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Bladder CancerPhase 1NCT05055050

A Window of Opportunity Phase I Study of UGN-201 in Patients With Bladder Cancer Undergoing Radical Cystectomy Protocol #: 2021-0630

A Phase 1 study testing UGN-201 in people with Bladder Cancer and Bladder, run by M.D. Anderson Cancer Center at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

he primary objective is to characterize the safety profile of UGN-201 in patients with urothelial carcinoma undergoing radical cystectomy. Corresponding primary endpoint: Toxicity of concern (TOX) will be monitored until 30 days after surgery or until the patient meets the surgery prevention or delay TOX definition… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Bladder Cancer, Bladder
What is being tested?
UGN-201
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
24 Jan 2022to 31 Aug 2026
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • In order to be eligible for participation in this trial, the subject must:
  • Be willing and able to provide written informed consent
  • Be ≥ 18 years of age
  • Have histological confirmation of predominant urothelial cancer (either non-muscle invasive or muscle invasive). Patients whose tumors are found to be invasive should not have stage 3 or greater disease.
  • Have absence of metastatic disease as determined by conventional imaging studies and be considered a good surgical candidate by the treating physician.
  • Be willing to participate in the collection of blood, tissue, stool, and urine for banking and future correlative studies as specified in the Study Flow Chart (Section 6.0).
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  • Have a performance status of 0 or 1 on the ECOG Performance Scale.
  • Demonstrate adequate organ function as defined in Table 1, all screening labs should be performed within 14 days of treatment initiation.
  • Female subject of childbearing potential should have a negative urine or serum pregnancy within 72 hours prior to 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 subjects of childbearing potential (Section 5.7.2) must be willing to use an adequate method of contraception as outlined in Section 5.7.2 - Contraception, for the course of the study through 30 days after the last dose of study medication.
  • 10. Male subjects of childbearing potential (Section 5.7.2) must agree to use an adequate method of contraception as outlined in Section 5.7.2- Contraception, starting with the first dose of study therapy through 30 days after the last dose of study therapy.
  • Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject.
  • -Patients must consent to the MD Anderson Immunotherapy Platform laboratory protocol PA13-0291.

Who cannot join

  • The subject must be excluded from participating in the trial if the subject:
  • Is currently participating and receiving UGN-201 or has participated in a study of an investigational agent and received UGN-201 or used an investigational device within 4 weeks of the first dose of study treatment.
  • Has ≥ cT3 and/or cN+ and/or cM+ urothelial carcinoma of the bladder, non-predominant urothelial carcinoma or histologic variants, such as small cell, carcinosarcoma, squamous cell or adenocarcinoma.
  • Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of study treatment.
  • Has a known history of active TB (Bacillus Tuberculosis).
  • Has a known history of hypersensitivity to UGN-201 or any of its excipients.
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  • Has had prior systemic anti-cancer therapy for the treatment of bladder cancer. Prior intravesical therapies, whether BCG, chemotherapy or otherwise, will remain eligible Has cT3 or bulkier urothelial carcinoma of the bladder
  • Has any other malignancy diagnosed within 2 years of screening with the exception of basal or squamous cell skin cancer, or non-invasive cancer of the cervix, or any other cancer deemed by the treating physician to be of low risk for progression or patient morbidity during the study period.
  • Has known metastatic disease as determined by conventional staging studies.
  • Has 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.
  • Has known history of, or any evidence of active, non-infectious pneumonitis.
  • Has a clinically significant active infection requiring systemic therapy and cannot be resolved prior to initiating treatment.
  • Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating physician.
  • Is pregnant or breastfeeding or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment.
  • Clinically significant urethral stricture that would preclude passage of a urethral catheter.
  • History of pelvic radiotherapy.
  • History of neurogenic bladder.
  • History of active urinary retention.
  • History of any other condition that would prohibit normal voiding.
  • Has received prior therapy with an anti-PD-1, anti-PD-L1, anti-PD-L2, or anti-CTLA-4 agent.
  • Has a known history of Human Immunodeficiency Virus (HIV) (HIV 1/2 antibodies).
  • Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected).
  • Has received a vaccine within 30 days of initiation of study therapy. Exceptions will be made for inactivated seasonal influenza and COVID-19 vaccines at the discretion of the treating physician. Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines; however intranasal influenza vaccines (e.g., Flu-Mist®) are live attenuated vaccines, and are not allowed under any circumstances.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • M D Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: A Window of Opportunity Phase I Study of UGN-201 in Patients With Bladder Cancer Undergoing Radical Cystectomy

he primary objective is to characterize the safety profile of UGN-201 in patients with urothelial carcinoma undergoing radical cystectomy.

Corresponding primary endpoint: Toxicity of concern (TOX) will be monitored until 30 days after surgery or until the patient meets the surgery prevention or delay TOX definition below, whichever comes first. A patient will be considered to have a TOX if any of the following apply:

Read more
  • Any 30-day grade 3 or higher surgical complication at least possibly related to UGI-201
  • Any toxicity at least possibly related to the treatment that prevents surgery or delays it more than 12 weeks from date of cystectomy decision with MDACC Urologist. Missing/delayed surgery due to progression or withdrawal not related to toxicity will not count as a TOX event. Rapid progression is not seen with UGN-201. For such a patient, TOX monitoring will follow for 30 days after the administered dose of UGN-201.
  • Death between the start of study and the 30-day post-surgical assessment will count if it is toxic death at least possibly related to the UGN-201 or surgery. Deaths clearly unrelated to treatment will not count as an event.

Adverse events will be recorded using CTCAE v5 and surgical complications will be recorded using Clavien-Dindo classification.

Exploratory objectives are:

  • To evaluate the efficacy of UGN-201 by pathologic T0 and ≤ pT1 rate (pathologic down-staging) after neoadjuvant treatment with UGN-201, in patients with NMBIC and MIBC undergoing radical cystectomy, respectively.
  • To assess the immunological/biomarker changes in tumor tissues, peripheral blood, and urine in response to UGN-201 treatment in patients with bladder cancer undergoing radical cystectomy and to explore any potential association between these biomarker measures and antitumor activity.

Patients with MIBC will be defined as having a response if their pathologic stage is pT1 stage or less. Patients with NMIBC will be defined as having a response if their pathologic stage is pT0.

Immunologic and other biomarker measures will be recorded by laboratory standards for each measure.

Read the detailed description

Primary Objective:

-To characterize the safety profile of UGN-201 in patients with urothelial carcinoma undergoing radical cystectomy.

Exploratory Objectives:

  • To evaluate the efficacy of UGN-201 by pathologic T0 and ≤ pT1 rate (pathologic downstaging) after neoadjuvant treatment with UGN-201, in patients with NMBIC and MIBC undergoing radical cystectomy, respectively.
  • To assess the immunological/biomarker changes in tumor tissues, peripheral blood, stool and urine in response to UGN-201 treatment in patients with bladder cancer undergoing radical cystectomy and to explore any potential association between these biomarker measures and antitumor activity.
Being tested
UGN-201
Medical subject headings
Urinary Bladder Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • UGN-201Drug

    Also called imiquimod intravesical solution

    Intravesical 200 mg /50 ml x 1 dose

Study groups in NCT05055050
Study groupRoleWhat they receive
UGN-201 Pre Radical CystectomyExperimentalUGN-201 200 mg/50ml
Receives: Drug: UGN-201

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To establish the safety profile of UGN-201 in patients with urothelial carcinoma undergoing radical cystectomy.

    Measured: through study completion, an average of 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Sep 2021
  2. Study start24 Jan 2022
  3. Primary completion31 Aug 2026 (estimated)
  4. Study completion31 Aug 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05055050
Sponsor's protocol ID
2021-0630
Other IDs
NCI-2021-09539
Status last verified
Aug 2026
First submitted
7 Sep 2021
Last updated
6 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing UGN-201 in people with Bladder Cancer and Bladder, run by M.D. Anderson Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 6 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To establish the safety profile of UGN-201 in patients with urothelial carcinoma undergoing radical cystectomy. (time frame: through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05055050.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2026.

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