A Study to Evaluate the Effectiveness and Safety of Pembrolizumab Combined with Bacillus Calmette-Guerin in High-Risk Non-Muscle Invasive Bladder Cancer


About this study

The purpose of this study is to assess the antitumor efficacy and safety of pembrolizumab in combination with BCG, compared to BCG monotherapy, in participants with HR NMIBC that is either persistent or recurrent following adequate BCG induction or that is naïve to BCG treatment.


Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- Have locally and blinded independent central review (BICR)-confirmed histological
diagnosis of high-risk non-muscle invasive (T1, high grade Ta and/or CIS) UC of the

- Has undergone cystoscopy/ transurethral resection of bladder tumor (TURBT) to remove
all resectable disease

- Has provided tissue for biomarker analysis

- Has Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2

- Has adequate organ function

- During the treatment period and for ≥7 days after the last dose of BCG, male
participants are EITHER abstinent from heterosexual intercourse as their preferred and
usual lifestyle and agree to remain abstinent, OR, must agree to use contraception
unless confirmed to be azoospermic

- Female participants who are not pregnant, not breastfeeding, and either not a woman of
child bearing potential (WOCBP); or are a WOCBP who agrees to use a contraception
method that is highly effective or remains abstinent from heterosexual intercourse
during the treatment period and for ≥7 days after the last dose of BCG or 120 days
after the last dose of pembrolizumab, whichever comes last

BCG Post-induction Cohort (Cohort A) Only

- Has been treated with one adequate course of BCG induction therapy for the treatment

- Following adequate BCG induction therapy, must have persistent or recurrent HR NMIBC

Exclusion Criteria:

- Has a history of or concurrent locally advanced (i.e., T2, T3, T4) or metastatic UC

- Has concurrent extra-vesical (i.e, urethra, ureter, renal pelvis) non-muscle invasive
urothelial carcinoma or a history of extra-vesical non-muscle invasive UC

- Has received prior therapy with anti-PD-1, anti-PD-L1, or anti-PD-L2 agent or with an
agent directed to another stimulatory or co-inhibitory T-cell receptor

- Has received prior systemic anti-cancer therapy including investigational agents
within 4 weeks of start of study treatment

- Is currently participating in or has participated in a study of an investigational
agent or has used an investigational device within 4 weeks of start of study treatment

- Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy
or any other form of immunosuppressive therapy within 7 days of start of study

- Has a known additional malignancy that is progressing or requires active treatment
within the past 3 years

- Has an active autoimmune disease that has required systemic treatment in past 2 years

- Has a history of (non-infectious) pneumonitis/interstitial lung disease that required
steroids or has current pneumonitis/interstitial lung disease

- Has one or more of the following contraindications to BCG: prior BCG sepsis or
systemic infection, total bladder incontinence, or an adverse experience to a previous
BCG instillation that resulted in treatment discontinuation and precludes retreating
with BCG

- Has an active infection or diagnosis requiring systemic antimicrobial therapy

- Has a known history of human immunodeficiency virus (HIV) infection

- Has a known history of Hepatitis B or known active Hepatitis C virus infection

- Has current active tuberculosis

- Has had an allogenic-tissue/solid organ transplant

- Has any contraindication(s) to IV contrast or is otherwise unable to have screening
imaging with IV contrast performed

BCG Post-induction Cohort (Cohort A) Only - Has persistent T1 disease following an
induction course of BCG

BCG Naïve Cohort (Cohort B) Only

- Has received any prior treatment with BCG for their NMIBC within the past 2 years prior
to study entry

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Timothy Lyon, M.D.

Open for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office


Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Mark Tyson, M.D., M.P.H.

Open for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

More information


Publications are currently not available

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