Clinical trial NCT02842749: Phase IV Study of the Safety and Efficacy of Everolimus in Adult Patients With Progressive pNET in China
Official title: Phase IV, Open-label, Multi-center, Single-arm Study of the Safety and Efficacy of Everolimus (Afinitor) in Adult Patients With Locally Advanced, Unresectable or Metastatic, Well Differentiated Progressive Pancreatic Neuroendocrine Tumors (pNET) in China.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Pancreatic Neuroendocrine Tumors
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2016-07-25
Enrollment: 61
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate safety and efficacy of everolimus (Afinitor®) in Chinese adult patients with local advanced or metastatic, well differentiated progressive pancreatic neuroendocrine tumors.

Detailed description: This was an open-label, multicenter, single-arm clinical study to evaluate the safety and efficacy of everolimus in Chinese adult patients with locally advanced, unresectable or metastatic, well differentiated progressive pancreatic neuroendocrine tumors. The inclusion and exclusion criteria, as well as the dosing and dose modification criteria are designed according to the approved Chinese Package Insert. The planned sample size of the study was approximately 60 subjects.

Subjects who were eligible received the treatment with everolimus provided by sponsor to treat pNET and followed the visit schedule in the protocol to collect safety and efficacy data until progression of disease, unacceptable toxicity, death, protocol deviation or other reason that may lead to discontinuation before the end of study. All subjects were followed-up for survival status every 6 months by the investigator until death, lost to follow-up, withdrawal of consent for survival or end of study.

The "End of study" is defined as either at least 75% of subjects have completed survival follow up or all subjects discontinued study treatment or the last subject finished 5-year survival follow up, whichever comes first. Final analysis was conducted at the end of the study.

Eligibility criteria: Inclusion Criteria:

* Patients must have histological confirmed G1 or G2 pancreatic neuroendocrine tumors(pNETs) (WHO 2010)
* Patients must have radiological documentation of progression of disease per RECIST 1.1 within 12 months prior to enrollment.
* Measurable disease per RECIST 1.1 criteria using triphasic computed tomography (CT) scan or multiphase MRI for radiologic assessment.
* everolimus treatment which is recommended by the treating physician

Exclusion Criteria:

* Hypersensitivity to everolimus, to other rapamycin derivatives, or to any of the excipients.
* Patient who is unwilling to receive Afinitor treatment due to any reason.
* Pregnant or nursing (lactating) women,
* Prior therapy with mTOR inhibitors (e.g. sirolimus, temsirolimus, everolimus).
* Use of an investigational drug within the 30 days prior to enrollment

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period of 30 days, up to a maximum duration of approximately 5 years.
Secondary outcomes:
measure: Overall Survival (OS)
timeFrame: Up to approximately 7 years and 6 months.
measure: Progression Free Survival (PFS) by Investigator Assessment Per RECIST 1.1
timeFrame: Up to approximately 2 years and 9 months
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Novartis Investigative Site
state: Shanghai
city: Beijing
country: China
facility: Novartis Investigative Site
city: Beijing
country: China
facility: Novartis Investigative Site
city: Guangzhou
country: China
facility: Novartis Investigative Site
city: Shanghai
country: China
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: