A Phase 3 interventional study of Pembrolizumab and Epacadostat in UC (Urothelial Cancer), sponsored by Incyte Corporation. Completed at 143 sites in 18 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-08-22.
Sponsored by Incyte Corporation · Phase 3, Interventional, and Treatment
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The purpose of this study was to evaluate the efficacy and safety of pembrolizumab + epacadostat vs pembrolizumab + placebo in participants with cisplatin-ineligible urothelial carcinoma.
Incyte Corporation is the lead sponsor of 288 studies on the registry; 34 are open to participants now.
Of its 149 completed or terminated interventional studies of FDA-regulated products, 95 (64%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
PD-1, PD-L1 and CTLA-4 are proteins that act as brakes on the immune system. Checkpoint inhibitors, such as pembrolizumab or nivolumab, block them so the immune system can attack a cancer.
PD-L1 can also be measured in a tumour sample. Some scores count stained tumour cells only (TPS); others also count certain immune cells (CPS). The study names the score and the minimum it uses.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
RECIST
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
PD-1, PD-L1 and checkpoint inhibitors
PD-1, PD-L1 and CTLA-4 are proteins that act as brakes on the immune system. Checkpoint inhibitors, such as pembrolizumab or nivolumab, block them so the immune system can attack a cancer.
PD-L1 can also be measured in a tumour sample. Some scores count stained tumour cells only (TPS); others also count certain immune cells (CPS). The study names the score and the minimum it uses.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Brain metastases
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Pembrolizumab + epacadostat
Drug: Pembrolizumab · Drug: Epacadostat
Pembrolizumab + placebo
Drug: Pembrolizumab · Drug: Placebo
Pembrolizumab administered intravenously every 3 weeks.
Also known as: MK-3475
Epacadostat administered orally twice daily.
Also known as: INCB024360
Matching placebo administered orally twice daily.
Objective Response Rate (ORR) With Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo
ORR was defined as the percentage of participants who had a confirmed complete response (CR) or partial response (PR) per RECIST v1.1 by investigator determination. Responses are based on Investigator assessments per RECIST 1.1 without confirmation using all scans up to the cutoff date.
Time frame: Week 9
Safety and Tolerability of Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo as Measured by Number of Participants Experiencing Adverse Events (AEs)
AE is defined as any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment.
Time frame: Up to approximately 25 months
Safety and Tolerability of Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo as Measured by Number of Participants Discontinuing Study Treatment Due to AE
AE is defined as any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment.
Time frame: Up to approximately 25 months
This study was conducted at 47 centers in 15 countries.
| Milestone | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID |
|---|---|---|
| Started | 44 | 49 |
| Intention-to-treat (itt) | 44 | 49 |
| All participants as treated (apat) | 43 | 49 |
| Completed | 25 | 22 |
| Not completed | 19 | 27 |
| Withdrew: Death | 17 | 18 |
| Withdrew: Physician decision | 1 | 9 |
| Withdrew: Withdrawal by subject | 1 | 0 |
ORR was defined as the percentage of participants who had a confirmed complete response (CR) or partial response (PR) per RECIST v1.1 by investigator determination. Responses are based on Investigator assessments per RECIST 1.1 without confirmation using all scans up to the cutoff date.
| percentage of participants | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID |
|---|---|---|
| Objective Response Rate (ORR) With Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo | 31.8 (22.46 to 55.24) | 24.5 (15.33 to 43.67) |
AE is defined as any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment.
| Participants | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID |
|---|---|---|
| Safety and Tolerability of Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo as Measured by Number of Participants Experiencing Adverse Events (AEs) | 43 | 47 |
AE is defined as any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment.
| Participants | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID |
|---|---|---|
| Safety and Tolerability of Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo as Measured by Number of Participants Discontinuing Study Treatment Due to AE | 11 | 15 |
Collected over up to approximately 25 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pembrolizumab 200 mg + Epacadostat 100 mg BID | 17/43 (39.5%) | 23/43 (53.5%) | 42/43 (97.7%) |
| Pembrolizumab 200 mg + Placebo BID | 19/49 (38.8%) | 23/49 (46.9%) | 47/49 (95.9%) |
| Total | 36/92 (39.1%) | 46/92 (50%) | 89/92 (96.7%) |
| Event | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID | Total |
|---|---|---|---|
| Malignant neoplasm progressionNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 8/43 | 5/49 | 13/92 |
| Urinary tract infectionInfections and infestations | 3/43 | 6/49 | 9/92 |
| Chronic kidney diseaseRenal and urinary disorders | 0/43 | 3/49 | 3/92 |
| Device occlusionProduct Issues | 2/43 | 0/49 | 2/92 |
| HaematuriaRenal and urinary disorders | 2/43 | 0/49 | 2/92 |
| UrosepsisInfections and infestations | 2/43 | 1/49 | 3/92 |
| Acute kidney injuryRenal and urinary disorders | 0/43 | 2/49 | 2/92 |
| AnaemiaBlood and lymphatic system disorders | 1/43 | 0/49 | 1/92 |
| DeathGeneral disorders | 1/43 | 1/49 | 2/92 |
| EncephalitisInfections and infestations | 1/43 | 0/49 | 1/92 |
| Event | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID | Total |
|---|---|---|---|
| AnaemiaBlood and lymphatic system disorders | 14/43 | 9/49 | 23/92 |
| RashSkin and subcutaneous tissue disorders | 10/43 | 14/49 | 24/92 |
| DiarrhoeaGastrointestinal disorders | 10/43 | 7/49 | 17/92 |
| PruritusSkin and subcutaneous tissue disorders | 10/43 | 6/49 | 16/92 |
| Urinary tract infectionInfections and infestations | 5/43 | 11/49 | 16/92 |
| AstheniaGeneral disorders | 9/43 | 10/49 | 19/92 |
| Back painMusculoskeletal and connective tissue disorders | 7/43 | 8/49 | 15/92 |
| Decreased appetiteMetabolism and nutrition disorders | 5/43 | 8/49 | 13/92 |
| FatigueGeneral disorders | 6/43 | 8/49 | 14/92 |
| ConstipationGastrointestinal disorders | 7/43 | 7/49 | 14/92 |
The Intention-to-Treat (ITT) population consisted of all randomized participants.
| Age, Continuous(years) | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID | Total |
|---|---|---|---|
| Mean | 73.3 ± 9.5 | 72.4 ± 8.9 | 72.8 ± 9.1 |
| Sex: Female, Male(Participants) | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID | Total |
|---|---|---|---|
| Female | 11 | 11 | 22 |
| Male | 33 | 38 | 71 |
| Ethnicity (NIH/OMB)(Participants) | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID | Total |
|---|---|---|---|
| Hispanic or Latino | 2 | 0 | 2 |
| Not Hispanic or Latino | 35 | 42 | 77 |
| Unknown or Not Reported | 7 | 7 | 14 |
| Race/Ethnicity, Customized(Participants) | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID | Total |
|---|---|---|---|
| Asian | 9 | 8 | 17 |
| White | 33 | 37 | 70 |
| Missing | 2 | 4 | 6 |
| Metastasis Status at Screening(Participants) | Pembrolizumab 200 mg + Epacadostat 100 mg BID | Pembrolizumab 200 mg + Placebo BID | Total |
|---|---|---|---|
| Metastatic | 38 | 45 | 83 |
| Advanced/Unresectable | 6 | 4 | 10 |
Showing the first 100 of 143 sites across 18 countries. Choose a country and city above to see the rest.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Dec 2017 to primary completion in Aug 2018, 8 months.
Results were first posted in Aug 2019, 1 year after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Epacadostat
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