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)

NCT ID: NCT02955069

Last Updated: 2021-04-12

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

116 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-02-14

Study Completion Date

2020-05-13

Brief Summary

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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.

Detailed Description

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Two groups of adult patients with advanced (unresectable or metastatic) were included in this study:

* Well-differentiated (G1/2), non-functional, neuroendocrine tumor of GI, pancreatic or thoracic (lung/thymus) origin who have progressed on prior treatment
* Poorly-differentiated GEP-NEC who have progressed on or after one prior chemotherapy regimen.

The study was comprised of the following periods: screening, treatment, end of treatment (EOT), safety follow-up (30-Days, 60-Days, 90-Days, 120-Days, and 150-Days after the last dose of PDR001) and post-treatment efficacy follow-up. Subjects were treated with PDR001 as an infusion at a flat dose of 400 mg every 4 weeks (Q4W). Subjects were to continue study treatment beyond disease progression by RECIST 1.1 until disease progression as per irRECIST, as per BIRC, unacceptable toxicity, start of new antineoplastic therapy, withdrawal of consent, physician's decision, lost to follow-up, death, or study termination by the Sponsor.

Conditions

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Well-differentiated Non-functional NET of Thoracic Origin Well-differentiated Non-functional NET of Gastrointestinal Origin Well-differentiated Non-functional NET of Pancreatic Origin Poorly-differentiated Gastroenteropancreatic Neuroendocrine Carcinoma

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PDR001

Subjects 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.

Group Type EXPERIMENTAL

PDR001

Intervention Type DRUG

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.

Interventions

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PDR001

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.

Intervention Type DRUG

Other Intervention Names

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Spartalizumab

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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City of Hope National Medical Center

Duarte, California, United States

Site Status

Rocky Mountain Cancer Centers SC-2

Denver, Colorado, United States

Site Status

H Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, United States

Site Status

Mayo Clinic - Rochester

Rochester, Minnesota, United States

Site Status

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

Site Status

University of TX MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Novartis Investigative Site

St Leonards, New South Wales, Australia

Site Status

Novartis Investigative Site

Vienna, , Austria

Site Status

Novartis Investigative Site

Brussels, , Belgium

Site Status

Novartis Investigative Site

Leuven, , Belgium

Site Status

Novartis Investigative Site

Toronto, Ontario, Canada

Site Status

Novartis Investigative Site

Montreal, Quebec, Canada

Site Status

Novartis Investigative Site

Lyon, , France

Site Status

Novartis Investigative Site

Marseille, , France

Site Status

Novartis Investigative Site

Toulouse, , France

Site Status

Novartis Investigative Site

Erlangen, , Germany

Site Status

Novartis Investigative Site

Essen, , Germany

Site Status

Novartis Investigative Site

Mainz, , Germany

Site Status

Novartis Investigative Site

Marburg, , Germany

Site Status

Novartis Investigative Site

Bologna, BO, Italy

Site Status

Novartis Investigative Site

Meldola, FC, Italy

Site Status

Novartis Investigative Site

Milan, MI, Italy

Site Status

Novartis Investigative Site

Milan, MI, Italy

Site Status

Novartis Investigative Site

Roma, RM, Italy

Site Status

Novartis Investigative Site

Napoli, , Italy

Site Status

Novartis Investigative Site

Nagoya, Aichi-ken, Japan

Site Status

Novartis Investigative Site

Kashiwa, Chiba, Japan

Site Status

Novartis Investigative Site

Fukuoka, Fukuoka, Japan

Site Status

Novartis Investigative Site

Chuo Ku, Tokyo, Japan

Site Status

Novartis Investigative Site

Amsterdam, , Netherlands

Site Status

Novartis Investigative Site

L'Hospitalet de Llobregat, Catalonia, Spain

Site Status

Novartis Investigative Site

Madrid, , Spain

Site Status

Novartis Investigative Site

Madrid, , Spain

Site Status

Novartis Investigative Site

London, , United Kingdom

Site Status

Countries

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United States Australia Austria Belgium Canada France Germany Italy Japan Netherlands Spain United Kingdom

Provided Documents

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Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

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2016-002522-36

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CPDR001E2201

Identifier Type: -

Identifier Source: org_study_id

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