Pancreatic Cancer Research

Clinical trialPhase 2Completed

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.

NCT02955069↗ ClinicalTrials.gov, retrieved 6 November 2026

Outcome
Outcome unknown
Phase
Phase 2
Status
Completed
Study type
Interventional
Start
14 February 2017
Expected end
13 May 2020
Enrollment
116 participants
Sites
35
Primary outcomes
Overall Response Rate (ORR) by RECIST 1.1 and as Per Blinded Independent Central Review (BIRC).
Sponsor
Industry
Results posted
2021-04-12
Last update
12 April 2021

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Brief summary public domain (ClinicalTrials.gov)

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 public domain (ClinicalTrials.gov)

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.

Posted results (ClinicalTrials.gov registry) · primary outcome public domain (ClinicalTrials.gov)

Overall Response Rate (ORR) by RECIST 1.1 and as Per Blinded Independent Central Review (BIRC). [From baseline up to approximately 1.5 years]. Number, Percentage of participants (95% Confidence Interval). Well-differentiated NET (n=95): 7.4 (3.0 to 14.6); Poorly-differentiated GEP-NEC (n=21): 4.8 (0.1 to 23.8).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

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]. Median, Days (Full Range). Well-differentiated NET (n=7): 250 (49 to 288); Poorly Differentiated GEP-NEC (n=1): 270 (270 to 270).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Disease Control Rate by RECIST 1.1 and as Per BIRC [From baseline up to approximately 1.5 years]. Number, Percentage of participants (95% Confidence Interval). Well-differentiated NET (n=95): 64.2 (53.7 to 73.8); Poorly-differentiated GEP-NEC (n=21): 19.0 (5.4 to 41.9).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

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]. Median, Days (Full Range). Well-differentiated NET (n=7): 110 (52 to 218); Poorly-differentiated GEP-NEC (n=1): 53 (53 to 53).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

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]. Median, Months (95% Confidence Interval). Well-differentiated NET (n=95): 3.8 (3.6 to 5.5); Poorly-differentiated GEP-NEC (n=21): 1.8 (1.5 to 2.0).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Immune Related Overall Response Rate (irORR) by irRECIST and as Per BIRC [From baseline up to approximately 1.5 years]. Number, Percentage of participants (95% Confidence Interval). Well-differentiated NET (n=95): 7.4 (3.0 to 14.6); Poorly-differentiated GEP-NEC (n=21): 4.8 (0.1 to 23.8).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Immune Related Duration of Response (irDoR) by irRECIST and as Per BIRC. [From the date of first documented confirmed response (irCR or irPR) until the first documented progression, assessed up to approximately 1.5 years]. Median, Days (Full Range). Well-differentiated NET (n=7): 228 (49 to 288); Poorly-differentiated GEP-NEC (n=1): 270 (270 to 270).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Immune Related Time to Response (irTTR) by irRECIST and as Per BIRC [From baseline to the first documented response, assessed up to approximately 1.5 years]. Median, Days (Full Range). Well-differentiated NET (n=7): 110 (52 to 218); Poorly-differentiated GEP-NEC (n=1): 53 (53 to 53).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Immune Related Disease Control Rate (irDCR) by irRECIST and as Per BIRC [From baseline up to approximately 1.5 years]. Number, Percentage of participants (95% Confidence Interval). Well-differentiated NET (n=95): 66.3 (55.9 to 75.7); Poorly-differentiated GEP-NEC (n=21): 19.0 (5.4 to 41.9).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Immune Related Progression Free Survival (irPFS) by irRECIST and as Per BIRC [From baseline until the date of the first documented immune related progression or death due to any cause, whichever comes first, assessed up to approximately 1.5 years]. Median, Months (95% Confidence Interval). Well-differentiated NET (n=95): 3.8 (3.6 to 5.5); Poorly-differentiated GEP-NEC (n=21): 1.8 (1.5 to 2.0).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Overall Survival (OS) [From baseline until death due to any cause, assessed up to approx. 3 years]. Median, Months (95% Confidence Interval). Well-differentiated NET (n=95): 23.4 (19.2 to NA) [NA: Upper limit was not evaluable]; Poorly-differentiated GEP-NEC (n=21): 6.8 (4.0 to 8.6).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Changes From Baseline in Chromogranin A (CgA) Levels [Baseline, day 1 of each cycle from Cycle 2 to end of treatment, assessed up to approx. 1.5 years. Cycle=28 days.]. Mean, microgram/liter (ug/L) (Standard Deviation). Cycle 2 Day 1: Well-differentiated NET (n=95): 874.6 (6694.64); Poorly-differentiated GEP-NEC (n=21): 6466.9 (23760.13). Cycle 3 Day 1: Well-differentiated NET (n=95): -1811.7 (21413.11); Poorly-differentiated GEP-NEC (n=21): 706.5 (2294.87). Cycle 4 Day 1: Well-differentiated NET (n=95): 579.9 (13437.06); Poorly-differentiated GEP-NEC (n=21): 1544.5 (2712.23). Cycle 5 Day 1: Well-differentiated NET (n=95): 598.2 (11724.93); Poorly-differentiated GEP-NEC (n=21): 1306.6 (3125.84). Cycle 6 Day 1: Well-differentiated NET (n=95): 1337.6 (7106.32); Poorly-differentiated GEP-NEC (n=21): 4531.7 (8694.65). Cycle 7 Day 1: Well-differentiated NET (n=95): -564.5 (12643.71); Poorly-differentiated GEP-NEC (n=21): 11089.5 (17551.10). Cycle 8 Day 1: Well-differentiated NET (n=95): 3726.0 (18813.63); Poorly-differentiated GEP-NEC (n=21): 9103.5 (14759.44). Cycle 9 Day 1: Well-differentiated NET (n=95): 22522.2 (94054.85); Poorly-differentiated GEP-NEC (n=21): -1342.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 10 Day 1: Well-differentiated NET (n=95): 54301.5 (250229.27); Poorly-differentiated GEP-NEC (n=21): -1376.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 11 Day 1: Well-differentiated NET (n=95): 84634.7 (344244.60); Poorly-differentiated GEP-NEC (n=21): -1393.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 12 Day 1: Well-differentiated NET (n=95): 8177.6 (36820.47); Poorly-differentiated GEP-NEC (n=21): -1362.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 13 Day 1: Well-differentiated NET (n=95): -379.8 (1509.95); Poorly-differentiated GEP-NEC (n=21): -1377.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 14 Day 1: Well-differentiated NET (n=95): 398.3 (1866.56). Cycle 15 Day 1: Well-differentiated NET (n=95): 13.1 (1120.60). Cycle 16 Day 1: Well-differentiated NET (n=95): -81.0 (1583.99). Cycle 17 Day 1: Well-differentiated NET (n=95): -176.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 18 Day 1: Well-differentiated NET (n=95): 42.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. End of treatment: Well-differentiated NET (n=95): 30115.3 (131958.16); Poorly-differentiated GEP-NEC (n=21): 3899.6 (7991.40).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Change From Baseline in Neuron Specific Enolase (NSE) Levels [Baseline, day 1 of each cycle from Cycle 2 to end of treatment, assessed up to approx. 1.5 years. Cycle= 28days]. Mean, microgram/liter (ug/L) (Standard Deviation). Cycle 2 Day 1: Well-differentiated NET (n=95): 0.8 (29.71); Poorly-differentiated GEP-NEC (n=21): 27.9 (40.77). Cycle 3 Day 1: Well-differentiated NET (n=95): 2.5 (29.33); Poorly-differentiated GEP-NEC (n=21): 32.6 (56.87). Cycle 4 Day 1: Well-differentiated NET (n=95): -0.1 (14.98); Poorly-differentiated GEP-NEC (n=21): 21.9 (21.64). Cycle 5 Day 1: Well-differentiated NET (n=95): 4.8 (30.34); Poorly-differentiated GEP-NEC (n=21): 31.8 (56.39). Cycle 6 Day 1: Well-differentiated NET (n=95): -3.6 (19.18); Poorly-differentiated GEP-NEC (n=21): 151.6 (204.21). Cycle 7 Day 1: Well-differentiated NET (n=95): 7.6 (39.44); Poorly-differentiated GEP-NEC (n=21): -3.0 (17.75). Cycle 8 Day 1: Well-differentiated NET (n=95): 1.0 (22.19); Poorly-differentiated GEP-NEC (n=21): 40.4 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 9 Day 1: Well-differentiated NET (n=95): 2.6 (8.64); Poorly-differentiated GEP-NEC (n=21): -12.7 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 10 Day 1: Well-differentiated NET (n=95): 10.3 (33.74); Poorly-differentiated GEP-NEC (n=21): -15.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 11 Day 1: Well-differentiated NET (n=95): 2.3 (8.89). Cycle 12 Day 1: Well-differentiated NET (n=95): -2.4 (23.04); Poorly-differentiated GEP-NEC (n=21): -12.3 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 13 Day 1: Well-differentiated NET (n=95): -3.9 (24.22); Poorly-differentiated GEP-NEC (n=21): -17.8 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 14 Day 1: Well-differentiated NET (n=95): -4.5 (22.71). Cycle 15 Day 1: Well-differentiated NET (n=95): -3.4 (29.73). Cycle 16 Day 1: Well-differentiated NET (n=95): 26.5 (35.38). Cycle 17 Day 1: Well-differentiated NET (n=95): 15.1 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 18 Day 1: Well-differentiated NET (n=95): 13.8 (NA) [NA: Not evaluable due to low number of participants analyzed]. End of treatment: Well-differentiated NET (n=95): 47.7 (136.78); Poorly-differentiated GEP-NEC (n=21): 44.3 (66.73).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

PDR001 Plasma Concentration [Cycle(C)1 Day(D)1 pre-dose and 30min post-infusion,C2D1 Pre-dose,C3D1 Pre-dose and 30min post-infusion,D1 pre-dose from C4 to C13, assessed up to approx. 1.5 years.Cycle=28 days]. Mean, nanogram/mililiter (ng/mL) (Standard Deviation). Cycle 1 Day 1 pre-dose: Well-differentiated NET (n=94): 0 (0); Poorly-differentiated GEP-NEC (n=18): 0 (0). Cycle 1 Day 1 30 min post-dose: Well-differentiated NET (n=94): 106 (32.9); Poorly-differentiated GEP-NEC (n=18): 100 (52.2). Cycle 2 Day 1 Pre-dose: Well-differentiated NET (n=94): 26.8 (9.76); Poorly-differentiated GEP-NEC (n=18): 25.8 (9.30). Cycle 3 Day 1 pre-dose: Well-differentiated NET (n=94): 43.7 (18.9); Poorly-differentiated GEP-NEC (n=18): 42.3 (19.6). Cycle 3 Day 1 30 min post-dose: Well-differentiated NET (n=94): 140 (46.7); Poorly-differentiated GEP-NEC (n=18): 142 (44.3). Cycle 4 Day 1 pre-dose: Well-differentiated NET (n=94): 52.8 (25.1); Poorly-differentiated GEP-NEC (n=18): 40.7 (17.2). Cycle 5 Day 1 pre-dose: Well-differentiated NET (n=94): 52.0 (32.3); Poorly-differentiated GEP-NEC (n=18): 41.8 (8.88). Cycle 6 Day 1 pre-dose: Well-differentiated NET (n=94): 58.2 (31.6); Poorly-differentiated GEP-NEC (n=18): 49.9 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 7 Day 1 pre-dose: Well-differentiated NET (n=94): 64.3 (37.6); Poorly-differentiated GEP-NEC (n=18): 66.9 (30.1). Cycle 8 Day 1 pre-dose: Well-differentiated NET (n=94): 59.6 (38.2); Poorly-differentiated GEP-NEC (n=18): 64.7 (31.4). Cycle 9 Day 1 pre-dose: Well-differentiated NET (n=94): 65.1 (38.1); Poorly-differentiated GEP-NEC (n=18): 93.7 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 10 Day 1 pre-dose: Well-differentiated NET (n=94): 70.2 (40.2). Cycle 11 Day 1 pre-dose: Well-differentiated NET (n=94): 69.2 (39.1); Poorly-differentiated GEP-NEC (n=18): 99.0 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 12 Day 1 pre-dose: Well-differentiated NET (n=94): 71.7 (38.1); Poorly-differentiated GEP-NEC (n=18): 111 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 13 Day 1 pre-dose: Well-differentiated NET (n=94): 91.8 (46.0).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) Global Health Status/Quality of Life Score [Baseline, every 8 weeks from Cycle 3 Day 1 for the first 13 cycles and every 12 weeks from Cycle 13 Day 1 thereafter and end of treatment, assessed up to approx. 1.5 years. Cycle=28 days]. Mean, score on a scale (Standard Deviation). Cycle 3 Day 1: Well-differentiated NET (n=95): -1.00 (19.491); Poorly-differentiated GEP-NEC (n=21): -11.11 (27.003). Cycle 5 Day 1: Well-differentiated NET (n=95): -1.91 (27.838); Poorly-differentiated GEP-NEC (n=21): -4.17 (17.347). Cycle 7 Day 1: Well-differentiated NET (n=95): 1.96 (29.945); Poorly-differentiated GEP-NEC (n=21): 25.00 (35.355). Cycle 9 Day 1: Well-differentiated NET (n=95): 1.67 (31.823); Poorly-differentiated GEP-NEC (n=21): 33.33 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 11 Day 1: Well-differentiated NET (n=95): -1.19 (28.048). Cycle 13 Day 1: Well-differentiated NET (n=95): 1.96 (27.088); Poorly-differentiated GEP-NEC (n=21): 41.67 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 16 Day 1: Well-differentiated NET (n=95): -16.67 (16.667). End of Treatment: Well-differentiated NET (n=95): -6.46 (23.607); Poorly-differentiated GEP-NEC (n=21): -22.92 (23.465).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

Change From Baseline in EQ-5D-5L Index Score [Baseline, every 8 weeks from Cycle 3 Day 1 for the first 13 cycles and every 12 weeks from Cycle 13 Day 1 thereafter, and end of treatment, assessed up to approx.1.5 year. Cycle=28 days]. Mean, score on a scale (Standard Deviation). Cycle 3 Day 1: Well-differentiated NET (n=95): 0.03 (0.169); Poorly-differentiated GEP-NEC (n=21): -0.09 (0.157). Cycle 5 Day 1: Well-differentiated NET (n=95): -0.02 (0.247); Poorly-differentiated GEP-NEC (n=21): 0.02 (0.111). Cycle 7 Day 1: Well-differentiated NET (n=95): -0.04 (0.241); Poorly-differentiated GEP-NEC (n=21): 0.16 (0.135). Cycle 9 Day 1: Well-differentiated NET (n=95): 0.02 (0.247); Poorly-differentiated GEP-NEC (n=21): 0.02 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 11 Day 1: Well-differentiated NET (n=95): 0.02 (0.262). Cycle 13 Day 1: Well-differentiated NET (n=95): -0.03 (0.299); Poorly-differentiated GEP-NEC (n=21): 0.03 (NA) [NA: Not evaluable due to low number of participants analyzed]. Cycle 16 Day 1: Well-differentiated NET (n=95): -0.17 (0.085). End of treatment: Well-differentiated NET (n=95): -0.10 (0.209); Poorly-differentiated GEP-NEC (n=21): -0.30 (0.263).

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

PDR001 Anti-drug Antibodies (ADA) Prevalence at Baseline [Baseline]. Count Of Participants, Participants. Well-differentiated NET (n=88): 1; Poorly-differentiated GEP-NEC (n=17): 0.

Posted results (ClinicalTrials.gov registry) · secondary outcome public domain (ClinicalTrials.gov)

PDR001 ADA Incidence On-treatment [Cycle(C)1 Day(D)1 pre-dose and 30min post-infusion,C2D1 Pre-dose,C3D1 Pre-dose and 30min post-infusion,D1 pre-dose from C4 to C13 and every 6 cycles until C25, and end of treatment, assessed up to approx. 1.5 years. Cycle=28 days]. Count Of Participants, Participants. Well-differentiated NET (n=84): 10; Poorly-differentiated GEP-NEC (n=17): 2.