Study of Efficacy and Safety of PDR001 in Patients With Advanced or Metastatic, Well-differentiated, Non-functional Neuroendocrine Tumors of Pancreatic, Gastrointestinal (GI), or Thoracic Origin or Poorly-differentiated Gastroenteropancreatic Neuroendocrine Carcinoma (GEP-NEC)
An Open Label Phase II Study to Evaluate the Efficacy and Safety of PDR001 in Patients With Advanced or Metastatic, Well-differentiated, Non-functional Neuroendocrine Tumors of Pancreatic, Gastrointestinal (GI), or Thoracic Origin or Poorly-differentiated Gastroenteropancreatic Neuroendocrine Carcinoma (GEP-NEC), That Have Progressed on Prior Treatment.
2 other identifiers
interventional
116
11 countries
34
Brief Summary
This study aimed to investigate the efficacy and safety of PDR001 in patients with advanced or metastatic, well-differentiated, non-functional neuroendocrine tumors of pancreatic, gastrointestinal (GI), or thoracic origin or poorly-differentiated gastroenteropancreatic neuroendocrine carcinoma (GEP-NEC) that progressed on prior treatment.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Feb 2017
Typical duration for phase_2
34 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 2, 2016
CompletedFirst Posted
Study publicly available on registry
November 4, 2016
CompletedStudy Start
First participant enrolled
February 14, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 10, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
May 13, 2020
CompletedResults Posted
Study results publicly available
April 12, 2021
CompletedApril 12, 2021
March 1, 2021
1.5 years
November 2, 2016
March 16, 2021
March 16, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Overall Response Rate (ORR) by RECIST 1.1 and as Per Blinded Independent Central Review (BIRC).
ORR is defined as the proportion of patients with best overall response (BOR) of complete response (CR) or partial response (PR), according to BIRC radiological assessment by RECIST 1.1. CR: disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to \< 10 mm. PR: at least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters.
From baseline up to approximately 1.5 years
Secondary Outcomes (17)
Duration of Response (DOR) by RECIST 1.1 and as Per BIRC
From the date of first documented response (CR or PR) until the first documented disease progression or death, whichever comes first, assessed up to approximately 1.5 years
Disease Control Rate by RECIST 1.1 and as Per BIRC
From baseline up to approximately 1.5 years
Time to Response (TTR) by RECIST 1.1 and as Per BIRC
From baseline to the first documented response, assessed up to approximately 1.5 years
Progression-free Survival (PFS) by RECIST 1.1 and as Per BIRC
From baseline until the date of the first documented radiological progression or death due to any cause, whichever comes first, assessed up to approximately 1.5 years
Immune Related Overall Response Rate (irORR) by irRECIST and as Per BIRC
From baseline up to approximately 1.5 years
- +12 more secondary outcomes
Study Arms (1)
PDR001
EXPERIMENTALSubjects with advanced or metastatic, well-differentiated, NET of pancreatic, GI, or thoracic origin or poorly-differentiated GEP-NEC, that have progressed on prior treatment were treated with 400mg PDR001 administered via intravenous infusion once every 4 weeks.
Interventions
PDR001 was administered at a dose of 400 mg via intravenous infusion once every 4 weeks (Q4W). PDR001 was administered on Day 1 of every cycle. Each cycle was 28 days.
Eligibility Criteria
You may qualify if:
- Pathologically confirmed, advanced (unresectable or metastatic):
- Well-differentiated (G1 or G2) based on local pathology report, non-functional neuroendocrine tumor of GI, pancreatic or thoracic (including lung and thymus) origin.
- Poorly-differentiated GEP-NEC based on local pathology report
- No active symptoms related to carcinoid syndrome during the last 3 months prior to start of study treatment.
- Patients must have been pretreated for advanced disease - the number of prior systemic therapy/regimen depended on which origin for NET and for GEP-NEC
- Tumor biopsy material must be provided for all patients for the purpose of biomarker analysis
- Radiological documentation of disease progression:
- Well-differentiated NET group: Disease progression while on/or after the last treatment, and this progression must have been observed within 6 months prior to start of study treatment (i.e. maximum of 24 weeks from documentation of progression until study entry). Disease must show evidence of radiological disease progression based on scans performed not more than 12 months apart.
- Poorly-differentiated GEP-NEC group: Disease progression while on or after prior treatment.
You may not qualify if:
- Well-differentiated grade 3 neuroendocrine tumors; poorly-differentiated neuroendocrine carcinoma of any origin (other than GEP-NEC); including NEC of unknown origin, adenocarcinoid, and goblet cell carcinoid
- Pretreatment with interferon as last treatment prior to start of study treatment.
- Prior treatment for study indication with:
- Antibodies or immunotherapy within 6 weeks before the first dose of study treatment.
- Peptide Radionuclide Receptor Therapy (PRRT) administered within 6 months of the first dose.
- Systemic antineoplastic therapy
- Tyrosine kinase inhibitors within 14 days or 5 half-lives, whichever is longer, before the first dose of study treatment.
- Prior Programmed Death-1 (PD-1) or Programmed Death-Ligand 1 (PD-L1) directed therapy.
- Cryoablation, radiofrequency ablation, or trans-arterial embolization of hepatic metastases
- History of severe hypersensitivity reactions to other monoclonal antibodies which in the opinion of the investigator may pose an increased risk of a serious infusion reaction.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (35)
City of Hope National Medical Center
Duarte, California, 91010, United States
Rocky Mountain Cancer Centers SC-2
Denver, Colorado, 80218, United States
H Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612, United States
Mayo Clinic - Rochester
Rochester, Minnesota, 55905, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
Montefiore Medical Center
The Bronx, New York, 10461, United States
University of TX MD Anderson Cancer Center
Houston, Texas, 77030, United States
Novartis Investigative Site
St Leonards, New South Wales, 2065, Australia
Novartis Investigative Site
Vienna, A-1090, Austria
Novartis Investigative Site
Brussels, BE-B-1200, Belgium
Novartis Investigative Site
Leuven, 3000, Belgium
Novartis Investigative Site
Toronto, Ontario, M4N 3M5, Canada
Novartis Investigative Site
Montreal, Quebec, H3T 1E2, Canada
Novartis Investigative Site
Lyon, 69437, France
Novartis Investigative Site
Marseille, 13273, France
Novartis Investigative Site
Toulouse, 31054, France
Novartis Investigative Site
Erlangen, 91054, Germany
Novartis Investigative Site
Essen, 45147, Germany
Novartis Investigative Site
Mainz, 55131, Germany
Novartis Investigative Site
Marburg, 35039, Germany
Novartis Investigative Site
Bologna, BO, 40138, Italy
Novartis Investigative Site
Meldola, FC, 47014, Italy
Novartis Investigative Site
Milan, MI, 20133, Italy
Novartis Investigative Site
Milan, MI, 20141, Italy
Novartis Investigative Site
Roma, RM, 00189, Italy
Novartis Investigative Site
Napoli, 80131, Italy
Novartis Investigative Site
Nagoya, Aichi-ken, 464 8681, Japan
Novartis Investigative Site
Kashiwa, Chiba, 277 8577, Japan
Novartis Investigative Site
Fukuoka, Fukuoka, 812-8582, Japan
Novartis Investigative Site
Chuo Ku, Tokyo, 104 0045, Japan
Novartis Investigative Site
Amsterdam, 1066 CX, Netherlands
Novartis Investigative Site
L'Hospitalet de Llobregat, Catalonia, 08907, Spain
Novartis Investigative Site
Madrid, 28034, Spain
Novartis Investigative Site
Madrid, 28041, Spain
Novartis Investigative Site
London, NW3 2PR, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study director
- Organization
- Novartis Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 2, 2016
First Posted
November 4, 2016
Study Start
February 14, 2017
Primary Completion
August 10, 2018
Study Completion
May 13, 2020
Last Updated
April 12, 2021
Results First Posted
April 12, 2021
Record last verified: 2021-03
Data Sharing
- IPD Sharing
- Will share
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.