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Testing the Combination of Two Experimental Drugs MK-3475 (Pembrolizumab) and Interferon-gamma for the Treatment of Mycosis Fungoides and Sézary Syndrome and Advanced Synovial Sarcoma

A Phase II Trial of MK-3475 (Pembrolizumab) and Interferon Gamma 1-b Combination Immunotherapy in Patients With Previously Treated Mycosis Fungoides and Sezary Syndrome (Treatment Group 1) and in Patients With Advanced Synovial Sarcoma (Treatment Group 2)

Status
Completed
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT03063632
Enrollment
28
Registered
2017-02-24
Start date
2017-12-14
Completion date
2023-03-14
Last updated
2023-10-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Metastatic Myxoid Liposarcoma, Metastatic Round Cell Liposarcoma, Metastatic Synovial Sarcoma, Recurrent Mycosis Fungoides and Sezary Syndrome, Refractory Mycosis Fungoides and Sezary Syndrome, Stage IB Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage IIA Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage IIB Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage IIIA Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage IIIB Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage III Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage II Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage IVA Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage IVB Mycosis Fungoides and Sezary Syndrome AJCC v7, Stage IV Mycosis Fungoides and Sezary Syndrome AJCC v7, Unresectable Synovial Sarcoma

Brief summary

This phase II trial studies how well pembrolizumab and interferon gamma-1b work in treating patients with stage IB-IVB mycosis fungoides and Sezary syndrome that has come back (relapsed) or has not responded to previous treatment (refractory). Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Interferon gamma-1b may boost the immune system activity. Giving pembrolizumab and interferon gamma-1b together may work better in treating patients with stage IB-IVB mycosis fungoides and Sezary syndrome.

Detailed description

PRIMARY OBJECTIVES: I. To assess the overall response rate (ORR) of MK-3475 (pembrolizumab) and interferon gamma-1b (IFN-G) (Actimmune) combination immunotherapy in subjects with previously treated mycosis fungoides or Sezary syndrome. (Treatment Group 1) II. To determine whether the combination of interferon gamma-1b (ACTIMMUNE) and MK-3475 (pembrolizumab) improves the ORR of pembrolizumab in patients with unresectable or metastatic synovial sarcoma. (Treatment Group 2) SECONDARY OBJECTIVES: I. To explore the safety/tolerability and clinical activity of MK-3475 (pembrolizumab) and IFN-G (Actimmune) in subjects with previously treated mycosis fungoides or Sezary syndrome with respect to (Treatment Group 1): Ia. Safety and tolerability. Ib. Time to response (TTR). Ic. Duration of response (DOR). Id. Progression-free survival (PFS). Ie. Event-free survival (EFS). If. Percentage of all patients who have a response duration of at least 12 months (ORR12). II. To determine the progression-free survival (PFS) and overall survival (OS) for patients with advanced synovial sarcoma receiving interferon gamma-1b and MK-3475 (pembrolizumab). (Treatment Group 2) III. To determine the tolerability of the combination of interferon gamma-1b and MK-3475 (pembrolizumab) based on Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. (Treatment Group 2) EXPLORATORY OBJECTIVES: I. To investigate the relationship between the following putative biomarkers for combination immunotherapy of MK-3475 pembrolizumab) and IFN-gamma (Actimmune) and clinical outcomes (as measured by safety/tolerability and ORR, DOR, PFS, EFS) in subjects with previously treated mycosis fungoides or Sezary syndrome, including tumor/microenvironment (PD-1/PD-L1/PD-L2 expression, cytotoxic T lymphocyte \[CTL\]s, regulatory T cell \[Treg\]s, macrophages, dendritic cell \[DC\]s; nanostring gene expression profile), systemic immune response (flow cytometry, mass cytometry \[CyTOF\], Luminex multiplexed cytokine profile), and molecular/genomic immune correlates (exome sequencing, high throughput sequencing \[HTS\] for T cell receptor \[TCR\]). (Treatment Group 1) II. To investigate paired, serial biopsy specimens from pre-treatment and 8-12 weeks after starting treatment for the following (Treatment Group 2): IIa. MHC class I expression (scored by pathologist). IIb. Number of infiltrating T cells per mm\^2. IIc. Tumor associated macrophage number and phenotype using multiplex immunohistochemistry. IId. T cell clonality. IIe. Gene expression profiling. III. To investigate peripheral blood samples from patients to determine (Treatment Group 2): IIIa. The number and phenotype of T cells specific for computed tomography (CT) antigens and potential neo-antigens. IIb. The phenotype and activation state of circulating monocytes and peripheral blood mononuclear cell (PBMC). IIc. Cytokines associated with response. OUTLINE: Patients are assigned to 1 of 2 groups. GROUP I: Patients with Mycosis Fungoides and Sezary Syndrome receive pembrolizumab intravenously (IV) over 30 minutes on day 1. Cycles repeat every 3 weeks for up to 2 years in the absence of disease progression or unexpected toxicity. Patients also receive interferon gamma-1b subcutaneously (SC) 3 times per week for 12 weeks, and then follow 3 weeks on and 3 weeks off schedule for up to 2 years in the absence of disease progression or unexpected toxicity. GROUP II: Patients with advanced synovial sarcoma receive pembrolizumab IV over 30 minutes on day 1 and interferon gamma-1b SC once a week. Cycles repeat every 3 weeks for up to 2 years in the absence of disease progression or unexpected toxicity. After completion of study treatment, patients are followed up for 30 days and then every 12 weeks for up to 1 year.

Interventions

BIOLOGICALInterferon Gamma-1b

Given SC

OTHERLaboratory Biomarker Analysis

Ancillary studies

BIOLOGICALPembrolizumab

Given IV

Sponsors

National Cancer Institute (NCI)
Lead SponsorNIH

Study design

Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No

Inclusion criteria

* MYCOSIS FUNGOIDES /SEZARY SYNDROME (TREATMENT GROUP I) * Stage IB-IVB Mycosis Fungoides or Sezary syndrome, and who have relapsed, are refractory, or progressed after at least one standard systemic therapy; maximal stage since diagnosis will determine eligibility; current disease stage at time of entry will also be documented but will not be used for eligibility * Subjects must have the following minimum wash-out from previous treatments and without treatment between documentation of relapse/progression and enrollment: * \>= 2 weeks for local radiation therapy * \>= 8 weeks for low dose (12 Gy or less) Total Skin Electron Beam Therapy (TSEBT) * \>= 4 weeks for systemic cytotoxic anticancer agents, anticancer investigational agents that are not defined as immunotherapy, or for tumor-targeting monoclonal antibodies (mAbs) with the exception of alemtuzumab for which the washout is at least 16 weeks * \>= 15 weeks for anti-CD137 or anti-CTLA-4 (including ipilimumab or any other antibody or drug specifically targeting T-cell co-stimulation or checkpoint pathways) * \>= 2 weeks from resolution (i.e., \< grade 1 or at baseline) from adverse event (AE)s due to procedures performed or therapeutic agents administered * \>= 2 weeks for retinoids, interferons, vorinostat, romidepsin and denileukin diftitox * \>= 4 weeks for doses of systemic corticosteroids greater than 10 mg/day of prednisone or equivalent; patients who are on physiologic doses of corticosteroids (prednisone equivalent 10 mg/day or less) may participate, however, they must be on a stable dose for at least 4 weeks before enrollment; patients who are on low or moderate potency topical corticosteroids may participate if they are on a stable dose for at least 4 weeks before enrollment; inhaled corticosteroids are acceptable; local injections of corticosteroids are acceptable; all corticosteroids will be reported as concomitant medications * \>= 2 weeks for phototherapy * \>= 1 week for topical therapy (including retinoid, nitrogen mustard, or imiquimod) * Patients with prior treatment with IFN-gamma will be eligible, if they previously tolerated IFN-gamma, however patients must be off of IFN-gamma for at least three weeks before initiation of therapy on this trial * Age \>= 18 years * Have measurable disease based on modified severity-weighted assessment tool (mSWAT); tumor lesions situated in a previously irradiated area are considered measurable if progression has been demonstrated in such lesions * Have a performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) performance scale * Absolute neutrophil count (ANC) \>= 1500/mcL (performed within 10 days of treatment initiation) * Platelets \>= 100000/mcL (performed within 10 days of treatment initiation) * Hemoglobin \>= 9 g/dL or \>= 5.6 mmol/L (performed within 10 days of treatment initiation) * Creatinine =\< 1.5 x upper limit normal (ULN) (performed within 10 days of treatment initiation) OR * Measured or calculated creatinine clearance \>= 60 mL/min for patient with creatinine levels \> 1.5 x institutional ULN (performed within 10 days of treatment initiation) * Creatinine clearance (CrCl) should be calculated per institutional standard; glomerular filtration rate (GFR) can also be used in place of creatinine or CrCl * Total bilirubin =\< 1.5 x ULN OR direct bilirubin =\< ULN for patients with total bilirubin levels \> 1.5 x ULN (performed within 10 days of treatment initiation) * Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2.5 x ULN OR =\< 5 x ULN for patients with liver metastases (performed within 10 days of treatment initiation) * The effects of MK-3475 (pembrolizumab) and interferon-gamma on the developing human fetus are unknown; for this reason and because anti-PD-1 agents and interferons may be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) before to study entry and for the duration of study participation * Female patients of childbearing potential must have a negative urine or serum pregnancy test within 72 hours before receiving the first dose of study medication; if the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required * Female patients of childbearing potential must be willing to use an adequate method of contraception for the course of the study through 120 days after the last dose of study medication; Note: abstinence is acceptable if this is the usual lifestyle and preferred contraception for the patient * Male patients of reproductive potential must agree to use an adequate method of contraception starting with the first dose of study therapy through 120 days after the last dose of study therapy; Note: abstinence is acceptable if this is the usual lifestyle and preferred contraception for the patient * 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 before the study, for the duration of study participation, and 4 months after completion of MK-3475 (pembrolizumab) and interferon-gamma administration * Ability to understand and the willingness to sign a written informed consent document * SYNOVIAL SARCOMA (TREATMENT GROUP II) * Diagnosis of translocation associated sarcoma that generally expresses NY-ESO-1 (e.g., synovial sarcoma or myxoid/round cell liposarcoma); tumor must have been reviewed by a bone and soft tissue pathologist; patient must have metastatic or unresectable disease * At least one prior line of chemotherapy * Age \>= 12 years; patients \>= 18 years of age must be able and willing to provide informed consent; patients under 18 years of age must have a parent or guardian willing and able to provide consent * Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2 * Life expectancy greater than or equal to (\>=) 12 weeks * Measurable disease, as defined by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 * Tumor safely accessible for biopsy * Adequate hematologic and end organ function * For female participants of childbearing potential and male participants with partners of childbearing potential, agreement (by participant and/or partner) to use highly effective form(s) of contraception

Exclusion criteria

* MYCOSIS FUNGOIDES /SEZARY SYNDROME (TREATMENT GROUP I): * Has disease that is suitable for local therapy administered with curative intent * Patients who have had chemotherapy or targeted small molecule therapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) before entering the study * Patients who have had an allogeneic stem cell transplant are excluded because such transplants disrupt the normal immune response to a very substantial degree; in addition, emerging data suggests exacerbation of lethal graft versus host disease (GVHD) may occur in such patients when treated post allotransplant with PD-1 blockade * Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 * Patients who have received an investigational agent or have used an investigational device within 4 weeks of the first dose of study drug * Has a history of a well-characterized and defined immune deficiency before the diagnosis of mycosis fungoides or Sezary syndrome or is receiving systemic steroid therapy greater than 10 mg/day of prednisone or equivalent within 4 weeks or any other form of immunosuppressive therapy within 7 days before the first dose of trial treatment * Has had a prior monoclonal antibody within 4 weeks before study day 1 or who has not recovered (i.e., =\< grade 1 or at baseline) from AEs due to agents administered more than 4 weeks earlier * Note: the following will not be exclusionary: patients may have any grade alopecia or lymphopenia and still participate if other inclusion/

Design outcomes

Primary

MeasureTime frameDescription
Overall Response Rate (ORR)Up to 2 yearsParticipants in Treatment Group 1 will be assessed for response and progression using standard response criteria in patients with Mycosis Fungoides and Sezary syndrome. Per Global Response Score determined by evaluating skin, lymph nodes, internal organs (viscera), and blood specimens: Complete Response (CR), complete disappearance of all clinical evidence of disease; Partial Response (PR), regression of measurable disease. Participants in Treatment Group 2 will be assessed for response and progression using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria. Complete Response (CR) disappearance of all lesions and no new lesion; Partial Response (PR), 30% or greater reduction in tumor size and no new lesions. The ORR is defined as CR combined with PR. Will be assessed using binomial proportion. Best response at any timepoint was used to determine ORR.

Secondary

MeasureTime frameDescription
Incidence of Adverse EventsUp to 2 years and 1 monthsWill be graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time to Response (TTR)Time interval between the date of first treatment and the date of response (complete response [CR]/partial response [PR]), up to 2 yearsWill use simple statistics.
Duration of Response (DOR)Time interval between the date of first response (CR/PR) and the date of progression, up to 2 years and 11 monthsWill be assessed using the Kaplan-Meier method.
Event-free Survival (EFS)Termination due to toxicity, initiation of next significant treatment, progressive disease, or death of any cause, up to 2 yearsWill be assessed using the Kaplan-Meier method.
Rate of Overall Response Duration Beyond 12 Months (ORR12)From the time measurement criteria are met for CR or PR (whichever is first recorded) until the first date beyond 12 months that recurrent disease is objectively documented, up to 2 yearsWill be assessed per global assessment of mycosis fungoides and Sezary syndrome (confirmed & investigator assessed). Will use binomial distribution.
Progression-free Survival (PFS)Time from enrollment to disease progression or death, whichever occurs earlier, based upon investigator assessment, up to 3 yearsWill be assessed using the Kaplan-Meier method

Other

MeasureTime frameDescription
Biomarkers in Tumor and Blood Assessed by Immunohistochemistry, Mass Spectrometry, Nanostring, Sequencing, and Enzyme-linked Immunosorbent AssayUp to 3 yearsClinical response (as measured by the primary and secondary endpoints) to combination immunotherapy regimen as a function of biomarkers will be evaluated. All biomarker assays will be run on the clinical trial samples in a blinded manner. Time to event endpoints (DOR, PFS, EFS) will be evaluated using Kaplan-Meier curves generated by biomarker scoring group using the log-rank test. Exploratory outcomes will be summarized with descriptive statistics (primarily proportions and medians) for all biomarkers described above, and additionally, serum IL-10, regulatory T-cells, and activated CD8 T cells. Scatterplots and Kendall's tau estimates will be produced for estimating correlation of PD-1, PDL1, or PD-L2 expression measures and clinical outcome across compartments (skin, lymph node, blood). Comparison of tissue immunohistochemistry markers between pre-treatment and with clinical response or disease progression will be assessed using Wilcoxon signed-rank test.

Countries

United States

Participant flow

Participants by arm

ArmCount
Group I (Pembrolizumab, Interferon Gamma-1b)
Patients receive pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 3 weeks for up to 2 years in the absence of disease progression or unexpected toxicity. Patients also receive interferon gamma-1b SC 3 times per week for 12 weeks, and then follow 3 weeks on and 3 weeks off schedule for up to 2 years in the absence of disease progression or unexpected toxicity. Interferon Gamma-1b: Given SC Laboratory Biomarker Analysis: Ancillary studies Pembrolizumab: Given IV
16
Group II (Pembrolizumab, Interferon Gamma-1b)
Patients pembrolizumab IV over 30 minutes on day 1 and interferon gamma-1b SC once a week. Cycles repeat every 3 weeks for up to 2 years in the absence of disease progression or unexpected toxicity. Interferon Gamma-1b: Given SC Laboratory Biomarker Analysis: Ancillary studies Pembrolizumab: Given IV
12
Total28

Withdrawals & dropouts

PeriodReasonFG000FG001
Overall StudyDeath46
Overall StudyLost to Follow-up01
Overall StudyOther: Disease progression; starting another treatment11
Overall StudyPhysician Decision20
Overall StudyWithdrawal by Subject02

Baseline characteristics

CharacteristicGroup I (Pembrolizumab, Interferon Gamma-1b)Group II (Pembrolizumab, Interferon Gamma-1b)Total
Age, Categorical
<=18 years
0 Participants0 Participants0 Participants
Age, Categorical
>=65 years
8 Participants1 Participants9 Participants
Age, Categorical
Between 18 and 65 years
8 Participants11 Participants19 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Asian
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Black or African American
4 Participants0 Participants4 Participants
Race (NIH/OMB)
More than one race
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants
Race (NIH/OMB)
White
12 Participants12 Participants24 Participants
Region of Enrollment
United States
16 participants12 participants28 participants
Sex: Female, Male
Female
5 Participants3 Participants8 Participants
Sex: Female, Male
Male
11 Participants9 Participants20 Participants

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
deaths
Total, all-cause mortality
4 / 166 / 12
other
Total, other adverse events
15 / 1612 / 12
serious
Total, serious adverse events
4 / 165 / 12

Outcome results

Primary

Overall Response Rate (ORR)

Participants in Treatment Group 1 will be assessed for response and progression using standard response criteria in patients with Mycosis Fungoides and Sezary syndrome. Per Global Response Score determined by evaluating skin, lymph nodes, internal organs (viscera), and blood specimens: Complete Response (CR), complete disappearance of all clinical evidence of disease; Partial Response (PR), regression of measurable disease. Participants in Treatment Group 2 will be assessed for response and progression using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria. Complete Response (CR) disappearance of all lesions and no new lesion; Partial Response (PR), 30% or greater reduction in tumor size and no new lesions. The ORR is defined as CR combined with PR. Will be assessed using binomial proportion. Best response at any timepoint was used to determine ORR.

Time frame: Up to 2 years

Population: Both Treatment Group 1 and Treatment Group 2 study participants received at least one dose of IV pembrolizumab on study, and 27 participants received at least one dose of Interferon gamma on study. While not all participants reached the first disease assessment timepoint specified in the protocol, all had documented disease status at the time of treatment or study discontinuation. Therefore, no study participants were excluded from ORR analysis due to missing data.

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
Group I (Pembrolizumab, Interferon Gamma-1b)Overall Response Rate (ORR)6 Participants
Group II (Pembrolizumab, Interferon Gamma-1b)Overall Response Rate (ORR)0 Participants
Secondary

Duration of Response (DOR)

Will be assessed using the Kaplan-Meier method.

Time frame: Time interval between the date of first response (CR/PR) and the date of progression, up to 2 years and 11 months

Population: Non-responders are excluded in analysis; censored at last follow-up or start of new significant therapy

ArmMeasureValue (MEDIAN)
Group I (Pembrolizumab, Interferon Gamma-1b)Duration of Response (DOR)505.0 days
Secondary

Event-free Survival (EFS)

Will be assessed using the Kaplan-Meier method.

Time frame: Termination due to toxicity, initiation of next significant treatment, progressive disease, or death of any cause, up to 2 years

Population: All efficacy or safety evaluable patients. Event is defined by early termination due to toxicity, start of new significant therapy, PD, or death of any cause.

ArmMeasureValue (MEDIAN)
Group I (Pembrolizumab, Interferon Gamma-1b)Event-free Survival (EFS)185.5 days
Group II (Pembrolizumab, Interferon Gamma-1b)Event-free Survival (EFS)73.0 days
Secondary

Incidence of Adverse Events

Will be graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

Time frame: Up to 2 years and 1 months

Population: All treated subjects.

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
Group I (Pembrolizumab, Interferon Gamma-1b)Incidence of Adverse Events15 Participants
Group II (Pembrolizumab, Interferon Gamma-1b)Incidence of Adverse Events12 Participants
Secondary

Progression-free Survival (PFS)

Will be assessed using the Kaplan-Meier method

Time frame: Time from enrollment to disease progression or death, whichever occurs earlier, based upon investigator assessment, up to 3 years

Population: All efficacy or safety evaluable patients. Censored at last follow-up or start of new significant therapy.

ArmMeasureValue (MEDIAN)
Group I (Pembrolizumab, Interferon Gamma-1b)Progression-free Survival (PFS)394.0 days
Group II (Pembrolizumab, Interferon Gamma-1b)Progression-free Survival (PFS)196.5 days
Comparison: Progression is assessed by the standard response criteria used in Mycosis Fungoides and Sezary Syndrome (skin, lymph nodes, viscera, blood, global). Per Global Response Score, progression is defined as Progressive Disease (PD) in any category (i.e., skin, lymph nodes, viscera, blood).
Secondary

Rate of Overall Response Duration Beyond 12 Months (ORR12)

Will be assessed per global assessment of mycosis fungoides and Sezary syndrome (confirmed & investigator assessed). Will use binomial distribution.

Time frame: From the time measurement criteria are met for CR or PR (whichever is first recorded) until the first date beyond 12 months that recurrent disease is objectively documented, up to 2 years

Population: Evaluable duration of response beyond 12 months for evaluable population.

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
Group I (Pembrolizumab, Interferon Gamma-1b)Rate of Overall Response Duration Beyond 12 Months (ORR12)2 Participants
Group II (Pembrolizumab, Interferon Gamma-1b)Rate of Overall Response Duration Beyond 12 Months (ORR12)0 Participants
Secondary

Time to Response (TTR)

Will use simple statistics.

Time frame: Time interval between the date of first treatment and the date of response (complete response [CR]/partial response [PR]), up to 2 years

Population: Non-responders are excluded in the analysis.

ArmMeasureValue (MEDIAN)
Group I (Pembrolizumab, Interferon Gamma-1b)Time to Response (TTR)126 days
Comparison: Per Global Response Score used in Mycosis Fungoides and Sezary Syndrome, Response is assessed by the standard response criteria: Complete Response (CR), complete disappearance of all clinical evidence of disease where all categories (i.e., skin, lymph nodes, viscera, blood) have complete response/noninvolved; Partial Response (PR), regression of measurable disease; Stable Disease (SD), failure to attain CR, PR, or PD.
Other Pre-specified

Biomarkers in Tumor and Blood Assessed by Immunohistochemistry, Mass Spectrometry, Nanostring, Sequencing, and Enzyme-linked Immunosorbent Assay

Clinical response (as measured by the primary and secondary endpoints) to combination immunotherapy regimen as a function of biomarkers will be evaluated. All biomarker assays will be run on the clinical trial samples in a blinded manner. Time to event endpoints (DOR, PFS, EFS) will be evaluated using Kaplan-Meier curves generated by biomarker scoring group using the log-rank test. Exploratory outcomes will be summarized with descriptive statistics (primarily proportions and medians) for all biomarkers described above, and additionally, serum IL-10, regulatory T-cells, and activated CD8 T cells. Scatterplots and Kendall's tau estimates will be produced for estimating correlation of PD-1, PDL1, or PD-L2 expression measures and clinical outcome across compartments (skin, lymph node, blood). Comparison of tissue immunohistochemistry markers between pre-treatment and with clinical response or disease progression will be assessed using Wilcoxon signed-rank test.

Time frame: Up to 3 years

Source: ClinicalTrials.gov · Data processed: Feb 18, 2026