Trial Outcomes & Findings for Niraparib and Dostarlimab for the Treatment of Germline or Somatic BRCA1/2 and PALB2 Mutated Metastatic Pancreatic Cancer (NCT NCT04493060)

NCT ID: NCT04493060

Last Updated: 2025-08-07

Results Overview

Will be assessed using the standard immune-modified Response Evaluation Criteria in Solid Tumors (iRECIST) criteria.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

At 12 weeks

Results posted on

2025-08-07

Participant Flow

Participant milestones

Participant milestones
Measure
Treatment (Niraparib, Dostarlimab)
Patients receive niraparib PO QD on days 1-21. Patients also receive dostarlimab IV over 30 minutes on day 1 Q3W for cycles 1-4 and Q6W for subsequent cycles. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.\>\> \>\> Dostarlimab: Given IV\>\> \>\> Niraparib: Given PO
Overall Study
STARTED
22
Overall Study
COMPLETED
18
Overall Study
NOT COMPLETED
4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

One patient did not give information on mismatch repair status

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Treatment (Niraparib, Dostarlimab)
n=18 Participants
Patients receive niraparib PO QD on days 1-21. Patients also receive dostarlimab IV over 30 minutes on day 1 Q3W for cycles 1-4 and Q6W for subsequent cycles. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.\>\> \>\> Dostarlimab: Given IV\>\> \>\> Niraparib: Given PO
Age, Continuous
61.3 years
STANDARD_DEVIATION 8.49 • n=18 Participants
Sex: Female, Male
Female
11 Participants
n=18 Participants
Sex: Female, Male
Male
7 Participants
n=18 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=18 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
18 Participants
n=18 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=18 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=18 Participants
Race (NIH/OMB)
Asian
0 Participants
n=18 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=18 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=18 Participants
Race (NIH/OMB)
White
17 Participants
n=18 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=18 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=18 Participants
Number of Prior Treatment(s)
1
3 Participants
n=18 Participants
Number of Prior Treatment(s)
2
7 Participants
n=18 Participants
Number of Prior Treatment(s)
3+
8 Participants
n=18 Participants
Mismatch Repair Status
Deficient
2 Participants
n=17 Participants • One patient did not give information on mismatch repair status
Mismatch Repair Status
Proficient
15 Participants
n=17 Participants • One patient did not give information on mismatch repair status
Deleterious Mutation
BRCA 1
5 Participants
n=18 Participants
Deleterious Mutation
BRCA 2
12 Participants
n=18 Participants
Deleterious Mutation
PALB2
1 Participants
n=18 Participants
Platinum Tx Exposure
Yes
16 Participants
n=18 Participants
Platinum Tx Exposure
No
2 Participants
n=18 Participants
Refractory To Cancer Tx
Yes
5 Participants
n=18 Participants
Refractory To Cancer Tx
No
13 Participants
n=18 Participants

PRIMARY outcome

Timeframe: At 12 weeks

Will be assessed using the standard immune-modified Response Evaluation Criteria in Solid Tumors (iRECIST) criteria.

Outcome measures

Outcome measures
Measure
Treatment (Niraparib, Dostarlimab)
n=18 Participants
Patients receive niraparib PO QD on days 1-21. Patients also receive dostarlimab IV over 30 minutes on day 1 Q3W for cycles 1-4 and Q6W for subsequent cycles. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.\>\> \>\> Dostarlimab: Given IV\>\> \>\> Niraparib: Given PO
Disease Control Rate at 12 Weeks (DCR12)
No Success
13 Participants
Disease Control Rate at 12 Weeks (DCR12)
Success
5 Participants

SECONDARY outcome

Timeframe: Up to 4 years and 5 months

The confirmed response rate (by iRECIST ) will be assessed.

Outcome measures

Outcome measures
Measure
Treatment (Niraparib, Dostarlimab)
n=18 Participants
Patients receive niraparib PO QD on days 1-21. Patients also receive dostarlimab IV over 30 minutes on day 1 Q3W for cycles 1-4 and Q6W for subsequent cycles. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.\>\> \>\> Dostarlimab: Given IV\>\> \>\> Niraparib: Given PO
Objective Response Rate (ORR)
0 Participants

SECONDARY outcome

Timeframe: From the end of study treatment to receiving the next treatment, assessed up to 5 years

TTNT will be estimated using the Kaplan-Meier method.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From study entry to death from any cause, assessed up to 4 years and 5 months

OS will be estimated using the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure
Treatment (Niraparib, Dostarlimab)
n=18 Participants
Patients receive niraparib PO QD on days 1-21. Patients also receive dostarlimab IV over 30 minutes on day 1 Q3W for cycles 1-4 and Q6W for subsequent cycles. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.\>\> \>\> Dostarlimab: Given IV\>\> \>\> Niraparib: Given PO
Overall Survival (OS)
27.9 months
Interval 27.3 to
Not enough events to calculate upper bound

SECONDARY outcome

Timeframe: From the first documented date of confirmed response (complete response [CR] or partial response [PR]) to the date at which progression is first documented, assessed up to 5 years

Population: Since 0 responses, Time to and duration of confirmed response is Not Applicable. We didn't have a single confirmed response.

Will be assessed using the Kaplan-Meier method.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From study entry to the first of either disease progression or death from any cause, assessed up to 4 years and 5 months

PFS will be estimated using the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure
Treatment (Niraparib, Dostarlimab)
n=18 Participants
Patients receive niraparib PO QD on days 1-21. Patients also receive dostarlimab IV over 30 minutes on day 1 Q3W for cycles 1-4 and Q6W for subsequent cycles. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.\>\> \>\> Dostarlimab: Given IV\>\> \>\> Niraparib: Given PO
Progression-free Survival (PFS)
16.4 months
Interval 13.6 to
Not enough events to calculate upper bound

SECONDARY outcome

Timeframe: Up to 4 years and 5 months

The maximum grade for each type of adverse event will be summarized using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. The frequency and percentage of grade 3+ adverse events will be estimated. Will also assess AEs at least possibly related to treatment as well.

Outcome measures

Outcome measures
Measure
Treatment (Niraparib, Dostarlimab)
n=18 Participants
Patients receive niraparib PO QD on days 1-21. Patients also receive dostarlimab IV over 30 minutes on day 1 Q3W for cycles 1-4 and Q6W for subsequent cycles. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.\>\> \>\> Dostarlimab: Given IV\>\> \>\> Niraparib: Given PO
Incidence of Adverse Events (AEs)
Any Grade 3 or Higher
15 Participants
Incidence of Adverse Events (AEs)
Any Grade 4 or Higher
3 Participants
Incidence of Adverse Events (AEs)
Grade 5
2 Participants
Incidence of Adverse Events (AEs)
Hematologic Grade 3 or Higher
9 Participants
Incidence of Adverse Events (AEs)
Hematologic Grade 4 or Higher
1 Participants
Incidence of Adverse Events (AEs)
Hematologic Grade 5
0 Participants
Incidence of Adverse Events (AEs)
Non-Hematologic Grade 3 or Higher
12 Participants
Incidence of Adverse Events (AEs)
Non-Hematologic Grade 4 or Higher
2 Participants
Incidence of Adverse Events (AEs)
Non-Hematologic Grade 5
2 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 years

Due to the limited sample size, these analyses will be hypothesis generating and descriptive in nature. Descriptive statistics will be summarized and the blood and tissue marker data will be correlated with clinical endpoints (response, DCR12, duration of response, OS, PFS, adverse events, etc.). For time-to-event data, the Kaplan-Meier method will be used. For categorical data, will use the Fisher's exact test. For marker data used to predict binary outcomes (i.e. response versus \[vs\]. no response), will use logistic regression models.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 5 years

Due to the limited sample size, these analyses will be hypothesis generating and descriptive in nature. Descriptive statistics will be summarized and the blood and tissue marker data will be correlated with clinical endpoints (response, DCR12, duration of response, OS, PFS, adverse events, etc.). For time-to-event data, the Kaplan-Meier method will be used. For categorical data, will use the Fisher's exact test. For marker data used to predict binary outcomes (i.e. response vs. no response), will use logistic regression models.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 years

Due to the limited sample size, these analyses will be hypothesis generating and descriptive in nature. Descriptive statistics will be summarized and the blood and tissue marker data will be correlated with clinical endpoints (response, DCR12, duration of response, OS, PFS, adverse events, etc.). For time-to-event data, the Kaplan-Meier method will be used. For categorical data, will use the Fisher's exact test. For marker data used to predict binary outcomes (i.e. response vs. no response), will use logistic regression models.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 years

Due to the limited sample size, these analyses will be hypothesis generating and descriptive in nature. Descriptive statistics will be summarized and the blood and tissue marker data will be correlated with clinical endpoints (response, DCR12, duration of response, OS, PFS, adverse events, etc.). For time-to-event data, the Kaplan-Meier method will be used. For categorical data, will use the Fisher's exact test. For marker data used to predict binary outcomes (i.e. response vs. no response), will use logistic regression models.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 5 years

Due to the limited sample size, these analyses will be hypothesis generating and descriptive in nature. Descriptive statistics will be summarized and the blood and tissue marker data will be correlated with clinical endpoints (response, DCR12, duration of response, OS, PFS, adverse events, etc.). For time-to-event data, the Kaplan-Meier method will be used. For categorical data, will use the Fisher's exact test. For marker data used to predict binary outcomes (i.e. response vs. no response), will use logistic regression models.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 5 years

Due to the limited sample size, these analyses will be hypothesis generating and descriptive in nature. Descriptive statistics will be summarized and the blood and tissue marker data will be correlated with clinical endpoints (response, DCR12, duration of response, OS, PFS, adverse events, etc.). For time-to-event data, the Kaplan-Meier method will be used. For categorical data, will use the Fisher's exact test. For marker data used to predict binary outcomes (i.e. response vs. no response), will use logistic regression models.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Niraparib, Dostarlimab)

Serious events: 2 serious events
Other events: 20 other events
Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure
Treatment (Niraparib, Dostarlimab)
n=20 participants at risk
Niraparib: Given PO
Cardiac disorders
Asystole
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Infections and infestations
Infections and infestations - Oth spec
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months

Other adverse events

Other adverse events
Measure
Treatment (Niraparib, Dostarlimab)
n=20 participants at risk
Niraparib: Given PO
Blood and lymphatic system disorders
Anemia
95.0%
19/20 • Number of events 59 • Up to 4 years and 5 months
Blood and lymphatic system disorders
Blood and lymph sys disorders - Oth Spec
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Endocrine disorders
Hypothyroidism
20.0%
4/20 • Number of events 11 • Up to 4 years and 5 months
Gastrointestinal disorders
Abdominal distension
5.0%
1/20 • Number of events 5 • Up to 4 years and 5 months
Gastrointestinal disorders
Abdominal pain
15.0%
3/20 • Number of events 4 • Up to 4 years and 5 months
Gastrointestinal disorders
Ascites
5.0%
1/20 • Number of events 6 • Up to 4 years and 5 months
Gastrointestinal disorders
Constipation
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Gastrointestinal disorders
Diarrhea
25.0%
5/20 • Number of events 6 • Up to 4 years and 5 months
Gastrointestinal disorders
Duodenal obstruction
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Gastrointestinal disorders
Esophageal hemorrhage
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Gastrointestinal disorders
Flatulence
5.0%
1/20 • Number of events 2 • Up to 4 years and 5 months
Gastrointestinal disorders
Gastrointestinal disorders - Oth spec
10.0%
2/20 • Number of events 2 • Up to 4 years and 5 months
Gastrointestinal disorders
Nausea
15.0%
3/20 • Number of events 4 • Up to 4 years and 5 months
Gastrointestinal disorders
Rectal hemorrhage
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Gastrointestinal disorders
Vomiting
15.0%
3/20 • Number of events 4 • Up to 4 years and 5 months
General disorders
Fatigue
85.0%
17/20 • Number of events 52 • Up to 4 years and 5 months
General disorders
Fever
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
General disorders
Gait disturbance
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
General disorders
Pain
65.0%
13/20 • Number of events 42 • Up to 4 years and 5 months
Hepatobiliary disorders
Hepatobiliary disorders - Other, specify
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Infections and infestations
Abdominal infection
5.0%
1/20 • Number of events 5 • Up to 4 years and 5 months
Infections and infestations
Anorectal infection
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Infections and infestations
Sepsis
10.0%
2/20 • Number of events 2 • Up to 4 years and 5 months
Investigations
Alanine aminotransferase increased
20.0%
4/20 • Number of events 6 • Up to 4 years and 5 months
Investigations
Alkaline phosphatase increased
5.0%
1/20 • Number of events 2 • Up to 4 years and 5 months
Investigations
Aspartate aminotransferase increased
30.0%
6/20 • Number of events 9 • Up to 4 years and 5 months
Investigations
Blood bilirubin increased
10.0%
2/20 • Number of events 2 • Up to 4 years and 5 months
Investigations
Creatinine increased
25.0%
5/20 • Number of events 19 • Up to 4 years and 5 months
Investigations
Lymphocyte count decreased
25.0%
5/20 • Number of events 13 • Up to 4 years and 5 months
Investigations
Neutrophil count decreased
10.0%
2/20 • Number of events 4 • Up to 4 years and 5 months
Investigations
Platelet count decreased
40.0%
8/20 • Number of events 20 • Up to 4 years and 5 months
Investigations
Weight loss
5.0%
1/20 • Number of events 3 • Up to 4 years and 5 months
Investigations
White blood cell decreased
10.0%
2/20 • Number of events 4 • Up to 4 years and 5 months
Metabolism and nutrition disorders
Hyperglycemia
55.0%
11/20 • Number of events 21 • Up to 4 years and 5 months
Metabolism and nutrition disorders
Hyperkalemia
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Metabolism and nutrition disorders
Hypoalbuminemia
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Metabolism and nutrition disorders
Hypocalcemia
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Metabolism and nutrition disorders
Hypokalemia
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Metabolism and nutrition disorders
Hyponatremia
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
5.0%
1/20 • Number of events 2 • Up to 4 years and 5 months
Nervous system disorders
Dizziness
10.0%
2/20 • Number of events 4 • Up to 4 years and 5 months
Nervous system disorders
Encephalopathy
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Nervous system disorders
Headache
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Psychiatric disorders
Confusion
5.0%
1/20 • Number of events 2 • Up to 4 years and 5 months
Psychiatric disorders
Delirium
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Renal and urinary disorders
Acute kidney injury
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Reproductive system and breast disorders
Pelvic pain
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Respiratory, thoracic and mediastinal disorders
Dyspnea
25.0%
5/20 • Number of events 7 • Up to 4 years and 5 months
Respiratory, thoracic and mediastinal disorders
Hypoxia
5.0%
1/20 • Number of events 2 • Up to 4 years and 5 months
Respiratory, thoracic and mediastinal disorders
Pleural effusion
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months
Respiratory, thoracic and mediastinal disorders
Pneumonitis
5.0%
1/20 • Number of events 2 • Up to 4 years and 5 months
Skin and subcutaneous tissue disorders
Photosensitivity
5.0%
1/20 • Number of events 4 • Up to 4 years and 5 months
Skin and subcutaneous tissue disorders
Rash acneiform
10.0%
2/20 • Number of events 3 • Up to 4 years and 5 months
Vascular disorders
Hypertension
5.0%
1/20 • Number of events 5 • Up to 4 years and 5 months
Vascular disorders
Thromboembolic event
5.0%
1/20 • Number of events 1 • Up to 4 years and 5 months

Additional Information

Dr. Robert R McWilliams

Mayo Clinic

Phone: 507-284-2511

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place