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Sandostatin LAR and Axitinib vs Pbo in Pnts With Advanced Well-differentiated Non-pancreatic Neuroendocrine Carcinomas

Status

Completed

Phase

Phase 2 / Phase 3

Enrollment

256

Locations

26

Results

Posted

Publications

2

Study summary

What the protocol is testing.

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.

Full detailed description

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%).

Interventions

Treatment arms and agents.

DRUG

Axitinib

Orally, 5mg, twice daily, until progression or until unacceptable toxicity, with or without food intake.

DRUG

Sandostatin LAR

Intramuscular, 30mg, single injection every 28 days, until disease progression or unacceptable toxicity

DRUG

Placebo

orally, twice daily, until disease progression or unacceptable toxicity, with or without food intake.

Timeline

From registration to results.

  1. First posted

    Dec 6, 2012

  2. Study start

    Nov 2011

  3. Primary completion

    Dec 31, 2023

  4. Study completion

    Dec 31, 2023

  5. Results posted

    Apr 28, 2026

  6. Registry updated

    Apr 28, 2026

Outcomes

What the study measures.

Primary outcomes

Efficacy of Axitinib in Terms of PFS (Investigator 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

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

Secondary outcomes

Objective Response Rate (ORR) (Investigator 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

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.

Biochemical Response (5-OH-indoleacetic Acid and Chromogranin A)

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

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.

Safety and Tolerability of Axitinib (National Cancer Institute Common Terminology Criteria for Adverse Events [NCI-CTCAE], Version 4.0)

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

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)

Efficacy of Axitinib in Terms of PFS (Central Blinded 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

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

Objective Response Rate (ORR) (Central Blinded 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

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.

Eligibility

Who can take part.

Minimum age
18 Years
Maximum age
Not reported
Sex
ALL
Healthy volunteers
No

Inclusion Criteria: 1. G1-G2 neuroendocrine tumor (WHO 2010) of histologically confirmed non-pancreatic origin, functioning and nonfunctioning 2. Metastatic or locally advanced disease not amenable to treatment with curative intent 3. Clinical and/or radiological disease progression documented in the 12 months prior to study entry. 4. Patients should have at least one measurable lesion as defined by RECIST 1.1 criteria. Patients should not have undergone local or regional ablative procedures (embolization, cryoablation, radiofrequency ablation, or others) in the 6 months prior to entering the study, unless there are other locations of measurable disease or clear radiological progression after carrying out these procedures (in these cases, local and regional ablation procedures shall be permitted if they have been performed at least 1 month prior to enrollment in the study). 5. Ki-67 \< 20% 6. Prior treatment with somatostatin analogues is allowed 7. Prior treatment with interferon is allowed 8. Prior treatment is allowed with up to 2 antineoplastic systemic treatment lines different from SAs or IFN (systemic treatment is understood as conventional cytotoxic chemotherapy or new drugs for therapeutic targets as mTOR or other, as long as it is not directed against VEGF/VEGFR). Treatment with SAs or IFN does not count as prior lines of antineoplastic treatment. 9. Prior treatment with targeted therapy against VEGF or VEGFR is not allowed. 10. Adequate organ function as defined by the following criteria: * Absolute neutrophil count ≥ 1500 cells/mm3, * Platelet count ≥ 75,000 cells/mm3, * Hemoglobin ≥ 9.0 g/dL, * AST y ALT ≤ 2.5 x upper limit of normal (ULN), except if liver metastases exist, in which case AST and ALT 5.0 ≤ x ULN is allowed, * Total bilirubin ≤ 1.5 x ULN, * Serum creatinine ≤ 1.5 x ULN or calculated creatinine clearance ≥ 60 mL/min, * Proteinuria \< 2+ by reactive strip. If the reactive strip is ≥ 2+, a 24-hour urine sample should be collected and the patient may be eligible if urinary protein excretion is \< 2 g every 24 hours. 11. Men or women aged ≥ 18 years. 12. ECOG performance status 0-2 13. Life expectancy ≥ 12 weeks 14. At least 4 weeks should pass from the end of the previous systemic treatment with resolution of all treatment-related toxicities to grade ≤ 1 according to NCI CTCAE Version 4.0 or to baseline, except for alopecia or properly treated hypothyroidism. 15. No prior evidence of uncontrolled hypertension should exist, as documented by 2 baseline blood pressure readings taken at least 1 hour apart. Baseline readings of systolic blood pressure should be ≤ 150 mm Hg and baseline readings of diastolic pressure should be ≤ 90 mm Hg. Patients whose hypertension is being controlled with antihypertensive therapy are eligible. 16. Women (or their partners) should be surgically sterilized or postmenopausal, or must agree to use an effective contraceptive method during and for at least 6 months after receiving study treatment. All women of childbearing age should have a negative pregnancy test (serum/urine) within 7 days prior to starting treatment. Men (or their partners) should be surgically sterilized or must agree to use an effective contraceptive method during and for at least 6 months after receiving study treatment. The definition of an effective contraceptive method must comply with local regulations and will be based on the criterion of the principal investigator or a designated associate. Lactating women may not participate in this study. 17. Signed and dated informed consent document stating that the patient has been informed of all the pertinent aspects of the trial prior to recruitment. 18. Willingness and ability to comply with scheduled visits, treatment plans (including willingness to take axitinib or placebo according to randomization), laboratory tests, and other study procedures. Exclusion Criteria: 1\. Subjects must be evaluated with regard to the following exclusion criteria: 1. The following types of endocrine tumors will not be included: paraganglioma, adrenal endocrine tumor, thyroid, parathyroid, or pituitary. 2. Major surgery within previous 4 weeks, or radiation therapy within 2 weeks prior to the start of treatment. Prior palliative radiotherapy for metastatic lesions is permitted if there is at least one measurable lesion that has not been irradiated (i.e., if there are other non-irradiated target lesions). 3. Gastrointestinal abnormalities, including: * Inability to swallow oral medication; * Need for intravenous feeding; * Prior surgical procedures that affect absorption, including total gastric resection; * Treatment for active peptic ulcer in the last 6 months; * Uncontrolled active gastrointestinal bleeding unrelated to cancer, as evidenced by hematemesis, hematochezia or clinically significant melena in the last 3 months without evidence of resolution documented by endoscopy or colonoscopy; * Malabsorption syndromes; 4. Current or anticipated need for treatment with drugs that are potent inhibitors of CYP3A4 (grapefruit juice, verapamil, ketoconazole, miconazole, itraconazole, erythromycin, telithromycin, clarithromycin, indinavir, saquinavir, ritonavir, nelfinavir, lopinavir, atazanavir, amprenavir, fosamprenavir, and delavirdine) unless they can be replaced by another medication with minimal potential for CYP3A4/5 inhibition. The use of low-dose oral steroids (\< 5 mg/day prednisone or equivalent) is allowed. Co-administration of steroids may increase plasma concentrations of axitinib. 5. Current use or anticipated need for treatment with drugs that are known potent CYP3A4/5 inducers (carbamazepine, dexamethasone, felbamate, phenobarbital, phenytoin, amobarbital, nevirapine, primidone, rifabutin, rifampicin, and St. John's wort) unless they can be replaced by another medication with minimal potential for CYP3A4 induction. Co-administration of CYP3A4/5 inducers may decrease plasma concentrations of axitinib. 6. Need for anticoagulant therapy with oral vitamin K antagonists. Low doses of anticoagulants to maintain the patency of a central venous access device or to prevent deep vein thrombosis are permitted. Use with therapeutic doses of low molecular weight heparin is allowed. 7. Clinically relevant history of bleeding in the last 6 months, including severe hemoptysis or hematuria, unless it has been due to a treated cause (e.g., completely resected bleeding intestinal tumor). 8. Active epilepsy or evidence of brain metastases, spinal cord compression, or carcinomatous meningitis. 9. Serious uncontrolled illness or active infections that may interfere with the patient's ability to receive the study treatment. 10. Any of the following events in the 12 months prior to administration of the study drug: myocardial infarction, uncontrolled angina, implantation of a coronary or peripheral bypass, symptomatic congestive heart failure, stroke or transient ischemic attack. Deep vein thrombosis or pulmonary embolism in the prior 6 months. 11. Ongoing grade ≥ 2 cardiac arrhythmias according to NCI CTCAE: atrial fibrillation of any grade or QTc interval \> 450 ms for men or \> 470 ms for women. 12. Patients with human immunodeficiency virus (HIV) infection or acquired immunodeficiency syndrome-related disease. 13. Prior history of cancer except those treated with curative intent for non-melanoma skin cancer in situ, breast or cervical cancer in situ, or those treated for any cancer with curative intent and no evidence of disease in the last 5 years prior to enrollment in the study. 14. Dementia or significantly altered mental status that could prevent compression, or submission of informed consent and compliance with the requirements of this protocol. 15. Any severe, acute or chronic medical or psychiatric condition, or laboratory abnormality that may increase the risk associated with participation in the study or with study drug administration, or that may interfere with the interpretation of results, and that could interfere with the patient's ability to take part in this study in the investigator's opinion. 16. The patient's participation or intention to participate (in the 4 weeks prior to starting drug administration) in a study in which the patient will receive an investigational medicinal product. 17. Subjects who are institutionalized by governmental or by judicial decision, or subjects who are dependent of the sponsor, the investigator or the trial site will be excluded from participation.

Study locations

26 registered sites.

Germany · Italy · Spain · United Kingdom. Showing up to 24 locations stored in the fast local snapshot.

Marburg Universitätsklinikum Giessen und Marburg GmbH

Marburg, Germany

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

Hospital de Donostia

Donostia / San Sebastian, Spain

Hospital Virgen de las Nieves

Granada, Spain

Hospital universitario de Leon

León, Spain

MD Anderson Cancer Center

Madrid, Spain

Hospital Clara Campal

Madrid, Spain

Hospital Clínico San Carlos

Madrid, Spain

Hospital Gregorio Marañón

Madrid, Spain

Hospital Univ La Paz

Madrid, Spain

Hospital Universitario 12 de Octubre

Madrid, Spain

Hospital Universitario Ramón y Cajal

Madrid, Spain

Hospital Univ de Salamanca

Salamanca, Spain

Hospital Marqués de Valdecilla

Santander, Spain

Hospital Universitario Virgen del Rocío

Seville, Spain

Hospital General Universitario de Valencia

Valencia, Spain

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