Trial · NCT02955069

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)

NCT02955069, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2021-04-12

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. 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). Trials, record NCT02955069. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02955069

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: 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 number: NCT02955069
Status: Completed
Phases: Phase 2
Enrollment: 116 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: 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
Interventions: PDR001
First posted: 2016-11-04
Last updated: 2021-04-12
Other registries' IDs: eudract:2016-002522-36
Results first posted: 2021-04-12

Source ClinicalTrials.gov

Registered outcomes18

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Overall Response Rate (ORR) by RECIST 1.1 and as Per Blinded Independent Central Review (BIRC).	From baseline up to approximately 1.5 years	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.
Secondary	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	DOR is defined as the time between the date of first documented response (CR or PR) and the date of first documented disease progression by RECIST 1.1 and as per BIRC or death due to underlying cancer. For DOR analysis, participants continuing without progression or death due to underlying cancer were censored at the date of their last adequate tumor assessment. An adequate tumour assessment is a tumour assessment with an overall response other than unknown. 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.
Secondary	Disease Control Rate by RECIST 1.1 and as Per BIRC	From baseline up to approximately 1.5 years	Disease control rate is defined as the proportion of patients with best overall response of CR, PR or stable disease (SD) according to RECIST 1.1 criteria and as per BIRC. 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. SD: neither sufficient shrinkage to qualify for PR or CR nor an increase in lesions which would qualify for progressive disease.
Secondary	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	TTR is defined as the time from the date of start of treatment to the first documented response of either CR or PR, which must be subsequently confirmed. TTR was evaluated according to RECIST 1.1 and as per BIRC. 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.
Secondary	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	PFS 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. PFS was evaluated according to RECIST 1.1 and as per BIRC. For participants who had not progressed or died at the analysis cut-off date, PFS was censored at the date of the last adequate tumor evaluation date. An adequate tumour assessment is a tumour assessment with an overall response other than unknown.
Secondary	Immune Related Overall Response Rate (irORR) by irRECIST and as Per BIRC	From baseline up to approximately 1.5 years	irORR is the proportion of patients with a best overall response of immune related Complete Response (irCR) or immune related partial response (irPR), according to BIRC assessment by irRECIST. irCR: Complete disappearance of all measurable and non-measurable lesions. In addition, any pathological lymph nodes must have a reduction in short axis to \< 10 mm. irPR: At least 30% decrease in the total measurable tumor burden (TMTB) compared to baseline and not qualifying for irCR or immune related progressive disease (irPD).
Secondary	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	irDOR is defined as the time from first documentation of irCR or irPR until the time of first documentation of progression per irRECIST based on BIRC assessment. Participants continuing without progression or death due to underlying cancer were censored at the date of their last adequate tumor assessment. An adequate tumour assessment is a tumour assessment with an overall response other than unknown for irRECIST. irCR: Complete disappearance of all measurable and non-measurable lesions. In addition, any pathological lymph nodes must have a reduction in short axis to \< 10 mm. irPR: At least 30% decrease in the TMTB compared to baseline and not qualifying for irPD or irCR
Secondary	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	irTTR is defined as the time between date of start of treatment until first documented response (confirmed irCR or irPR) by irRECIST and as per BIRC. irCR: Complete disappearance of all measurable and non-measurable lesions. In addition, any pathological lymph nodes must have a reduction in short axis to \< 10 mm. irPR: At least 30% decrease in the TMTB compared to baseline and not qualifying for irPD or irCR.
Secondary	Immune Related Disease Control Rate (irDCR) by irRECIST and as Per BIRC	From baseline up to approximately 1.5 years	irDCR is defined as the proportion of patients with a best overall response of irCR or irPR or immune related stable disease (irSD) according to irRECIST and as per BIRC. irCR: Complete disappearance of all measurable and non-measurable lesions. In addition, any pathological lymph nodes must have a reduction in short axis to \< 10 mm. irPR: At least 30% decrease in the TMTB compared to baseline and not qualifying for irPD or irCR. irSD: Neither a sufficient shrinkage to qualify for irPR or irCR, nor an increase in lesions, or a clear and unequivocal progression of existing nontarget or new non-measurable lesions that would qualify for irPD.
Secondary	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	irPFS is defined as the time from date of start of treatment to the date of event defined as the first documented assessment of immune related progression that is confirmed or death due to any cause. If a patient has not had an event, immune related progression-free survival was censored at the date of last adequate tumor assessment. An adequate tumor assessment is a tumor assessment with overall response other than unknown for irRECIST.

1 to 10 of 18Source ClinicalTrials.gov

See all 18 registered outcomes

Outcomes19

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Overall Response Rate (ORR) by RECIST 1.1 and as Per Blinded Independent Central Review (BIRC).	From baseline up to approximately 1.5 years	The FAS comprised of all subjects to whom study treatment had been assigned and who received at least one dose of PDR001 (including incomplete infusion).	Percentage of participants	NUMBER
SECONDARY	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	Participants in the FAS with confirmed CR or PR	Days	MEDIAN
SECONDARY	Disease Control Rate by RECIST 1.1 and as Per BIRC	From baseline up to approximately 1.5 years	The FAS comprised of all subjects to whom study treatment had been assigned and who received at least one dose of PDR001 (including incomplete infusion).	Percentage of participants	NUMBER
SECONDARY	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	Participants in the FAS with confirmed CR or PR.	Days	MEDIAN
SECONDARY	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	The FAS comprised of all subjects to whom study treatment had been assigned and who received at least one dose of PDR001 (including incomplete infusion).	Months	MEDIAN
SECONDARY	Immune Related Overall Response Rate (irORR) by irRECIST and as Per BIRC	From baseline up to approximately 1.5 years	The FAS comprised of all subjects to whom study treatment had been assigned and who received at least one dose of PDR001 (including incomplete infusion).	Percentage of participants	NUMBER
SECONDARY	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	Participants in the FAS with confirmed irCR or irPR	Days	MEDIAN
SECONDARY	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	Participants in the FAS with confirmed irCR or irPR	Days	MEDIAN
SECONDARY	Immune Related Disease Control Rate (irDCR) by irRECIST and as Per BIRC	From baseline up to approximately 1.5 years	The FAS comprised of all subjects to whom study treatment had been assigned and who received at least one dose of PDR001 (including incomplete infusion).	Percentage of participants	NUMBER
SECONDARY	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	The FAS comprised of all subjects to whom study treatment had been assigned and who received at least one dose of PDR001 (including incomplete infusion).	Months	MEDIAN

1 to 10 of 19Source ClinicalTrials.gov

See all 19 outcomes

Results by group147

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Overall Response Rate (ORR) by RECIST 1.1 and as Per Blinded Independent Central Review (BIRC).	Well-differentiated NET	7.4	95% Confidence Interval 3.0 to 14.6	Percentage of participants
Overall Response Rate (ORR) by RECIST 1.1 and as Per Blinded Independent Central Review (BIRC).	Poorly-differentiated GEP-NEC	4.8	95% Confidence Interval 0.1 to 23.8	Percentage of participants
Duration of Response (DOR) by RECIST 1.1 and as Per BIRC	Well-differentiated NET	250	Full Range 49 to 288	Days
Duration of Response (DOR) by RECIST 1.1 and as Per BIRC	Poorly Differentiated GEP-NEC	270	Full Range 270 to 270	Days
Disease Control Rate by RECIST 1.1 and as Per BIRC	Well-differentiated NET	64.2	95% Confidence Interval 53.7 to 73.8	Percentage of participants
Disease Control Rate by RECIST 1.1 and as Per BIRC	Poorly-differentiated GEP-NEC	19.0	95% Confidence Interval 5.4 to 41.9	Percentage of participants
Time to Response (TTR) by RECIST 1.1 and as Per BIRC	Well-differentiated NET	110	Full Range 52 to 218	Days
Time to Response (TTR) by RECIST 1.1 and as Per BIRC	Poorly-differentiated GEP-NEC	53	Full Range 53 to 53	Days
Progression-free Survival (PFS) by RECIST 1.1 and as Per BIRC	Well-differentiated NET	3.8	95% Confidence Interval 3.6 to 5.5	Months
Progression-free Survival (PFS) by RECIST 1.1 and as Per BIRC	Poorly-differentiated GEP-NEC	1.8	95% Confidence Interval 1.5 to 2.0	Months

1 to 10 of 147Source ClinicalTrials.gov

See all 147 results by group

Participant flow16

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Well-differentiated NET	95
Overall Study	STARTED	Poorly-differentiated GEP-NEC	21
Overall Study	Full Analysis Set (FAS)	Well-differentiated NET	95
Overall Study	Full Analysis Set (FAS)	Poorly-differentiated GEP-NEC	21
Overall Study	Safety Set	Well-differentiated NET	95
Overall Study	Safety Set	Poorly-differentiated GEP-NEC	21
Overall Study	Pharmacokinetic Analysis Set (PAS)	Well-differentiated NET	94
Overall Study	Pharmacokinetic Analysis Set (PAS)	Poorly-differentiated GEP-NEC	18
Overall Study	Immunogenicity (IG) Prevalence Set	Well-differentiated NET	94
Overall Study	Immunogenicity (IG) Prevalence Set	Poorly-differentiated GEP-NEC	21

1 to 10 of 16Source ClinicalTrials.gov

See all 16 participant flow

Adverse events276

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	Well-differentiated NET	18	95
other	Anaemia	Blood and lymphatic system disorders	Poorly-differentiated GEP-NEC	3	21
other	Anaemia	Blood and lymphatic system disorders	All Subjects	21	116
other	Hypothyroidism	Endocrine disorders	Well-differentiated NET	7	95
other	Hypothyroidism	Endocrine disorders	Poorly-differentiated GEP-NEC	0	21
other	Hypothyroidism	Endocrine disorders	All Subjects	7	116
other	Abdominal distension	Gastrointestinal disorders	Well-differentiated NET	5	95
other	Abdominal distension	Gastrointestinal disorders	Poorly-differentiated GEP-NEC	2	21
other	Abdominal distension	Gastrointestinal disorders	All Subjects	7	116
other	Abdominal pain	Gastrointestinal disorders	Well-differentiated NET	17	95

1 to 10 of 276Source ClinicalTrials.gov

See all 276 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Well-differentiated NET	1	95
Total, all-cause mortality	Poorly-differentiated GEP-NEC	1	21
Total, all-cause mortality	All Subjects	2	116
Total, other adverse events	Well-differentiated NET	86	95
Total, other adverse events	Poorly-differentiated GEP-NEC	18	21
Total, other adverse events	All Subjects	104	116
Total, serious adverse events	Well-differentiated NET	29	95
Total, serious adverse events	Poorly-differentiated GEP-NEC	6	21
Total, serious adverse events	All Subjects	35	116

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Well-differentiated NET vs Poorly-differentiated GEP-NEC: PDR001 Anti-drug Antibodies (ADA) Prevalence at Baseline	Trial comparisons
Well-differentiated NET vs Poorly-differentiated GEP-NEC: PDR001 ADA Incidence On-treatment	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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