Assess whether therapy with axitinib, a potent angiogenic inhibitor of the tyrosine kinase receptors of VEGF bioavailable by oral administration, is capable of improving PFS in patients with advanced G1-G2 NETs of nonpancreatic origin with progressive disease documented in the 12 months prior to entering the study.
Phase II/III, prospective, multicenter, randomized (1:1), double-blind study to evaluate the efficacy and tolerability of axitinib in patients diagnosed with advanced G1-G2 neuroendocrine tumors (WHO 2010) of nonpancreatic origin that have presented documented disease progression in the 12 months prior to entering the study. In the first part of the study (Phase II), 105 patients were enrolled. The second part of the study is the expansion to Phase III, which is expected to include 148 additional patients. Patients will be randomized to receive Sandostatin LAR with axitinib or Sandostatin LAR with placebo until disease progression or unacceptable toxicity occurs. Randomization will be stratified by the time from diagnosis to enrollment in the study (more vs less than or equal to 12 months), the origin of the primary tumor (gastrointestinal tract vs non-gastrointestinal tract \[lung or other sites\]) and ki-67 (\< 5% vs \> 5%).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
256
Orally, 5mg, twice daily, until progression or until unacceptable toxicity, with or without food intake.
Intramuscular, 30mg, single injection every 28 days, until disease progression or unacceptable toxicity
orally, twice daily, until disease progression or unacceptable toxicity, with or without food intake.
Marburg Universitätsklinikum Giessen und Marburg GmbH
Marburg, Germany
Efficacy of Axitinib in Terms of PFS (Investigator Assessment)
Calculated from the date of random assignment until the date of first progressive disease or death, whichever occurs first. Progression of the disease was assessed by investigators using tumor imaging computed tomography scans and Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 PFS was analysed by Kaplan-Meier method and compared with log-rank tests. Patients alive with no event at data cut off were censored on their last tumor assessment
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 approximately 25 months
Objective Response Rate (ORR) (Investigator Assessment)
Measured changes in the sum of longest diameter of target lesions measured in mm according to RECIST 1.1 criteria. Assessed by investigators. Patients are categorized depending on the percentage of tumor redution: Complete response (CR): dissapearance of all lesions Partial response (PR): reduction \> 30% in the sum of longest diameter of target lesions Stable disease (SD): Tumor size betwee 30% reduction and 20% increase in the sum of longest diameter of target lesions Progressive disease (PD): increase \> 20% in the sum of longest diameter of target lesions ORR comprise all patients who achieved at least a CR or PR at any timepoint during follow-up.
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 approximately 25 months
Biochemical Response (5-OH-indoleacetic Acid and Chromogranin A)
measurable in mL/ 24h and ng/ml respectively, through blood and urine test in patients with baseline elevation of CgA or 5-HIAA levels. This endpoint measures the negativization of these two tumor biomarkers.
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 approximately 25 months
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Azienda Ospedaliera Universitaria di Perugia
Perugia, Italy
Sapienza, Universitá di Roma, Ospedale sant'Andrea
Rome, Italy
Institut Català d'Oncologia L'Hospitalet
L'Hospitalet de Llobregat, Barcelona, Spain
Hospital Universitario Virgen de la Victoria
Málaga, Malaga, Spain
Hospital Alvaro Cunqueiro
Vigo, Pontevedra, Spain
Hospital Central de Asturias
Oviedo, Principality of Asturias, Spain
Complejo Hospitalario Univ A Coruña
A Coruña, Spain
Hospital Universitari Vall d'Hebron
Barcelona, Spain
Hospital Universitario de Burgos
Burgos, Spain
...and 16 more locations
Safety and Tolerability of Axitinib (National Cancer Institute Common Terminology Criteria for Adverse Events [NCI-CTCAE], Version 4.0)
All adverse events and serious adverse events will be monitored with regular monitoring of hematology and blood chemistry parameters and regular physical examinations. Adverse events will be evaluated continuously throughout the study. Safety and tolerability will be assessed according to the National Institute of Health/National Cancer Institute (NIH/NCI) Common Terminology Criteria for Adverse Events version 4 (CTCAE v4)
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 approximately 25 months
Efficacy of Axitinib in Terms of PFS (Central Blinded Assessment)
Calculated from the date of random assignment until the date of first progressive disease or death, whichever occurs first. Progression of the disease was assessed by central blinded reviewers using tumor imaging by computed tomography scans and Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 PFS was analysed by Kaplan-Meier method and compared with log-rank tests. Patients alive with no event at data cut off were censored on their last tumor assessment
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 approximately 25 months
Objective Response Rate (ORR) (Central Blinded Assessment)
Measured changes in the sum of longest diameter of target lesions measured in mm according to RECIST 1.1 criteria. Assessed by central blinded reviewers. Patients are categorized depending on the percentage of tumor redution: Complete response (CR): dissapearance of all lesions Partial response (PR): reduction \> 30% in the sum of longest diameter of target lesions Stable disease (SD): Tumor size betwee 30% reduction and 20% increase in the sum of longest diameter of target lesions Progressive disease (PD): increase \> 20% in the sum of longest diameter of target lesions ORR comprise all patients who achieved at least a CR or PR at any timepoint during follow-up.
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 approximately 25 months