Bladder CancerPhase 2OPTIMA IINCT03558503
A Phase 2b Study of UGN-102 for Low Grade Intermediate Risk Non-Muscle-Invasive Bladder Cancer
A Phase 2 study testing UGN-102 in people with Bladder Cancer and Urothelial Carcinoma, run by UroGen Pharma Ltd. at 19 sites in 2 countries.
Trial summary
This Phase 2b, single-arm, multicenter study evaluated the efficacy and safety of UGN-102 as primary chemoablative therapy in patients with low grade intermediate risk non-muscle-invasive bladder cancer (LG IR NMIBC). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bladder Cancer, Urothelial Carcinoma, Urothelial Carcinoma Bladder
- What is being tested?
- UGN-102
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- 19 sitesin 2 countries
- Study dates
- 15 Oct 2018to 21 Oct 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Willing and able to sign an informed consent and comply with the protocol.
- Has newly diagnosed or historic LG NMIBC (Ta) histologically confirmed by cold cup biopsy at screening or within 6 weeks of screening.
- Is at intermediate risk of recurrence, defined as having 1 or 2 of the following:
- Presence of multiple tumors;
- Solitary tumor > 3 cm;
- Recurrence (≥ 1 occurrence of LG NMIBC within 1 year of the current diagnosis).
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- Has negative voiding cytology for high grade (HG) disease at or within 6 weeks of enrollment.
- Willing to use 2 acceptable forms of effective contraception from enrollment through 6 months post treatment if the participant is female or the female partner of a male participant and is of childbearing potential (defined as premenopausal women who have not been sterilized).
- Has adequate organ and bone marrow function as determined by routine laboratory tests as below:
- Leukocytes ≥ 3,000 per μL;
- Absolute neutrophil count ≥ 1,500 per μL;
- Platelets ≥ 100,000 per μL;
- Hemoglobin ≥ 9.0 g/dL;
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN);
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN;
- Alkaline phosphatase ≤ 2.5 × ULN;
- Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min.
- Has no evidence of active urinary tract infection (UTI) at Screening and Baseline visits.
- In the case of symptomatic UTI, the patient will be treated with a full course of antibiotics, and study drug will be postponed until resolution. In the case of asymptomatic bacteriuria, the use of prophylactic antibiotics and postponement of study drug is left to the discretion of the Principal Investigator (PI).
Who cannot join
- History of carcinoma in situ (CIS) on preliminary cystoscopy within 5 years of enrollment.
- Received Bacillus Calmette-Guérin (BCG) treatment for urothelial carcinoma (UC) within previous 2 years.
- History of HG papillary UC in the past 2 years.
- Known allergy or sensitivity to mitomycin.
- Clinically significant urethral stricture that would preclude passage of a urethral catheter.
- History of pelvic radiotherapy.
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- History of:
- Neurogenic bladder;
- Active urinary retention;
- Any other condition that would prohibit normal voiding.
- Past or current muscle invasive (ie, T2, T3, T4) or metastatic UC or concurrent upper tract urothelial carcinoma (UTUC).
- Has participated in a study with an investigational agent or device within 30 days of enrollment.
- History of prior treatment with an intravesical chemotherapeutic agent with the exception of a single dose of chemotherapy immediately after any previous transurethral resection of bladder tumors (TURBT).
- Has an underlying substance abuse or psychiatric disorder such that, in the opinion of the investigator, the patient would be unable to comply with the protocol.
Trial locations and contacts
19 sites in 2 countries.
19 sites in 2 countries
United States17 sites
John Hopkins University
Baltimore, Maryland, 21218
The University of North Carolina
Chapel Hill, North Carolina, 27572
Urology San Antonio
Fredericksburg, Texas, 78233
Penn State Hershey State College
Hershey, Pennsylvania, 17033
Urology Las Vegas
Las Vegas, Nevada, 89144
Arkansas Urology
Little Rock, Arkansas, 72211
Loma Linda University
Loma Linda, California, 92350
Carolina Urologic Research Center
Myrtle Beach, South Carolina, 29572
Urology Associates, PC
Nashville, Tennessee, 37209
Manhattan Medical Research
New York, New York, 10016
Weill Cornell Medical College
New York, New York, 10065
Montefiore Medical Center (Albert Einstein)
New York, New York, 10461
Western New York Urology Associates
New York, New York, 14225
Adult & Pediatric Urology, PC
Omaha, Nebraska, 68114
Mayo Clinic Cancer Center
Phoenix, Arizona, 85054
Clinical Research Center of Florida
Pompano Beach, Florida, 33060
Providence Medical Institute
Santa Monica, California, 90404
Israel2 sites
Carmel Medical Center
Haifa, 3436212
Rabin Medical Center
Petah Tikva, 49372
About this study
Official title: A Phase 2b, Single-Arm, Multicenter Trial to Evaluate the Efficacy and Safety of UGN-102 as Primary Chemoablative Therapy in Patients With Low Grade (LG) Non-Muscle-Invasive Bladder Cancer (NMIBC) at Intermediate Risk of Recurrence
This Phase 2b, single-arm, multicenter study evaluated the efficacy and safety of UGN-102 as primary chemoablative therapy in patients with low grade intermediate risk non-muscle-invasive bladder cancer (LG IR NMIBC).
Read the detailed description
Eligible patients were treated with 6 once-weekly intravesical instillations of UGN-102.
The ablative effect of UGN-102 was evaluated at the 3-month Visit, which occurred 5 weeks ± 1 week after the last weekly instillation (3 months after initiation of study drug). Response was determined based on visual evaluation by cystoscopy (appearance, number, and size of the lesions) and, if there were remaining lesions, by histopathology of the remaining lesions. Complete response (CR) was defined as having no detectable disease (NDD) and was assessed visually during cystoscopy and also by urine cytology. In the event that the investigator was not sure, and there was suspect tissue, a small biopsy was taken from the suspect tissue to confirm CR in addition to cystoscopy and urine cytology.
Patients who achieved a CR continued to have monthly telephone contacts to document any adverse events (AEs) and changes in concomitant medications and were assessed at 6, 9, and 12 months after the first instillation of UGN-102 for evidence of disease recurrence. Patients who had a non-complete response (non-CR) discontinued the study and continued with standard of care according to their treating physician.
Safety was determined based on a review of AEs, laboratory assessments, and physical examination findings.
- Being tested
- UGN-102
- Keywords
- Non-muscle invasive bladder cancer, Low grade non-muscle invasive bladder cancer, Intermediate risk non-muscle invasive bladder cancer, NMIBC, UGN-102, Mitomycin
- Medical subject headings
- Urinary Bladder Neoplasms, Carcinoma, Transitional Cell, Non-Muscle Invasive Bladder Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UGN-102Drug
Also called UGN-102 (mitomycin) for intravesical solution
UGN-102 consists of mitomycin and sterile hydrogel (a proprietary thermally responsive gel) that is used to reconstitute mitomycin before instillation. The reverse thermal properties of UGN-102 allow for local administration of mitomycin as a liquid, with subsequent conversion to a semi-solid gel depot following instillation into the bladder.
The UGN-102 admixture for intravesical instillations contains 75 mg mitomycin in 56 mL admixture (1.33 mg mitomycin per 1 mL).
| Study group | Role | What they receive |
|---|---|---|
| UGN-102 | Experimental | Patients were treated with 6 once-weekly intravesical instillations of UGN-102. Receives: Drug: UGN-102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response (CR) Rate for UGN-102 Treatment
To evaluate the tumor ablative effect of UGN-102 in patients with LG NMIBC. CR rate was defined as the percentage of patients with no detectable disease (NDD) as determined by cystoscopy, for cause biopsy, and urine cytology.
Measured: 3 months after the first instillation of UGN-102
Secondary outcomes (6)
Durable Complete Response (DCR) Rate
To evaluate the durability of response in patients with LG NMIBC who achieve CR. DCR rate was defined as the percentage of patients who maintained CR at the respective time point.
Measured: 6, 9, and 12 months after the first instillation of UGN-102
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Study Drug or Procedure Related TEAEs, TEAEs by Maximum Severity, TEAEs Leading to Treatment Discontinuation, and Serious TEAEs.
The number of patients with each type of event will be summarized. Adverse event severity was graded according to Common Terminology Criteria for Adverse Events version 5.0 as follows: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe or medically significant), Grade 4 (life-threatening), Grade 5 (fatal).
Measured: Up to 12 months
Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values
The number of patients who met PCS criteria for chemistry or hematology parameters post-baseline will be summarized
Measured: Up to 12 months
Number of Participants With Post-baseline PCS Vital Signs Values
The number of patients who met PCS criteria for vital signs parameters post-baseline will be summarized
Measured: Up to 12 months
Number of Participants With Post-baseline Clinically Significant (CS) Physical Examination Findings
The number of patients with abnormal, CS physical examination findings post-baseline will be summarized
Measured: Up to 12 months
Number of Participants With Post-baseline CS Urology-oriented Physical Examination Findings
The number of patients with abnormal, CS urology-oriented physical examination findings post-baseline will be summarized
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2018
- Study start15 Oct 2018
- Primary completion3 Jan 2020
- Study completion21 Oct 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Bladder CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- UroGen Pharma Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elyse Seltzer, MD, Study director, UroGen Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03558503
- Sponsor's protocol ID
- TC-BC-12
- Acronym
- OPTIMA II
- Status last verified
- Sep 2022
- First submitted
- 8 May 2018
- Last updated
- 16 Sep 2022
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Chevli KK, Shore ND, Trainer A, Smith AB, Saltzstein D, Ehrlich Y, Raman JD, Friedman B, D'Anna R, Morris D, Hu B, Tyson M, Sankin A, Kates M, Linehan J, Scherr D, Kester S, Verni M, Chamie K, Karsh L, Cinman A, Meads A, Lahiri S, Malinowski M, Gabai N, Raju S, Schoenberg M, Seltzer E, Huang WC. Primary Chemoablation of Low-Grade Intermediate-Risk Nonmuscle-Invasive Bladder Cancer Using UGN-102, a Mitomycin-Containing Reverse Thermal Gel (Optima II): A Phase 2b, Open-Label, Single-Arm Trial. J Urol. 2022 Jan;207(1):61-69. doi: 10.1097/JU.0000000000002186. Epub 2021 Aug 26.
PubMed 34433303Prasad SM, Louie MJ, Burger B, Tsurutis V, Ugwuoke N, Strauss-Ayali D. Pharmacokinetics of UGN-102, an investigational mitomycin-containing reverse thermal gel for the treatment of non-muscle invasive bladder cancer. Cancer Chemother Pharmacol. 2025 Oct 11;95(1):100. doi: 10.1007/s00280-025-04821-5.
PubMed 41074972Stover AM, Basak R, Mueller D, Lipman R, Teal R, Hilton A, Giannone K, Waheed M, Smith AB. Minimal Patient-Reported Side Effects for a Chemoablative Gel (UGN-102) Used as Frontline Treatment in Adults with Nonmuscle-Invasive Bladder Cancer. J Urol. 2022 Sep;208(3):580-588. doi: 10.1097/JU.0000000000002747. Epub 2022 May 31.
PubMed 35640276
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b, Single-Arm, Multicenter Trial to Evaluate the Efficacy and Safety of UGN-102 as Primary Chemoablative Therapy in Patients With Low Grade (LG) Non-Muscle-Invasive Bladder Cancer (NMIBC) at Intermediate Risk of Recurrence
- ClinicalTrials.gov ID
- NCT03558503
- Acronym / sponsor ID
- OPTIMA II / TC-BC-12
- Keywords
- Non-muscle invasive bladder cancer, Low grade non-muscle invasive bladder cancer, Intermediate risk non-muscle invasive bladder cancer, NMIBC, UGN-102, Mitomycin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- UroGen Pharma Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Oct 2018 (actual)
- Primary completion
- 3 Jan 2020 (actual)
- Study completion
- 21 Oct 2020 (actual)
- First posted
- 15 Jun 2018 (actual)
- Results
- Posted 25 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase 2b, single-arm, multicenter study evaluated the efficacy and safety of UGN-102 as primary chemoablative therapy in patients with low grade intermediate risk non-muscle-invasive bladder cancer (LG IR NMIBC).
Detailed description
Eligible patients were treated with 6 once-weekly intravesical instillations of UGN-102.
The ablative effect of UGN-102 was evaluated at the 3-month Visit, which occurred 5 weeks ± 1 week after the last weekly instillation (3 months after initiation of study drug). Response was determined based on visual evaluation by cystoscopy (appearance, number, and size of the lesions) and, if there were remaining lesions, by histopathology of the remaining lesions. Complete response (CR) was defined as having no detectable disease (NDD) and was assessed visually during cystoscopy and also by urine cytology. In the event that the investigator was not sure, and there was suspect tissue, a small biopsy was taken from the suspect tissue to confirm CR in addition to cystoscopy and urine cytology.
Patients who achieved a CR continued to have monthly telephone contacts to document any adverse events (AEs) and changes in concomitant medications and were assessed at 6, 9, and 12 months after the first instillation of UGN-102 for evidence of disease recurrence. Patients who had a non-complete response (non-CR) discontinued the study and continued with standard of care according to their treating physician.
Safety was determined based on a review of AEs, laboratory assessments, and physical examination findings.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UGN-102 | Experimental | Drug: UGN-102Patients were treated with 6 once-weekly intravesical instillations of UGN-102. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Willing and able to sign an informed consent and comply with the protocol.
- Has newly diagnosed or historic LG NMIBC (Ta) histologically confirmed by cold cup biopsy at screening or within 6 weeks of screening.
- Is at intermediate risk of recurrence, defined as having 1 or 2 of the following:
- Has negative voiding cytology for high grade (HG) disease at or within 6 weeks of enrollment.
- Willing to use 2 acceptable forms of effective contraception from enrollment through 6 months post treatment if the participant is female or the female partner of a male participant and is of childbearing potential (defined as premenopausal women who have not been sterilized).
Key exclusion criteria (14 listed)
- History of carcinoma in situ (CIS) on preliminary cystoscopy within 5 years of enrollment.
- Received Bacillus Calmette-Guérin (BCG) treatment for urothelial carcinoma (UC) within previous 2 years.
- History of HG papillary UC in the past 2 years.
- Known allergy or sensitivity to mitomycin.
- Clinically significant urethral stricture that would preclude passage of a urethral catheter.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UGN-102Also: UGN-102 (mitomycin) for intravesical solutionUGN-102 consists of mitomycin and sterile hydrogel (a proprietary thermally responsive gel) that is used to reconstitute mitomycin before instillation. The reverse thermal properties of UGN-102 allow for local administration of mitomycin as a liquid, with subsequent conversion to a semi-solid gel depot following instillation into the bladder. The UGN-102 admixture for intravesical instillations contains 75 mg mitomycin in 56 mL admixture (1.33 mg mitomycin per 1 mL). | Drug | UGN-102 |
Primary outcome
Complete Response (CR) Rate for UGN-102 Treatment
Time frame: 3 months after the first instillation of UGN-102
To evaluate the tumor ablative effect of UGN-102 in patients with LG NMIBC. CR rate was defined as the percentage of patients with no detectable disease (NDD) as determined by cystoscopy, for cause biopsy, and urine cytology.
6 secondary outcomes
Durable Complete Response (DCR) Rate
Time frame: 6, 9, and 12 months after the first instillation of UGN-102
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Study Drug or Procedure Related TEAEs, TEAEs by Maximum Severity, TEAEs Leading to Treatment Discontinuation, and Serious TEAEs.
Time frame: Up to 12 months
Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values
Time frame: Up to 12 months
Number of Participants With Post-baseline PCS Vital Signs Values
Time frame: Up to 12 months
Number of Participants With Post-baseline Clinically Significant (CS) Physical Examination Findings
Time frame: Up to 12 months
Number of Participants With Post-baseline CS Urology-oriented Physical Examination Findings
Time frame: Up to 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 34433303Chevli KK, Shore ND, Trainer A, Smith AB, Saltzstein D, Ehrlich Y, Raman JD, Friedman B, D'Anna R, Morris D, Hu B, Tyson M, Sankin A, Kates M, Linehan J, Scherr D, Kester S, Verni M, Chamie K, Karsh L, Cinman A, Meads A, Lahiri S, Malinowski M, Gabai N, Raju S, Schoenberg M, Seltzer E, Huang WC. Primary Chemoablation of Low-Grade Intermediate-Risk Nonmuscle-Invasive Bladder Cancer Using UGN-102, a Mitomycin-Containing Reverse Thermal Gel (Optima II): A Phase 2b, Open-Label, Single-Arm Trial. J Urol. 2022 Jan;207(1):61-69. doi: 10.1097/JU.0000000000002186. Epub 2021 Aug 26.
- Derived from this study: PubMed 41074972Prasad SM, Louie MJ, Burger B, Tsurutis V, Ugwuoke N, Strauss-Ayali D. Pharmacokinetics of UGN-102, an investigational mitomycin-containing reverse thermal gel for the treatment of non-muscle invasive bladder cancer. Cancer Chemother Pharmacol. 2025 Oct 11;95(1):100. doi: 10.1007/s00280-025-04821-5.
- Derived from this study: PubMed 35640276Stover AM, Basak R, Mueller D, Lipman R, Teal R, Hilton A, Giannone K, Waheed M, Smith AB. Minimal Patient-Reported Side Effects for a Chemoablative Gel (UGN-102) Used as Frontline Treatment in Adults with Nonmuscle-Invasive Bladder Cancer. J Urol. 2022 Sep;208(3):580-588. doi: 10.1097/JU.0000000000002747. Epub 2022 May 31.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elyse Seltzer, MD study director
UroGen Pharma
19 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing UGN-102 in people with Bladder Cancer and Urothelial Carcinoma, run by UroGen Pharma Ltd. at 19 sites in 2 countries.
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 19 inclusion and 14 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: finished, as of the sponsor's last update on 16 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Israel. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Jul 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Complete Response (CR) Rate for UGN-102 Treatment (time frame: 3 months after the first instillation of UGN-102). 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 NCT03558503, which also lists 3 linked publications.
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 16 Sep 2022.