrolamellar Liver Cancer, run by Memorial Sloan Kettering Cancer Center at 6 sites in United States.
Trial summary
There is no effective standard treatment for fibrolamellar liver cancer that cannot be removed by surgery. The investigators want to find out what effects, good and/or bad, 3 drugs called letrozole, leuprolide and everolimus will have on cancer. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Fibrolamellar Carcinoma, Fibrolamellar Liver Cancer
- What is being tested?
- everolimus, letrozole plus leuprolide and combination of everolimus, letrozole and leuprolide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 6 sitesUnited States
- Study dates
- 12 Jul 2012to 16 Jul 2021
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients ≥ 12 years old.
- Pathologically confirmed diagnosis of advanced and/or unresectable FLL-HCC. This will be performed by the participating centers on submitted specimens. If the submitted material is insufficient for analysis, a repeat biopsy is recommended.
- ECOG performance status 0-2 ; Lansky performance score of ≥ 60% for patients 12-16 years old
- Adequate hematologic, renal and hepatic function defined as:
- Hematologic: ANC ≥ 1.0 x 10^9/L, platelets ≥ 50 x 10^9/L o Renal: creatinine ≤ 2 x upper limit of normal, or creatinine Clearance of ≥60 cc/mL/1.73 m^2 for patients > 16 years old. For patients ≤ 16 years of age, creatinine Clearance of ≥70 cc/mL/1.73 m^2 or serum creatinine based on the following chart: Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m^2 or serum creatinine based on age/gender as follows: Age Maximum Serum Creatinine (mg/dL) Male Female 10 to < 13 years 1.2 1.2 13 to < 16 years 1.5 1.4
- ≥ 16 years 1.7 1.4 The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC.
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- Hepatic: total bilirubin ≤ 2 mg/dL, alanine and aminotransferase levels ≤ 5 x upper limit of normal for age.
- Fasting blood glucose <1.5 x upper limit of normal . If fasting glucose > 1.5 x upper limit of normal, adequate glycemic control (fasting glucose < 1.5 x upper limit of normal ) for three weeks is recommended before starting protocol therapy.
- At least 1 target lesion measurable by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) guidelines.
- Target lesion(s) must not lie within a previously resected, irradiated, ablated, or chemoembolized area. If a lesion does lie in such an area, there must be evidence of a ≥ 20% increase in diameter and/or the appearance of a new lesion on subsequent imaging in order for such a lesion to be considered a target lesion.
- Prior systemic therapy is allowed. Prior surgery, locoregional ablative or embolic therapies are also permitted provided that the criteria for measurable disease as outlined above are met.
- Concurrent antiviral therapy for hepatitis B is permitted
- Women of childbearing potential must be practicing an effective method of birth control that may include intrauterine devices (both hormonal and non-hormonal are acceptable), double-barrier method, male partner sterilization or abstinence, before enrollment, and throughout the study and for 6 months after receiving the last dose of study drug.
- Men must agree to use a double barrier method of birth control and to not donate sperm during the study and for 6 months after receiving the last dose of study drugs. Sperm banking is acceptable for interested male patients enrolled on study prior to initiating treatment. Prescription oral contraceptives, contraceptive injections, and contraceptive patch are not approved methods of contraception in this study.
- Negative pregnancy test (serum hCG) result (applicable to women of child bearing potential) within 7 days before Cycle 1 Day 1 of study treatment.
Who cannot join
- Concurrent anticancer, or radiation therapy. Patients must have completed all anticancer therapy > 4 weeks before the start of study therapy. The date of last palliative radiation must be > 2 weeks from the start of study therapy. Palliative radiation is permitted on protocol with MSK PI discretion on treatment modifications.
- Patients, who have had a major surgery or significant traumatic injury within 4 weeks of start of study drug, patients who have not recovered from the side effects of any major surgery (defined as requiring general anesthesia) .
- Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Topical or inhaled corticosteroids are allowed.
- Concurrent oral contraceptive use or hormonal replacement therapy.
- Use of an aromatase inhibitor, GnRH agonist and/or tamoxifen within the past 30 days. Patients previously on fulvestrant or a q3 month GnRH agonist must have discontinued these medications for at least 3 months.
- Concurrent use of potent CYP3A4 and/or P-glycoprotein inhibitors or potent CYP3A4 inducers (please see Appendices 3 and 4). Where possible, otherwise eligible patients should be switched to alternative agents; otherwise, they will be excluded from the study.
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- Potent CYP3A4 inducers decrease serum everolimus levels and should not be given concomitantly. Dose modifications of everolimus are not indicated in the presence of moderate CYP3A4 inducers [108]. Please refer to Appendix 3 for a complete list of potent and moderate inducers of CYP3A4.
- Potent CYP3A4 and/or P-glycoprotein inhibitors can increase serum levels of everolimus and should not be co-administered. Moderate inhibitors may mildly-moderately increase serum everolimus levels, though there is no definitive evidence supporting a dose reduction [108]. Please refer to Appendix 4 for a complete list of potent and moderate inhibitors of CYP3A4.
- Any investigational drug received within one month of study enrollment.
- Any severe, uncontrolled medical conditions that, in the opinion of the investigator, may be exacerbated by study therapy including infection, diabetes and cardiopulmonary disease.
- Any psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant or nursing women.
- Patients with a known hypersensitivity to everolimus, letrozole, leuprolide and/or related compounds or their excipients.
- Patients who received any form of transplant and who are on any form of immunosuppressive therapy. However transplanted patients who are off immunosuppressive therapy for at least 4 weeks are allowed on the study, provided that any of their immunosuppressive-related toxicities have recovered to at least a grade 1.
- Known HIV positive with a CD4 count < 500 cells/mm3.
- Immunization with a live vaccine < 1 week of initiating study therapy or during therapy.
- BSA <1 m^2
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
John Hopkins Medical Center
Baltimore, Maryland, 21287
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
University of California San Francisco
San Francisco, California, 94143
About this study
Official title: A Randomized Three Arm Phase II Study of (1) Everolimus, (2) Estrogen Deprivation Therapy (EDT) With Leuprolide + Letrozole and (3) Everolimus + EDT in Patients With Unresectable Fibrolamellar Hepatocellular Carcinoma (FLL-HCC)
There is no effective standard treatment for fibrolamellar liver cancer that cannot be removed by surgery. The investigators want to find out what effects, good and/or bad, 3 drugs called letrozole, leuprolide and everolimus will have on cancer. All of these drugs are FDA approved for the treatment of different cancers. Letrozole and leuprolide stop the body from producing estrogen, a normal hormone produced by the body. Too much estrogen may help fibrolamellar liver cancer grow. Everolimus is a drug that may block other chemicals in the body that can help cancer grow. The combination of letrozole and leuprolide plus everolimus may work well together.
- Being tested
- everolimus, letrozole plus leuprolide, combination of everolimus, letrozole and leuprolide
- Keywords
- LETROZOLE, LUPRON DEPOT, RAD001 (EVEROLIMUS), 11-211
- Medical subject headings
- Fibrolamellar hepatocellular carcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
everolimusDrug
letrozole plus leuprolideDrug
combination of everolimus, letrozole and leuprolideDrug
| Study group | Role | What they receive |
|---|---|---|
| Arm A everolimus | Experimental | Everolimus will be administered at the following doses:
* Patients with a BSA ≤ 1.5 m2 will receive everolimus 5 mg PO QD.
* Patients with a BSA > than or = to 1.5 m2 will receive everolimus 7.5 mg PO QD. If the patient is randomized to everolimus alone (1) or leuprolide and letrozole (2) and the cancer continues to grow, they may have the option to receive all three drugs together. Receives: Drug: everolimus |
| Arm B letrozole plus leuprolide | Experimental | Day 1 of Cycle 1: Leuprolide 7.5 mg IM will be administered by a nurse in clinic.
Letrozole will be dispensed and will be taken at home. Patients will be instructed to take letrozole at the same time each day, consistently with food or without food, and swallowed whole with a glass of water. If the patient is randomized to everolimus alone (1) or leuprolide and letrozole (2) and the cancer continues to grow, they may have the option to receive all three drugs together. Receives: Drug: letrozole plus leuprolide |
| Arm C combination everolimus, letrozole and leuprolide | Experimental | * Patients with a BSA ≤ 1.5 m2 will receive everolimus 5 mg PO QD.
* Patients with a BSA > than or = to 1.5 m2 will receive everolimus 7.5 mg PO QD. Leuprolide 7.5 mg IM will be given every 4 weeks (+/- 7 days). Everolimus and letrozole will be administered continuously using the same dose, schedule and administration. Everolimus, letrozole and leuprolide should be administered concurrently at all times. Receives: Drug: combination of everolimus, letrozole and leuprolide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy Endpoints for Part 1 of the Study is Progression-free Survival at 6 Months (PFS6)
for Part 1 of the study is progression-free survival at 6 months (PFS6). A progression event refers to the first evidence of radiographic disease progression, clinical progression as determined by study investigators, or death. Imaging performed in 6 months will be used to determine PFS6.
Measured: 6 months
Secondary outcomes (5)
Median PFS
Kaplan-Meier PFS will be measured from the date that study therapy is initiated until the date of first evidence of radiographic disease progression, global clinical deterioration as determined by study investigators, or death.
Measured: 2 years
Median Overall Survival (OS)
Kaplan-Meier OS will be measured from the date that study therapy was commenced until the date of death.
Measured: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months
Percentage of Participants With Stable Disease
Objective responses will be reported using RECIST guidelines (version 1.1). Objective response will be estimated using binomial proportions and exact 95% CIs will be provided. Stable Disease is defined as neither sufficient shrinkage (compared to baseline) to qualify for Partial Reponse nor sufficient increase (taking as reference the smallest sum diameters while on study) to qualify for Progressive Disease.
Measured: 2 years
Number of Participants With One or More Adverse Events/Toxicity
Adverse events/toxicity will be monitored and recorded using the CTCAE version 4.0 and summarized descriptively.
Measured: 2 years
Number of Participants With Tissue Biomarkers Collected
Associations between baseline tissue biomarkers and PFS6 will be assessed using Fisher's exact test for categorical biomarkers, trend tests for ordinal biomarkers and Wilcoxon rank-sum test for continuous biomarkers. For patients undergoing surgery, changes in tissue biomarkers from baseline to surgery will be summarized descriptively in an exploratory fashion.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2012 (estimated)
- Study start12 Jul 2012
- Primary completion16 Jul 2021
- Study completion16 Jul 2021
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 Fibrolamellar CarcinomaStudies 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
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Fibrolamellar Cancer Foundation, Dana-Farber Cancer Institute, Johns Hopkins University, University of California, San Francisco, Abbott, Novartis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Ghassan Abou-Alfa, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01642186
- Sponsor's protocol ID
- 11-211
- Status last verified
- Jul 2021
- First submitted
- 12 Jul 2012
- Last updated
- 29 Dec 2022
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Lim II, Farber BA, LaQuaglia MP. Advances in fibrolamellar hepatocellular carcinoma: a review. Eur J Pediatr Surg. 2014 Dec;24(6):461-6. doi: 10.1055/s-0034-1396420. Epub 2014 Dec 8.
PubMed 25486412
Study at a glance
From the registry record, last updated by the sponsor on 29 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Three Arm Phase II Study of (1) Everolimus, (2) Estrogen Deprivation Therapy (EDT) With Leuprolide + Letrozole and (3) Everolimus + EDT in Patients With Unresectable Fibrolamellar Hepatocellular Carcinoma (FLL-HCC)
- ClinicalTrials.gov ID
- NCT01642186
- Acronym / sponsor ID
- 11-211
- Keywords
- LETROZOLE, LUPRON DEPOT, RAD001 (EVEROLIMUS), 11-211
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Fibrolamellar Cancer Foundation, Dana-Farber Cancer Institute, Johns Hopkins University, University of California, San Francisco, Abbott, Novartis Pharmaceuticals
- Study start
- 12 Jul 2012 (actual)
- Primary completion
- 16 Jul 2021 (actual)
- Study completion
- 16 Jul 2021 (actual)
- First posted
- 17 Jul 2012 (estimated)
- Results
- Posted 29 Dec 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
There is no effective standard treatment for fibrolamellar liver cancer that cannot be removed by surgery. The investigators want to find out what effects, good and/or bad, 3 drugs called letrozole, leuprolide and everolimus will have on cancer. All of these drugs are FDA approved for the treatment of different cancers. Letrozole and leuprolide stop the body from producing estrogen, a normal hormone produced by the body. Too much estrogen may help fibrolamellar liver cancer grow. Everolimus is a drug that may block other chemicals in the body that can help cancer grow. The combination of letrozole and leuprolide plus everolimus may work well together.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Arm A everolimus | Experimental | Drug: everolimusEverolimus will be administered at the following doses: * Patients with a BSA ≤ 1.5 m2 will receive everolimus 5 mg PO QD. * Patients with a BSA > than or = to 1.5 m2 will receive everolimus 7.5 mg PO QD. If the patient is randomized to everolimus alone (1) or leuprolide and letrozole (2) and the cancer continues to grow, they may have the option to receive all three drugs together. |
| Arm B letrozole plus leuprolide | Experimental | Drug: letrozole plus leuprolideDay 1 of Cycle 1: Leuprolide 7.5 mg IM will be administered by a nurse in clinic. Letrozole will be dispensed and will be taken at home. Patients will be instructed to take letrozole at the same time each day, consistently with food or without food, and swallowed whole with a glass of water. If the patient is randomized to everolimus alone (1) or leuprolide and letrozole (2) and the cancer continues to grow, they may have the option to receive all three drugs together. |
| Arm C combination everolimus, letrozole and leuprolide | Experimental | Drug: combination of everolimus, letrozole and leuprolide* Patients with a BSA ≤ 1.5 m2 will receive everolimus 5 mg PO QD. * Patients with a BSA > than or = to 1.5 m2 will receive everolimus 7.5 mg PO QD. Leuprolide 7.5 mg IM will be given every 4 weeks (+/- 7 days). Everolimus and letrozole will be administered continuously using the same dose, schedule and administration. Everolimus, letrozole and leuprolide should be administered concurrently at all times. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Patients ≥ 12 years old.
- Pathologically confirmed diagnosis of advanced and/or unresectable FLL-HCC. This will be performed by the participating centers on submitted specimens. If the submitted material is insufficient for analysis, a repeat biopsy is recommended.
- ECOG performance status 0-2 ; Lansky performance score of ≥ 60% for patients 12-16 years old
- Adequate hematologic, renal and hepatic function defined as:
- Hematologic: ANC ≥ 1.0 x 10^9/L, platelets ≥ 50 x 10^9/L o Renal: creatinine ≤ 2 x upper limit of normal, or creatinine Clearance of ≥60 cc/mL/1.73 m^2 for patients > 16 years old. For patients ≤ 16 years of age, creatinine Clearance of ≥70 cc/mL/1.73 m^2 or serum creatinine based on the following chart: Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m^2 or serum creatinine based on age/gender as follows: Age Maximum Serum Creatinine (mg/dL) Male Female 10 to < 13 years 1.2 1.2 13 to < 16 years 1.5 1.4
Key exclusion criteria (17 listed)
- Concurrent anticancer, or radiation therapy. Patients must have completed all anticancer therapy > 4 weeks before the start of study therapy. The date of last palliative radiation must be > 2 weeks from the start of study therapy. Palliative radiation is permitted on protocol with MSK PI discretion on treatment modifications.
- Patients, who have had a major surgery or significant traumatic injury within 4 weeks of start of study drug, patients who have not recovered from the side effects of any major surgery (defined as requiring general anesthesia) .
- Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Topical or inhaled corticosteroids are allowed.
- Concurrent oral contraceptive use or hormonal replacement therapy.
- Use of an aromatase inhibitor, GnRH agonist and/or tamoxifen within the past 30 days. Patients previously on fulvestrant or a q3 month GnRH agonist must have discontinued these medications for at least 3 months.
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 |
|---|---|---|
| everolimus | Drug | Arm A everolimus |
| letrozole plus leuprolide | Drug | Arm B letrozole plus leuprolide |
| combination of everolimus, letrozole and leuprolide | Drug | Arm C combination everolimus, letrozole and leuprolide |
Primary outcome
Efficacy Endpoints for Part 1 of the Study is Progression-free Survival at 6 Months (PFS6)
Time frame: 6 months
for Part 1 of the study is progression-free survival at 6 months (PFS6). A progression event refers to the first evidence of radiographic disease progression, clinical progression as determined by study investigators, or death. Imaging performed in 6 months will be used to determine PFS6.
5 secondary outcomes
Median PFS
Time frame: 2 years
Median Overall Survival (OS)
Time frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months
Percentage of Participants With Stable Disease
Time frame: 2 years
Number of Participants With One or More Adverse Events/Toxicity
Time frame: 2 years
Number of Participants With Tissue Biomarkers Collected
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Dec 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 25486412Lim II, Farber BA, LaQuaglia MP. Advances in fibrolamellar hepatocellular carcinoma: a review. Eur J Pediatr Surg. 2014 Dec;24(6):461-6. doi: 10.1055/s-0034-1396420. Epub 2014 Dec 8.
- 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.
Ghassan Abou-Alfa, MD principal investigator
Memorial Sloan Kettering Cancer Center
6 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 everolimus, letrozole plus leuprolide and combination of everolimus, letrozole and leuprolide in people with Fibrolamellar Carcinoma and Fibrolamellar Liver Cancer, run by Memorial Sloan Kettering Cancer Center at 6 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 15 inclusion and 17 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 29 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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 29 Dec 2022). 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?
Efficacy Endpoints for Part 1 of the Study is Progression-free Survival at 6 Months (PFS6) (time frame: 6 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 NCT01642186, 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 29 Dec 2022.