Trial Outcomes & Findings for A Study of Pembrolizumab in Patients With Neuroendocrine Tumors (NCT NCT02939651)

Last Updated: 2021-03-09

Results Overview

To estimate objective response rate (ORR), using RECIST 1.1, in previously treated metastatic High Grade Neuroendocrine Tumors (HGNET) patients treated with pembrolizumab monotherapy. The Response Evaluation Criteria in Solid Tumors (RECIST 1.1) criteria will be used for objective tumor response assessment: Complete Response (CR):Disappearance of all target lesions. Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

Upto 3 years

Results posted on

2021-03-09

Participant Flow

Participant milestones

Participant milestones
Measure	Pembrolizumab Monotherapy with PD-1 antibody pembrolizumab Pembrolizumab: Pembrolizumab given intravenously at a fixed dose of 200mg every 3 weeks
Overall Study STARTED	21
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study of Pembrolizumab in Patients With Neuroendocrine Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pembrolizumab n=21 Participants Monotherapy with PD-1 antibody pembrolizumab Pembrolizumab: Pembrolizumab given intravenously at a fixed dose of 200mg every 3 weeks
Age, Continuous	56 years n=39 Participants
Sex: Female, Male Female	10 Participants n=39 Participants
Sex: Female, Male Male	11 Participants n=39 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=39 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	18 Participants n=39 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants
Region of Enrollment United States	21 participants n=39 Participants

PRIMARY outcome

Timeframe: Upto 3 years

Population: Analysis was completed using data from an external site which accrued 8 patients

Outcome measures

Outcome measures
Measure	Pembrolizumab n=21 Participants Monotherapy with PD-1 antibody pembrolizumab Pembrolizumab: Pembrolizumab given intravenously at a fixed dose of 200mg every 3 weeks
Objective Response Rate (ORR)	1 Participants

SECONDARY outcome

Timeframe: Upto 3 years

Population: Analysis was completed using data from an external site which accrued 8 patients

To evaluate progression free survival (PFS) (using RECIST 1.1) in metastatic HGNET patients treated with pembrolizumab monotherapy. PFS is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as a 20% increase (at least 5 mm absolute increase) in the sum of the longest diameter of target lesions. PFS was estimated according to the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	Pembrolizumab n=21 Participants Monotherapy with PD-1 antibody pembrolizumab Pembrolizumab: Pembrolizumab given intravenously at a fixed dose of 200mg every 3 weeks
Progression Free Survival (PFS)	8.86 weeks Interval 6.0 to 9.43

SECONDARY outcome

Timeframe: Upto 3 years

Population: Analysis was completed using data from an external site which accrued 8 patients. 95% confidence interval upper limit not estimated

To evaluate overall survival (OS) in metastatic HGNET patients treated with pembrolizumab monotherapy. OS was estimated according to the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	Pembrolizumab n=21 Participants Monotherapy with PD-1 antibody pembrolizumab Pembrolizumab: Pembrolizumab given intravenously at a fixed dose of 200mg every 3 weeks
Overall Survival	20.43 weeks Interval 12.86 to Upper band of median survival cannot be estimated

SECONDARY outcome

Timeframe: Upto 3 years

Population: Analysis was completed using data from an external site which accrued 8 patients

Occurence of toxicity, graded according to National Cancer Institute Common Toxicity Criteria (NCI-CTC) version 4.0.

Outcome measures

Outcome measures
Measure	Pembrolizumab n=21 Participants Monotherapy with PD-1 antibody pembrolizumab Pembrolizumab: Pembrolizumab given intravenously at a fixed dose of 200mg every 3 weeks
Frequency With Treatment-Related Adverse Events as Assessed by CTCAE v4.0	34 adverse events

Adverse Events

Pembrolizumab

Serious events: 5 serious events

Other events: 21 other events

Deaths: 15 deaths

Serious adverse events

Serious adverse events
Measure	Pembrolizumab n=21 participants at risk Monotherapy with PD-1 antibody pembrolizumab Pembrolizumab: Pembrolizumab given intravenously at a fixed dose of 200mg every 3 weeks
Renal and urinary disorders Acute kidney injury	4.8% 1/21 • 30 months
Infections and infestations Cholangitis	4.8% 1/21 • 30 months
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	4.8% 1/21 • 30 months
Respiratory, thoracic and mediastinal disorders Pleural effusion	4.8% 1/21 • 30 months
Musculoskeletal and connective tissue disorders Polymyaglia	4.8% 1/21 • 30 months
Investigations Elevated ALT	4.8% 1/21 • 30 months
Investigations Elevated ALK	4.8% 1/21 • 30 months
Gastrointestinal disorders Upper GI bleed	4.8% 1/21 • 30 months
General disorders Anemia	4.8% 1/21 • 30 months

Other adverse events

Other adverse events
Measure	Pembrolizumab n=21 participants at risk Monotherapy with PD-1 antibody pembrolizumab Pembrolizumab: Pembrolizumab given intravenously at a fixed dose of 200mg every 3 weeks
Blood and lymphatic system disorders Alkaline phosphatase increased	14.3% 3/21 • 30 months
Psychiatric disorders Anorexia	14.3% 3/21 • 30 months
Hepatobiliary disorders Aspartate aminotransferase increased	14.3% 3/21 • 30 months
Gastrointestinal disorders Bloating	4.8% 1/21 • 30 months
Gastrointestinal disorders Diarrhea	9.5% 2/21 • 30 months
General disorders Dry mouth	4.8% 1/21 • 30 months
Blood and lymphatic system disorders Edema limbs	9.5% 2/21 • 30 months
General disorders Fatigue	33.3% 7/21 • 30 months
Gastrointestinal disorders Gastrointestinal disorders	4.8% 1/21 • 30 months
General disorders General disorders and administration site conditions	9.5% 2/21 • 30 months
Endocrine disorders Hypercalcemia	4.8% 1/21 • 30 months
Endocrine disorders Hyperkalemia	4.8% 1/21 • 30 months
General disorders Nausea	9.5% 2/21 • 30 months
Skin and subcutaneous tissue disorders Pruritus	9.5% 2/21 • 30 months
Gastrointestinal disorders Rectal pain	4.8% 1/21 • 30 months
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	4.8% 1/21 • 30 months
General disorders Vomiting	4.8% 1/21 • 30 months

Additional Information

Namrata Vijayvergia

Fox Chase Cancer Center

Phone: +1 215-214-4283

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place