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Protocol Details

Phase 1 with Expansion Cohorts in a Study of NEO-201 In Adults with Chemo-Resistant Solid Tumors

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Cancer Institute (NCI)

Recruitment Detail

Type: Participants currently recruited/enrolled
Gender: Male & Female
Min Age: 18 Years
Max Age: N/A

Referral Letter Required


Population Exclusion(s)

Pregnant Women


Monoclonal Antibody;
Mucinous Cancer;
Gastrointestinal Cancer;

Recruitment Keyword(s)



Carcinoma, Solid;
Carcinoma, Gastrointestinal;
Carcinoma, Mucinous;
Carcinoma, Colorectal;
Carcinoma, Pancreatic;
Endometrial Cancer

Investigational Drug(s)

NEO-201 (h16C3)

Investigational Device(s)



Biological/Vaccine: Pembrolizumab
Biological/Vaccine: NEO-201

Supporting Site

National Cancer Institute


Typical cancer treatment includes surgery, radiation, and chemotherapy. Some people have tumors that do not respond to these. In other cases, the tumors respond to treatment, but then come back. Researchers want to see if the new antibody/drug NEO-201 can help.


To evaluate the safety of NEO-201.


Adults age 18 and older who have certain solid tumor (cancer) that has not responded to standard therapy. They must not be eligible for standard therapy that is known to confer clinical benefit.


Participants will be screened with

Medical history

Physical exam

Blood and urine tests

Electrocardiogram (ECG) test of heart function


Study of tissue sample

Participants will get NEO-201 as an infusion through a catheter (small plastic tube) in a large vein in the arm or neck over 2-8 hours.

Participants will have their vital signs monitored during and after the infusion.

Participants will get a dose every 2 weeks for 4 doses. Then they will have a 2-week break (a total of 8 weeks). Four doses, plus a visit 2 weeks after the last dose, will equal 1 course of treatment. One course will last about 57 days.

Participants will have additional physical exams and blood tests during the study. At the end of each course, they will have scans to evaluate their disease.

Participants may continue to get NEO-201 until their disease worsens or they have unacceptable side effects.

After stopping the study drug, participants will have a follow-up visit. This will include blood tests and a physical exam.

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Eligible subjects must meet the following inclusion criteria:

AGE: >= 18 years of age.


-Subjects must have histologically or cytologically confirmed recurrent, locally advanced unresectable or metastatic cancer confirmed by the Laboratory of Pathology, NCI, who have progressed, recurred or been unable to tolerate front-line therapy.

-Must have archived tissue (10 unstained slides or tissue block), or must have tumor which can be safely biopsied percutaneously and be willing to undergo a tumor biopsy.

-Expansion Cohorts: please see additional inclusion criteria.

MEASURABLE/EVALUABLE DISEASE: Subjects must have disease that is measurable by RECIST 1.1 or evaluable by bone scan, peritoneal or pleural effusions, or carcinomatosis.

PERFORMANCE STATUS: ECOG <= 2; or Karnofsky performance status of >= 50%


Screening laboratory data within 21 days of the first dose of study drug. Subject must have adequate organ function:

-Hemoglobin > 9 g/dL, or on stable doses (hematocrit stable within 1 gram and dose stable for one month) of erythropoietin or similar medication.

-Absolute neutrophil count (ANC) >=1,500/mm3.

-Platelets >=100,000/mm3.

-Total bilirubin <= 2.0 mg/dL

-ALT and AST <= 3 times the ULN, or, if the subject has liver metastases, <= 5 times the ULN.

-Creatinine <= 1.5 mg/dL or creatinine clearance > 40 mL/min/1.73 m^2 for subjects with creatinine levels above institutional normal, as calculated by the

Cockcroft Gault formula.

INFORMED CONSENT: Voluntary written informed consent before performance of any study-related procedure that is not part of normal medical care.


-At least 14 days must have elapsed since treatment with oral tyrosine kinase inhibitors, or until toxicities associated with TKI therapy have resolved.

-At least 21 days must have elapsed since treatment with previous monoclonal antibodies aside from immune checkpoint inhibitors, or until toxicities associated with mAb therapy have resolved.

-At least 4 weeks must have elapsed since any chemotherapeutic agents at the time of enrollment (or 6 weeks for regimens containing BCNU or mitomycin C).

-At least 3 weeks must have elapsed if the patient has previously been treated with pembrolizumab or any other type of immunotherapy including immune checkpoint inhibitors, tumor vaccines, or cellular immunotherapies.

-At least 2 weeks must have elapsed since any systemic corticosteroids at the time of enrollment

-XRT: At least 7 days after local palliative XRT (small port)

Subjects must have recovered from any acute toxicity related to prior therapy, except for alopecia. Toxicity should be <= grade 1, or <= grade 2 for peripheral neuropathy or hypothyroidism.

Subject is expected to be able to remain on a study protocol for at least 8 weeks.

BIRTH CONTROL: Female subject is post-menopausal, surgically sterilized, or willing to use acceptable methods of birth control (i.e., a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, or condom with spermicide, or abstinence) for the duration of the study and 4 months after the last dose of pembrolizumab.

Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after the last dose of pembrolizumab.

Additional Inclusion Criteria for Disease-Specific Expansion Cohorts

Subjects enrolled in the expansion cohorts must have advanced non-small cell lung cancer, HNSCC, uterine cancer, or cervical cancer that has progressed during or after at least one front-line standard of care treatment, including chemotherapy and/or targeted therapy, and also:

-Tumor is positive for NEO-201 antigen expression (defined as at least 10% of tumor cells expressing NEO-201).

-Patient is not a candidate for potentially curative surgery or radiation.

-Tumor has the additional characteristics described below for the disease specific expansion cohorts:


-Tumor(s) must express PD-L1 (TPS >= 1%) as determined by an FDA approved test and/or is microsatellite instability-high (MSI-H) or mismatch repair deficient, and/or is tumor mutational burden-high (TMB-H) [ (Bullet)10 mutations/megabase (mut/Mb)], as determined by an FDA-approved test.

-Patients with EGFR, ALK1, ROS1 or BRAF V600E genomic tumor aberrations must have had disease progression on FDA-approved agents for these aberrations.

-Patients without these genomic tumor aberrations must have received immune-checkpoint inhibitor previously, either as a single agent or in combination with chemotherapy.

-Cervical Cancer:

-tumor(s) express a combined positive score (CPS) >= 1, as determined by an FDA approved test and/or is microsatellite instability-high (MSI-H) or mismatch repair deficient, and/or is tumor mutational burden-high (TMB-H)[>=10 mutations/megabase (mut/Mb)], as determined by an FDA-approved test


-Patients are allowed to have received pembrolizumab previously, either alone

or as part of a multiagent regimen.

-Uterine carcinoma

-Patients who are eligible to have received the combination of pembrolizumab plus lenvatinib (i.e. patients with tumors that are not MSI or dMMR) must have received this regimen, unless considered unsafe due to comorbid conditions (e.g. hypertension, proteinuria).


Subjects meeting any of the following exclusion criteria are not to be enrolled in the study:

-Subject has history of disseminated or uncontrolled brain metastases or central nervous system disease. Brain metastases will be considered controlled if SD on two consecutive brain MRIs, performed at least 2 months apart, and subject is without seizures.

-History of allergic reactions attributed to compounds of similar chemical or biologic composition to NEO-201, pembrolizumab or other agents used in this study.

-Any major surgery within 14 days of enrollment.

-Subjects who are receiving any other investigational agents.

-Subject does not have archival tissue available and does not have a lesion(s) that can be safely biopsied via percutaneous route, or is unwilling to undergo biopsy.

-Subject has an uncontrolled concomitant illness including, but not limited to, ongoing or active infection, uncontrolled diabetes mellitus, symptomatic congestive heart failure, unstable angina pectoris, hypokalemia, family history of Long QT Syndrome or presence of cardiac arrhythmia.

-Subjects who are assessed to have unacceptable risk of developing infection from neutropenia will be excluded at the Investigator s discretion.

-HIV-positive subjects on combination antiretroviral therapy are ineligible because of the unknown potential for pharmacokinetic interactions with NEO-201. In addition, these subjects are at increased risk of lethal infections which could complicate the toxicity assessment of this study. Appropriate studies will be undertaken in subjects receiving combination antiretroviral therapy when indicated.

-Subject has other serious medical illness, including a second malignancy, or psychiatric illness that could, in the Investigator s opinion, potentially interfere with the completion of treatment according to this protocol.

-Pregnant women are excluded from this study because the potential for teratogenic or abortifacient effects due to NEO-201 is unknown. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with NEO- 201, breastfeeding should be discontinued if the mother is treated with NEO-201.

Additional Exclusion Criteria for Expansion Cohorts

Because patients in the expansion cohort will be receiving pembrolizumab, the following additional exclusion criteria apply:

-Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy (in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of

immunosuppressive therapy within 7 days prior to start of study therapy.

-Active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g.., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.

-Subjects who experienced severe or life-threatening immune-related AEs with prior immune checkpoint therapy requiring medical intervention (steroid or immunosuppressant drugs) and permanent discontinuation of therapy, will be excluded. These include, but not limited to colitis, autoimmune hepatitis, hypophysitis, hyperthyroidism, nephritis, myocarditis, GBS, encephalitis.

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Not Provided

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Principal Investigator

Referral Contact

For more information:

Kevin C. Conlon, M.D.
National Cancer Institute (NCI)
(240) 760-6087

Ann C. McCoy, R.N.
National Cancer Institute (NCI)
National Institutes of Health
Building 82
Room 233
9030 Old Georgetown Road
Bethesda, Maryland 20892
(240) 760-6021

NCI Referral Office
National Institute of Health Clinical Center (CC), 9000 Rockville Pike, Bethesda, Maryland 20892, United States: NCI Clinical Trials Referral Office

Clinical Trials Number:


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