Poorly Differentiated Malignant Neuroendocrine CarcinomaPhase 2EVINECNCT02113800
Safety and Tolerability of Everolimus as Second-line Treatment in Poorly Differentiated Neuroendocrine Carcinoma / Neuroendocrine Carcinoma G3 (WHO 2010) and Neuroendocrine Tumor G3 - an Investigator Initiated Phase II Study
A Phase 2 study testing Everolimus (Afinitor®) in people with Poorly Differentiated Malignant Neuroendocrine Carcinoma and Neuroendocrine Carcinoma, Grade 3, run by AIO-Studien-gGmbH at 1 site in Germany.
Trial summary
The study is designed as an open-label, prospective, single arm, multicenter study of everolimus in histologically confirmed, neuroendocrine carcinoma G3 /neuroendocrine tumor G3 after failure of first-line platin-based chemotherapy (open-label pilot study). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Poorly Differentiated Malignant Neuroendocrine Carcinoma, Neuroendocrine Carcinoma, Grade 3, Neuroendocrine Carcinoma, Grade 1 [Well-differentiated Neuroendocrine Carcinoma] That Switched to G3
- What is being tested?
- Everolimus (Afinitor®)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteGermany
- Study dates
- Aug 2015to Apr 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Signed written informed consent
- Male or female ≥ 18 years of age
- Patients with poorly differentiated neuroendocrine carcinoma, neuroendocrine carcinoma G3 (NEC - G3 according to WHO 2010) or well or moderately differentiated neuroendocrine carcinoma (NET - G1 / G2) that switched to G3 (confirmed by histology) or neuroendocrine tumor G3 (NET G3) and disease progression as measured by RECIST 1.1
- Progression during or after treatment with first-line platinbased chemotherapy. In NET G3 that switched from NET G2 the line of therapy is determined from the time of revised histology (confirming a G3 NEN)
- Measurable disease according to RECIST 1.1
- Performance Status according to Eastern Cooperative Oncology Group (ECOG) status 0 - 2 (Karnofsky Performance status ≥ 80%)
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- Women of child-bearing potential must have a negative pregnancy test
- Laboratory requirements:
- Hematology
- Absolute neutrophil count ≥ 1.5 x 109/L
- Platelet count ≥ 100 x 10^9/L
- Leukocyte count ≥ 3.0 x 10^9/L
- Hemoglobin ≥ 9 g/dL or 5.59 mmol/L
- Hepatic Function
- Total bilirubin ≤ 1.5 time the upper limit normal (ULN)
- Aspartate Aminotransferase (AST) ≤ 3 x ULN in absence of liver metastases, or ≤ 5 x ULN in presence of liver metastases
- Alanine Aminotransferase (ALT) ≤ 3 x ULN in absence of liver metastases, or ≤ 5 x ULN in presence of liver metastases
- Renal Function
- Creatinine clearance ≥ 50 mL/min according to cockroft-Gault formula
- Metabolic Function
- Magnesium ≥ lower limit of normal
- Calcium ≥ lower limit of normal
- Others:
- CRP (PCT if CRP is elevated to exclude infection)
- negative urinary screening test for leukocytes and nitrite (U - stix) to exclude urinary tract infection
Who cannot join
- Known or suspected allergy or hypersensitivity reaction to any of the components of study treatment or their excipients.
- Previous therapy with mTOR inhibitor
- Radiotherapy :
- Concurrent radiotherapy involving target lesions used for this study.
- Concurrent palliative radiation (but radiation for non-target lesions is allowed if other target lesions are available outside the involved field)
- previous pre-operative or post-operative radiotherapy within 3 months before study treatment
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- History of other malignant tumors within the last 5 years, except basal cell carcinoma or curatively excised cervical carcinoma in situ
- Known brain metastases unless adequately treated (surgery or radiotherapy) with no evidence of progression and neurologically stable off anticonvulsants and steroids
- Clinically significant cardiovascular disease (including myocardial infarction, unstable angina, symptomatic congestive heart failure, serious uncontrolled cardiac arrhythmia) ≤ 1 year before enrolment
- Inadequate pulmonary function according to the Investigator's judgment, history of interstitial lung disease e.g. pneumonitis or pulmonary fibrosis or evidence of interstitial lung disease on baseline chest CT scan
- Known active Hepatitis B Virus (HBV), Hepatitis C Virus (HCV) or HIV infection
- Serious concomitant disease or medical condition that in the judgment of the investigator renders the patient at high risk from treatment complication
- Any systemic disease requiring oral intake of corticosteroids (except for replacement therapy of corticosteroids - hydrocortisone in case of adrenal or pituitary insufficiency)
- Hearing loss ≥ Grade 3 (CTCAE v4.03)
- Patient pregnant or breast feeding, or planning to become pregnant within 8 weeks after the end of treatment
- Patient (male or female) is not willing to use highly effective methods of contraception (per institutional standard) during treatment and for 8 weeks (male or female) after the end of treatment.
- Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 28 days prior to treatment start
- Concurrent treatment with inhibitors (e.g. itraconazole, ketoconazole) and inducers (e.g. phenytoin, rifampicin) of Cytochrome P450 3A4 (CYP3A4) and / or the multidrug efflux pump P-glycoprotein (PgP).
- Known drug abuse/alcohol abuse
- Peripheral polyneuropathy ≥ Grade 2 (CTCAE v4.03)
- Active chronic inflammatory bowel disease
- Any condition which might interfere with study objectives (e.g. infections) or would limit the patient's ability to complete the study in the opinion of the investigator
- Patient who has been incarcerated or involuntarily institutionalized by court order or by the authorities. (AMG §40, Abs. 1 No. 4)
- Affected persons who might be dependent on the sponsor or the investigator
Trial locations and contacts
1 site in 1 country.
Germany1 site
Charité-Universitätsmedizin, Medizinische Klinik m. S. Hepatologie und Gastroenterologie, Campus Virchow-Klinikum
Berlin, 13353
About this study
The study is designed as an open-label, prospective, single arm, multicenter study of everolimus in histologically confirmed, neuroendocrine carcinoma G3 /neuroendocrine tumor G3 after failure of first-line platin-based chemotherapy (open-label pilot study).
The aim of this study is to provide a second line therapy to patients with any type of platinum based first line chemotherapy, to gather data on disease control rate and progression free survival.
Read the detailed description
As more efficient drugs are urgently needed for the treatment of neuroendocrine tumors the investigator evaluated phosphorylated Mammalian target of rapamycin (mTOR) and effectors in a series of NEC G3 at the Charité Center. Everolimus showed antiproliferative effects in bronchial NET.
In a second approach the data of this study should be the basis to generate another study to further explore everolimus as maintenance therapy in NEC G3/ NET G3.
- Conditions
- Poorly Differentiated Malignant Neuroendocrine Carcinoma, Neuroendocrine Carcinoma, Grade 3, Neuroendocrine Carcinoma, Grade 1 [Well-differentiated Neuroendocrine Carcinoma] That Switched to G3, Neuroendocrine Carcinoma, Grade 2 [Moderately Differentiated Neuroendocrine Carcinoma] That Switched to G3, Neuroendocrine Tumor, Grade 3 and Disease Progression as Measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1.)
- Being tested
- Everolimus (Afinitor®)
- Medical subject headings
- Carcinoma, Neuroendocrine, Lymphoma, Follicular, Neuroendocrine Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Everolimus (Afinitor®)Drug
Formulation: 10 mg/day Route: oral (tablet)
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Patients receive Everolimus orally, 10 mg/day.
The end of study will be performed when tumor progression has been observed for 28 patients. Patients who are still under treatment at that time may continue with chemotherapy at the discretion of the investigator, but will be excluded from the study. Receives: Drug: Everolimus (Afinitor®) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events (AEs)
Incidence of adverse events (AEs) overall and by severity, and serious adverse events (SAEs). Severity will be assessed using the National Cancer Institute-Common Toxicity Criteria (NCI-CTC) for Adverse Events, version 4.03 (CTCAEv4.03).
To evaluate tolerability and safety of everolimus in second-line treatment of poorly differentiated neuroendocrine carcinoma / neuroendocrine carcinoma G3 according to WHO 2010 and neuroendocrine tumors G3.
Measured: approx. 18 month
Secondary outcomes (11)
Progression free survival (PFS)
Progression free survival (PFS) as the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse as per local radiology assessment using Response Evaluation Criteria in Solid Tumors (RECIST 1.1.)
Measured: approx. 18 month
Objective response rate (ORR)
Objective response rate defined as the rate of best overall response (CR+PR), determined by RECIST V 1.1.
Measured: approx. 18 month
Disease control rate (DCR)
Disease control rate defined as the rate of best overall response and stable disease (CR+PR+SD), determined by RECIST V 1.1.
Measured: approx. 18 month
Duration of response (DR)
Duration of response is defined as the time from onset of the first objective tumor response (CR/PR) to objective tumor progression or death from any cause.
Measured: approx. 18 month
Overall Survival (OS)
OS is defined as the time from date of randomization to the date of death from any cause. If a patient is not known to have died at the date of analysis cut-off, the OS will be censored at the last date of contact.
Measured: approx. 18 month
Quality of life
Quality of life (HRQoL) will be evaluated using the European Organisation for Research and Treatment of Cancer (EORTC), to assess the quality of life of cancer patients questionnaire (QLQ-C30)
Measured: approx. 18 month
chromogranin A & B
Percentage of patients showing normalization or a decrease of chromogranin A \& B
Measured: approx. 12 month
Time to Progression (TTP)
Time to progression (TTP) is the time from date of start of treatment to the date of event defined as the first documented progression due to underlying cancer.
Measured: approx. 18 month
neuron-specific enolase
Percentage of patients showing normalization or a decrease of neuron-specific enolase
Measured: approx. 12 month
progastrin releasing peptide
Percentage of patients showing normalization or a decrease of progastrin releasing peptide.
Measured: approx. 12 month
Correlation mTOR pathway components in tumor tissue to tumor response
To explore expression of mTOR pathway components in tumor tissue (archive) in correlation to tumor response
Measured: approx. 18 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2014 (estimated)
- Study startAug 2015
- Primary completionApr 2020
- Study completionApr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Poorly Differentiated Malignant Neuroendocrine CarcinomaStudies of the same condition, open and under way.
- 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
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- AIO-Studien-gGmbH
- Sponsor type
- University, hospital or other
- Collaborators
- Assign Data Management and Biostatistics GmbH, Novartis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Marianne Pavel, Prof. Dr., Principal investigator, Charité-Universitätsmedizin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02113800
- Sponsor's protocol ID
- AIO-NET-0112
- Other IDs
- CRAD001KDE55T, 2012-004550-28
- Acronym
- EVINEC
- Status last verified
- Oct 2020
- First submitted
- 8 Apr 2014
- Last updated
- 28 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
Publications
Jungels C, Deleporte A. State of the art and future directions in the systemic treatment of neuroendocrine neoplasms. Curr Opin Oncol. 2021 Jul 1;33(4):378-385. doi: 10.1097/CCO.0000000000000740.
PubMed 33973550
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Tolerability of Everolimus as Second-line Treatment in Poorly Differentiated Neuroendocrine Carcinoma / Neuroendocrine Carcinoma G3 (WHO 2010) and Neuroendocrine Tumor G3 - an Investigator Initiated Phase II Study
- ClinicalTrials.gov ID
- NCT02113800
- Acronym / sponsor ID
- EVINEC / AIO-NET-0112
- Conditions
- Poorly Differentiated Malignant Neuroendocrine Carcinoma, Neuroendocrine Carcinoma, Grade 3, Neuroendocrine Carcinoma, Grade 1 [Well-differentiated Neuroendocrine Carcinoma] That Switched to G3, Neuroendocrine Carcinoma, Grade 2 [Moderately Differentiated Neuroendocrine Carcinoma] That Switched to G3, Neuroendocrine Tumor, Grade 3 and Disease Progression as Measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1.)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- AIO-Studien-gGmbHUniversity, hospital or other
- Collaborators
- Assign Data Management and Biostatistics GmbH, Novartis Pharmaceuticals
- Study start
- Aug 2015
- Primary completion
- Apr 2020 (actual)
- Study completion
- Apr 2020 (actual)
- First posted
- 15 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is designed as an open-label, prospective, single arm, multicenter study of everolimus in histologically confirmed, neuroendocrine carcinoma G3 /neuroendocrine tumor G3 after failure of first-line platin-based chemotherapy (open-label pilot study).
The aim of this study is to provide a second line therapy to patients with any type of platinum based first line chemotherapy, to gather data on disease control rate and progression free survival.
Detailed description
As more efficient drugs are urgently needed for the treatment of neuroendocrine tumors the investigator evaluated phosphorylated Mammalian target of rapamycin (mTOR) and effectors in a series of NEC G3 at the Charité Center. Everolimus showed antiproliferative effects in bronchial NET.
In a second approach the data of this study should be the basis to generate another study to further explore everolimus as maintenance therapy in NEC G3/ NET G3.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: Everolimus (Afinitor®)Patients receive Everolimus orally, 10 mg/day. The end of study will be performed when tumor progression has been observed for 28 patients. Patients who are still under treatment at that time may continue with chemotherapy at the discretion of the investigator, but will be excluded from the study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (25 listed)
- Signed written informed consent
- Male or female ≥ 18 years of age
- Patients with poorly differentiated neuroendocrine carcinoma, neuroendocrine carcinoma G3 (NEC - G3 according to WHO 2010) or well or moderately differentiated neuroendocrine carcinoma (NET - G1 / G2) that switched to G3 (confirmed by histology) or neuroendocrine tumor G3 (NET G3) and disease progression as measured by RECIST 1.1
- Progression during or after treatment with first-line platinbased chemotherapy. In NET G3 that switched from NET G2 the line of therapy is determined from the time of revised histology (confirming a G3 NEN)
- Measurable disease according to RECIST 1.1
Key exclusion criteria (24 listed)
- Known or suspected allergy or hypersensitivity reaction to any of the components of study treatment or their excipients.
- Previous therapy with mTOR inhibitor
- Radiotherapy :
- History of other malignant tumors within the last 5 years, except basal cell carcinoma or curatively excised cervical carcinoma in situ
- Known brain metastases unless adequately treated (surgery or radiotherapy) with no evidence of progression and neurologically stable off anticonvulsants and steroids
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 (Afinitor®)Formulation: 10 mg/day Route: oral (tablet) | Drug | Single Arm |
Primary outcome
Incidence of adverse events (AEs)
Time frame: approx. 18 month
Incidence of adverse events (AEs) overall and by severity, and serious adverse events (SAEs). Severity will be assessed using the National Cancer Institute-Common Toxicity Criteria (NCI-CTC) for Adverse Events, version 4.03 (CTCAEv4.03). To evaluate tolerability and safety of everolimus in second-line treatment of poorly differentiated neuroendocrine carcinoma / neuroendocrine carcinoma G3 according to WHO 2010 and neuroendocrine tumors G3.
11 secondary outcomes
Progression free survival (PFS)
Time frame: approx. 18 month
Objective response rate (ORR)
Time frame: approx. 18 month
Disease control rate (DCR)
Time frame: approx. 18 month
Duration of response (DR)
Time frame: approx. 18 month
Overall Survival (OS)
Time frame: approx. 18 month
Quality of life
Time frame: approx. 18 month
chromogranin A & B
Time frame: approx. 12 month
Time to Progression (TTP)
Time frame: approx. 18 month
neuron-specific enolase
Time frame: approx. 12 month
progastrin releasing peptide
Time frame: approx. 12 month
Correlation mTOR pathway components in tumor tissue to tumor response
Time frame: approx. 18 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2020.
- Derived from this study: PubMed 33973550Jungels C, Deleporte A. State of the art and future directions in the systemic treatment of neuroendocrine neoplasms. Curr Opin Oncol. 2021 Jul 1;33(4):378-385. doi: 10.1097/CCO.0000000000000740.
- Working Group for Medical Oncology (AIO) from the German Cancer Society (DKG)Link 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.
Marianne Pavel, Prof. Dr. principal investigator
Charité-Universitätsmedizin
1 site 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 (Afinitor®) in people with Poorly Differentiated Malignant Neuroendocrine Carcinoma and Neuroendocrine Carcinoma, Grade 3, run by AIO-Studien-gGmbH at 1 site in Germany.
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 25 inclusion and 24 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 28 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of adverse events (AEs) (time frame: approx. 18 month). 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 NCT02113800, 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 28 Oct 2020.