Clinical Trial Summary

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Phase

Phase 2/Phase 3

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Funding Agency/Sponsor

Industrial

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Disease

Prostate, Bladder, and Kidney Cancers

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Enrollment Eligibility

Inclusion Criteria:
- Male or female patients 18 years of age or older
- Histologic confirmation of non-muscle invasive bladder cancer of the transitional cell
carcinoma high-grade subtype (mixed histology tumors allowed if transitional cell
histology is predominant histology).
- Histologically confirmed presence of BCG-unresponsive CIS (with or without Ta or T1
disease) or histologically confirmed presence of BCG-unresponsive high-grade Ta or T1
disease.
- Absence of resectable disease after transurethral resection (TURBT) procedures
(residual carcinoma in situ (CIS) acceptable; patients with T1 tumors must undergo
repeat resection and biopsy [inclusive of muscularis propria] if initial biopsy did
not include muscularis propria). Patients with high-grade Ta and/or T1 disease should
have complete resection before study treatment.
- BCG-unresponsive disease as defined as: (a) Persistent or recurrent CIS (+/- recurrent
Ta/T1 disease) within 12 months of receiving adequate BCG (at least five of six doses
doses of an initial induction course plus either at least two of three doses of
maintenance therapy or at least two of six doses of a second induction course); or (b)
Recurrent high-grade Ta/T1 disease within 6 months of completion of adequate BCG (at
least five of six doses of an initial induction course plus either at least two of
three doses of maintenance therapy or at least two of six doses of a second induction
course); or (c) T1 high-grade disease at the first evaluation following an induction
BCG course alone (at least five of six doses of an initial induction course).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- Voluntary written informed consent and HIPAA authorization and agree to comply with
all protocol-specified procedures and follow-up evaluations
Exclusion Criteria:
- Recurrence of BCG unresponsive Ta/T1 disease (without presence of CIS) > 6 months
after last BCG instillation or BCG unresponsive CIS > 12 months after last BCG
instillation.
- Life expectancy <2 years
- Any of the following clinical laboratory values at the time of enrollment: (1)
Absolute neutrophil count (ANC) <800/µL or (2) Platelets < 50,000/µL
- Liver function abnormalities as indicated by ongoing hepatic enzyme elevation (AST or
ALT) >2 times upper limit of normal (ULN)
- Renal insufficiency as indicated by a creatinine level >3 times ULN
- History of or evidence of muscle-invasive, locally advanced, metastatic and/or
extravesical bladder cancer (inclusive of the prostatic urethra); or any other cancer
within the past 5 years that is progressing or requires active treatment. Exceptions
are adequately treated basal cell or squamous cell skin cancer that has undergone
potentially curative therapy or in situ cervical cancer; and adequately treated stage
I or II cancer or stable prostate cancer from which the patient is currently in
complete remission, and is under active surveillance or hormone control.
- Symptomatic congestive heart failure (CHF), New York Heart Association (NYHA) Class
III or IV heart failure or other clinical signs of severe cardiac dysfunction
- Severe/unstable angina pectoris, or myocardial infarction within 6 months prior to
study entry
- History or evidence of uncontrollable central nervous system (CNS) disease
- Active systemic infection requiring parenteral antibiotic therapy. All prior
infections must have resolved following optimal therapy
- Concurrent febrile illness, active urinary tract infection, active tuberculosis, a
history of hypotension or anaphylactic reactions
- Ongoing chronic systemic steroid therapy required (>10 mg oral prednisone daily or
equivalent)
- Women who are pregnant or nursing. Female patients of childbearing potential must have
a negative pregnancy test and must adhere to using a medically acceptable method of
birth control prior to screening and agree to continue its use during the study and
for 30 days after the last dose of study drug, or be surgically sterilized (e.g.,
hysterectomy or tubal ligation). Women of childbearing potential are defined as any
female who has experienced menarche and who is NOT permanently sterile or
postmenopausal. Postmenopausal is defined as 12 consecutive months with no menses
without an alternative medical cause. Males must agree to use barrier methods of birth
control while on study and for 90 days post last dose of study drug.
- Patients currently receiving investigational or commercial anti-cancer agents or
anti-cancer therapies other than BCG, ALT-803 and supportive care therapies for active
disease.
- Concurrent use of other investigational agents (not including FDA authorized drugs for
the prevention and treatment of COVID-19).
- Other illness or condition, including laboratory abnormalities, which in the opinion
of the Investigator would exclude the patient from participating in this study. This
includes, but is not limited to, serious medical conditions or psychiatric illness
likely to interfere with participation in the study.

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