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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)

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ClinicalTrials.gov Identifier: NCT02955069
Recruitment Status : Active, not recruiting
First Posted : November 4, 2016
Last Update Posted : December 7, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
This is a study 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 have progressed on prior treatment.

Condition or disease Intervention/treatment Phase
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 Drug: PDR001 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 116 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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 EUDRACT 2016-002522-36
Actual Study Start Date : February 14, 2017
Actual Primary Completion Date : August 10, 2018
Estimated Study Completion Date : April 3, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: PDR001
110 patients will be administered PDR001 infusion.
Drug: PDR001
PDR001 is a high-affinity, ligand-blocking, humanized IgG4 antibody directed against Programmed Death-1 (PD-1) receptor that blocks the binding of PD-L1 and PD-L2. PDR001 dose is 400 mg infusion every 4 weeks.




Primary Outcome Measures :
  1. Overall response rate [ Time Frame: 1 year after last patient first treatment ]
    overall response rate is defined as the proportion of patients with best overall response (BOR) of complete response (CR) or partial response (PR), as per RECIST 1.1 and blinded independent central review.


Secondary Outcome Measures :
  1. Duration of response [ Time Frame: baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Duration of Response by RECIST 1.1 and as per BIRC

  2. Disease Control [ Time Frame: Baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Disease Control is defined as the proportion of patients with best overall response of CR, PR or SD according to RECIST 1.1 criteria and as per central review

  3. Time to response [ Time Frame: Baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Time to response (TTR) is defined as the time from the date of start of treatment to the first documented response of either complete response (CR) or partial response (PR), which must be subsequently confirmed (although date of initial response is used, not date of confirmation). TTR will be evaluated according to RECIST 1.1

  4. Progression-free survival [ Time Frame: Baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Progression-free survival is defined as the time from the date of first dose to the date of the first documented radiological progression or death due to any cause

  5. Overall survival [ Time Frame: Every 3 months after last visit up to 2 year after last patient first treatment ]
    Overall survival is defined as the time from date of start of treatment to date of death due to any cause

  6. Immune Response Criteria by irRECIST(immune response overall response rate) [ Time Frame: Baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Immune Response Criteria by irRECIST and as per BIRC (immune response objective response rate)

  7. Immune Response Criteria by irRECIST (immune response duration of response) [ Time Frame: Baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Immune Response Criteria by irRECIST and as per BIRC (immune response duration of response)

  8. Immune Response Criteria by irRECIST (immune response time to response) [ Time Frame: Baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Immune Response Criteria by irRECIST and as per BIRC (immune response time to response)

  9. Immune Response Criteria by irRECIST (immune response disease control rate) [ Time Frame: Baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Immune Response Criteria by irRECIST and as per BIRC (immune response disease control rate)

  10. Patient's health-related quality of life [ Time Frame: Baseline, every 8 weeks up to 1 year after last patient first treatment ]
    Global health status/quality of life score of the EORTC QLQ-C30 and the index score of the EQ-5D-5L

  11. Biochemical response [ Time Frame: Baseline, every 28 days until treatment completion (2 yrs max) ]
    Changes from baseline in chromogranin-A and neuron-specific enolase



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 depends 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.
  • 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 PD-1- or 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02955069


  Hide Study Locations
Locations
United States, California
City of Hope National Medical Center
Duarte, California, United States, 91010
University of California San Francisco
San Francisco, California, United States, 94115
United States, Colorado
Rocky Mountain Cancer Centers SC-2
Denver, Colorado, United States, 80218
United States, Florida
H Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, United States, 33612
United States, Kentucky
University of Kentucky SC-2
Lexington, Kentucky, United States, 40536
United States, Massachusetts
Dana Farber Cancer Center
Boston, Massachusetts, United States, 02215
United States, Minnesota
Mayo Clinic - Rochester
Rochester, Minnesota, United States, 55905
United States, New York
Montefiore Medical Center
Bronx, New York, United States, 10461
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
United States, Oregon
OHSU Knight Cancer Institute
Portland, Oregon, United States, 97201
United States, Texas
University of TX MD Anderson Cancer Center
Houston, Texas, United States, 77030
Australia, New South Wales
Novartis Investigative Site
St Leonards, New South Wales, Australia, 2065
Australia, Victoria
Novartis Investigative Site
Melbourne, Victoria, Australia, 3000
Austria
Novartis Investigative Site
Wien, Austria, A-1090
Belgium
Novartis Investigative Site
Brussels, Belgium, BE-B-1200
Novartis Investigative Site
Leuven, Belgium, 3000
Canada, Ontario
Novartis Investigative Site
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Novartis Investigative Site
Montreal, Quebec, Canada, H3T 1E2
France
Novartis Investigative Site
Lyon, France, 69437
Novartis Investigative Site
Marseille, France, 13273
Novartis Investigative Site
Toulouse Cedex 4, France, 31054
Novartis Investigative Site
Villejuif Cedex, France, 94805
Germany
Novartis Investigative Site
Erlangen, Germany, 91054
Novartis Investigative Site
Essen, Germany, 45147
Novartis Investigative Site
Mainz, Germany, 55131
Novartis Investigative Site
Marburg, Germany, 35039
Novartis Investigative Site
Wuerzburg, Germany, 97080
Israel
Novartis Investigative Site
Haifa, Israel, 3525408
Novartis Investigative Site
Tel Aviv, Israel, 64239
Italy
Novartis Investigative Site
Bologna, BO, Italy, 40138
Novartis Investigative Site
Meldola, FC, Italy, 47014
Novartis Investigative Site
Milano, MI, Italy, 20133
Novartis Investigative Site
Milano, MI, Italy, 20141
Novartis Investigative Site
Roma, RM, Italy, 00189
Novartis Investigative Site
Napoli, Italy, 80131
Japan
Novartis Investigative Site
Nagoya, Aichi, Japan, 464-8681
Novartis Investigative Site
Kashiwa, Chiba, Japan, 277-8577
Novartis Investigative Site
Fukuoka city, Fukuoka, Japan, 812-8582
Novartis Investigative Site
Chuo-ku, Tokyo, Japan, 104-0045
Netherlands
Novartis Investigative Site
Amsterdam, Netherlands, 1066 CX
Spain
Novartis Investigative Site
Hospitalet de LLobregat, Catalunya, Spain, 08907
Novartis Investigative Site
Madrid, Spain, 28034
Novartis Investigative Site
Madrid, Spain, 28041
Sweden
Novartis Investigative Site
Uppsala, Sweden, SE-751 85
Switzerland
Novartis Investigative Site
Basel, Switzerland, 4031
Novartis Investigative Site
Bern, Switzerland, 3010
United Kingdom
Novartis Investigative Site
London, United Kingdom, NW3 2PR
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02955069     History of Changes
Other Study ID Numbers: CPDR001E2201
2016-002522-36 ( EudraCT Number )
First Posted: November 4, 2016    Key Record Dates
Last Update Posted: December 7, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description:

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.


Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Advanced or metastatic
NET
pNET
GI NET
thoracic NET
GEP-NEC

Additional relevant MeSH terms:
Carcinoma
Neuroendocrine Tumors
Carcinoma, Neuroendocrine
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Nerve Tissue
Adenocarcinoma