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
255
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
Effectiveness of axitinib in terms of PFS
calculated from the date of random assignment until the date of first progressive disease or tumor-related death
Time frame: until disease progression, end of treatment or minimum 6 months
Objective response rate (ORR) and the duration of response.
Measured according to RECIST 1.1 criteria; Sum of longest diameter of target lesions measured in mm
Time frame: Until disease progression, end of treatment or minimum 6 months
Functional response rate using F-DOPA-PET (optional, depending on availability)
measured in SUV (standardized uptake value)
Time frame: Until disease progression, end of treatment or minimum 6 months
Biochemical response (5-OH-indoleacetic acid and chromogranin A)
measurable in mL/ 24h and ng/ml respectively, through blood and urine test
Time frame: Until death, last follow-up, or minimum 6 months
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: Until disease progression, end of treatment or minimum 6 months
Explore potential biomarkers
The following parameters will be measured: circulating tumor cells, circulating endothelial cells, hypertension (mmHg), and other serum or tumoral biomarkers of angiogenesis). Peripheral blood samples (9ml) will be obtained before treatment, after 1 month since the start of treatment and after disease progression and or the end of study visit. The blood samples will be processed for mRNA extraction. Hypoxia dependant genes and marker genes which transcription depends on the activation of VEGF, will be analyzed using qPCR. The dynamic profile of those genes will then be analyzed in relation to the response to axitinib, evaluating their predictive value of response. Paraffin-embedded tumor tissue will also be collected from all patients to investigate the prognostic value and predictive potential of the different intracellular pathways related to VEGFR, PDGFR and other signaling pathways.
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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
Time frame: Until disease progression or or minimum 6 months
Evaluate overall survival.
Time frame: from the date of randomization to the date of death from any cause whichever came first, assessed up to 50 months.