Trial Outcomes & Findings for Study of GVAX (With CY) and Pembrolizumab in MMR-p Advanced Colorectal Cancer (NCT NCT02981524)

Last Updated: 2021-02-10

Results Overview

ORR is defined as the number of patients achieving a complete response (CR) or partial response (PR) based on the Response Evaluation Criteria in Solid Tumors (RECIST 1.1) at any time during the study. CR = disappearance of all target lesions, PR is =\>30% decrease in sum of diameters of target lesions, progressive disease (PD) is \>20% increase in sum of diameters of target lesions, stable disease (SD) is \<30% decrease or \<20% increase in sum of diameters of target lesions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

up to 1 year

Results posted on

2021-02-10

Participant Flow

Participant milestones

Participant milestones
Measure	CY/GVAX With Pembrolizumab During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Overall Study STARTED	17
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	17

Reasons for withdrawal

Reasons for withdrawal
Measure	CY/GVAX With Pembrolizumab During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Overall Study Progressive Disease	14
Overall Study Death (unrelated)	1
Overall Study Treatment Related Toxicity	1
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Study of GVAX (With CY) and Pembrolizumab in MMR-p Advanced Colorectal Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CY/GVAX With Pembrolizumab n=17 Participants During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	4 Participants n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants
Race (NIH/OMB) White	13 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	17 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status ECOG 0	6 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status ECOG 1	11 Participants n=5 Participants
Baseline Carcinoembryonic Antigen (CEA)	84.8 ng/ml n=5 Participants

PRIMARY outcome

Timeframe: up to 1 year

Population: Data to assess objective response was only collected from 14/17 participants. The remaining 3 patients were withdrawn from study therapy for early clinical progression and were not evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	CY/GVAX With Pembrolizumab n=14 Participants During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Objective Response Rate (ORR)	0 Participants

SECONDARY outcome

Timeframe: up to 1 year

Number of participants who experience treatment related adverse events ≥ grade 3 as defined by CTCAE 4.0.

Outcome measures

Outcome measures
Measure	CY/GVAX With Pembrolizumab n=17 Participants During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Number of Participants With Adverse Events as a Measure of Safety and Tolerability	6 Participants

SECONDARY outcome

Timeframe: up to 1 year

Progression-free Survival (PFS) is defined as the number of days from cycle 1, day 1 of immunotherapy until first documented local progression or death due to any cause. PD is \>20% increase in sum of diameters of target lesions as assessed using RECIST (version 1.1).

Outcome measures

Outcome measures
Measure	CY/GVAX With Pembrolizumab n=17 Participants During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Progression Free Survival (PFS)	82 days Interval 48.0 to 97.0

SECONDARY outcome

Timeframe: Up to 1 year

OS is defined as the number of days from start of study treatment to time of death. Individuals will be censored at the date of the last study visit if no event occurs. The estimation method used was Kaplan-Meier.

Outcome measures

Outcome measures
Measure	CY/GVAX With Pembrolizumab n=17 Participants During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Overall Survival (OS)	213 days Interval 179.0 to 441.0

SECONDARY outcome

Timeframe: 1 year

Population: There were no patients that had a CR or PR. Therefore, data could not be collected to assess this outcome measure.

Number of weeks from the start date of PR or CR (whichever response is recorded first) and subsequently confirmed to the first date that recurrent or progressive disease or death is documented. Per RECIST 1.1, CR = disappearance of all target lesions, PR is =\>30% decrease in sum of diameters of target lesions.

Outcome measures

Outcome data not reported

Adverse Events

CY/GVAX With Pembrolizumab

Serious events: 6 serious events

Other events: 17 other events

Deaths: 14 deaths

Serious adverse events

Serious adverse events
Measure	CY/GVAX With Pembrolizumab n=17 participants at risk During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Eye disorders Eye disorders-Corneal transplant rejection	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Injury, poisoning and procedural complications Hip fracture	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Death, Disease progression	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms, progression (Brain metastases)	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Respiratory, thoracic and mediastinal disorders Dyspnea	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.

Other adverse events

Other adverse events
Measure	CY/GVAX With Pembrolizumab n=17 participants at risk During each 21 day cycles, Cyclophosphamide (CY) is administered on Day 1 at 200 mg/m2 followed by Pembrolizumab at 200mg, the colon cancer vaccine (GVAX) is administered on Day 2 at 5E8 colon cancer cells + 5E7 GM-CSF secreting cells. CY: CY is administered intravenously at 200 mg/m2 GVAX: GVAX is administered intradermally at 5E8 colon cancer cells + 5E7 GM-CSF secreting Pembrolizumab: Pembrolizumab is administered intravenously at 200 mg
Blood and lymphatic system disorders Anemia	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Blood and lymphatic system disorders Hemolytic anemia	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Endocrine disorders Hypothyroidism	23.5% 4/17 • Number of events 4 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Eye disorders Blurred vision	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Eye disorders Conjunctivitis	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Eye disorders Flashing lights	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Abdominal distension	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Abdominal pain	17.6% 3/17 • Number of events 3 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Bloating	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Constipation	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Diarrhea	17.6% 3/17 • Number of events 3 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Nausea	17.6% 3/17 • Number of events 3 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Rectal hemorrhage	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Rectal pain	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Gastrointestinal disorders Vomiting	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
General disorders Edema-limbs	17.6% 3/17 • Number of events 3 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
General disorders Fatigue	23.5% 4/17 • Number of events 4 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
General disorders Fever	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
General disorders Flu-like symptoms	29.4% 5/17 • Number of events 5 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
General disorders Pain	58.8% 10/17 • Number of events 10 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Injury, poisoning and procedural complications Fall	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Infections and infestations Urinary tract infection	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Investigations AST increased	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Investigations CPK increased	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Metabolism and nutrition disorders Anorexia	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Metabolism and nutrition disorders Hyponatremia	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Musculoskeletal and connective tissue disorders Myalgia	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Nervous system disorders Headache	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Nervous system disorders Lethargy	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Nervous system disorders Paresthesia	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Psychiatric disorders Confusion	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Psychiatric disorders Depression	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Renal and urinary disorders Urine discoloration	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Renal and urinary disorders Urinary incontinence	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Renal and urinary disorders Urinary pain	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Renal and urinary disorders Urinary retention	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Reproductive system and breast disorders Erectile dysfuntion	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Respiratory, thoracic and mediastinal disorders Dyspnea	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Respiratory, thoracic and mediastinal disorders Post-nasal drip	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Respiratory, thoracic and mediastinal disorders Sore throat	17.6% 3/17 • Number of events 3 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Bruising, vaccine site reaction	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Hyperhidrosis, nightsweats	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Erythema, vaccine site reaction	88.2% 15/17 • Number of events 15 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Induration, vaccine site reaction	94.1% 16/17 • Number of events 16 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Pain, vaccine site reaction	17.6% 3/17 • Number of events 3 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Macular papular rash	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Pruritis	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Pruritis, vaccine site reaction	88.2% 15/17 • Number of events 15 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Rash	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Skin and subcutaneous tissue disorders Warmth, vaccine site reaction	11.8% 2/17 • Number of events 2 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Vascular disorders Hot Flashes	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.
Nervous system disorders Memory Impairment	5.9% 1/17 • Number of events 1 • 1 year Adverse reporting collection was conducted by regular investigator assessment and laboratory measurements.

Additional Information

Nilofer Azad, MD

Sidney Kimmel Cancer Center at Johns Hopkins

Phone: 410-614-9169

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place