UGN-103 for Bladder Cancer

(UTOPIA Trial)

Recruiting at 13 trial locations

Overseen By Renee Sumlin

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: UroGen Pharma Ltd.

Must not be taking: BCG treatment

Disqualifiers: HG bladder cancer, Neurogenic bladder, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 10

Duration: 6 weeks

92 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called UGN-103, a form of mitomycin gel, for individuals with non-muscle invasive bladder cancer. The main goal is to assess the safety and effectiveness of this treatment when administered directly into the bladder. Participants will receive the treatment once a week for six weeks. It may suit those who have experienced a recurrence of bladder cancer with multiple or large tumors. As a Phase 3 trial, this study represents the final step before FDA approval, providing participants an opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss your specific medications with the trial team to get a clear answer.

Is there any evidence suggesting that UGN-103 is likely to be safe for humans?

Research has shown that UGN-103 is generally well-tolerated by patients. This treatment uses a special gel to slowly release medicine in the bladder and is an updated version of UGN-102.

In similar treatments using mitomycin, the active ingredient in UGN-103, about 12% of patients experienced serious side effects, including rare cases of difficulty urinating and narrowing of the urethra. However, these side effects are uncommon.

UGN-103 is currently in a Phase 3 trial, indicating that earlier studies found it safe enough for broader testing. This suggests that most people handle it well, though some may still experience side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for bladder cancer?

Researchers are excited about UGN-103 for bladder cancer because it offers a unique delivery method that could enhance treatment effectiveness. Unlike many standard treatments which often involve surgical procedures or systemic chemotherapy, UGN-103 is administered directly into the bladder. This approach uses mitomycin, a chemotherapy drug, and delivers it precisely where it's needed, potentially minimizing side effects and improving patient outcomes. Additionally, the treatment is designed to be completed in just six weeks, offering a more convenient option compared to some traditional therapies that require more prolonged treatment schedules.

What evidence suggests that UGN-103 might be an effective treatment for bladder cancer?

Research has shown that UGN-103, an updated version of UGN-102, may effectively treat low-grade non-muscle invasive bladder cancer (LG-NMIBC). In this trial, participants will receive UGN-103, which uses mitomycin as its main ingredient. Studies have found that mitomycin led to a complete response in about 79.6% of patients three months after treatment. Furthermore, 72.2% of patients who initially responded well continued to show positive results after 24 months. These findings suggest that UGN-103 could be a promising treatment option for people with this type of bladder cancer. ^{2 4 5 6 7}

Who Is on the Research Team?

Sunil Raju, MBBS

Principal Investigator

UroGen Pharma

Are You a Good Fit for This Trial?

This trial is for patients with a type of bladder cancer called low-grade non-muscle invasive bladder cancer (LG-NMIBC). Participants must have had at least one prior episode, may have multiple tumors or a tumor larger than 3 cm, and should show signs of early or frequent recurrence. They need normal blood counts and no high-grade disease in recent tests. Patients must consent to follow the study rules.

Inclusion Criteria

I have signed and agreed to all the terms in the consent form.

My bladder cancer was confirmed with a specific biopsy recently.

I have had bladder cancer before, but not including my current diagnosis.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive UGN-103 instilled in the urinary bladder once weekly for 6 weeks

6 weeks

6 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with evaluations every 3 months

12 months

4 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• UGN-103

Trial Overview The trial is testing UGN-103, which is an updated version of UGN-102. It's given as a treatment directly into the bladder for those with LG-NMIBC. The goal is to see how effective and safe this new formulation is when used by itself in patients.

How Is the Trial Designed?

Group I: UGN-103 Experimental Treatment 1 Intervention

Patients will receive UGN-103 (75 mg mitomycin) once weekly for 6 weeks (a total of 6 doses).

Who Is Running the Clinical Trial?

UroGen Pharma Ltd.

Lead Sponsor

Trials

20

Recruited

1,400+

Published Research Related to This Trial

In a study of 52 patients treated with UGN-101 after complete endoscopic ablation of upper tract urothelial carcinoma, 69% showed no visible disease at the first evaluation, indicating promising efficacy as an adjuvant therapy.

UGN-101 treatment after ablation resulted in fewer disease recurrences compared to primary chemoablative therapy, and while 19% of patients experienced ureteral stenosis, this was not significantly higher than the 29% seen in those receiving chemoablation, suggesting a potentially better safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Mitomycin Gel (UGN-101) as an Adjuvant Therapy After Complete Endoscopic Management of Upper Tract Urothelial Carcinoma. Labbate, C., Woldu, S., Murray, K., et al.[2023]

UGN-101, a novel drug using a sustained-release hydrogel formulation of mitomycin-C, has shown promising results in treating low-grade upper tract urothelial carcinomas (UTUCs), with a 59% complete response rate in the OLYMPUS trial.

The treatment demonstrated a good safety profile, with 68% of patients experiencing only mild to moderate urinary adverse events, suggesting it could become a standard treatment option for UTUCs at risk of relapse.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An evaluation of UGN-101, a sustained-release hydrogel polymer-based formulation containing mitomycin-C, for the treatment of upper urothelial carcinomas. Porta, C., Giannatempo, P., Rizzo, M., et al.[2022]

UGN-101, a chemoablative treatment for low-grade upper tract urothelial carcinoma, was successfully administered antegrade via nephrostomy in 8 patients, with a complete response observed in 4 of them during follow-up.

While there were some adverse events reported, including one asymptomatic ureteral stricture, the overall safety profile appears manageable, suggesting that this method could enhance patient comfort and treatment logistics.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antegrade Instillation of UGN-101 (Mitomycin for Pyelocalyceal Solution) for Low-Grade Upper Tract Urothelial Carcinoma: Initial Clinical Experience. Rosen, GH., Nallani, A., Muzzey, C., et al.[2022]

In a phase 2b trial involving 63 patients with low-grade intermediate-risk nonmuscle-invasive bladder cancer, 65% achieved a complete response after treatment with UGN-102, a mitomycin-containing gel, indicating its efficacy as a non-surgical treatment option.

The treatment demonstrated sustained durability, with 73% of patients remaining disease-free 9 months after achieving a complete response, suggesting that UGN-102 could reduce the need for repeated surgeries in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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. Chevli, KK., Shore, ND., Trainer, A., et al.[2022]

In a phase 3 trial involving 71 patients with low-grade upper tract urothelial carcinoma, 58% achieved a complete response to UGN-101 treatment, with 56% maintaining this response after 12 months, indicating its long-term efficacy.

UGN-101 demonstrated a durable response, with an estimated 82% durability at 12 months, although the treatment was associated with a notable incidence of ureteric stenosis (44%), suggesting careful monitoring for side effects is necessary.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Durability of Response to Primary Chemoablation of Low-Grade Upper Tract Urothelial Carcinoma Using UGN-101, a Mitomycin-Containing Reverse Thermal Gel: OLYMPUS Trial Final Report. Matin, SF., Pierorazio, PM., Kleinmann, N., et al.[2022]

UGN-101 (Jelmyto™) is a novel formulation of mitomycin C that uses a hydrogel to enhance urinary dwell time, improving the efficacy of treatment for low-grade upper tract urothelial carcinoma (UTUC).

Preliminary results from the phase III OLYMPUS study indicate that UGN-101 is an effective chemo-ablative agent for low-grade UTUC, leading to its recent FDA approval as the first treatment for this condition, which could significantly improve patient care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

UGN-101 (mitomycin gel): a novel treatment for low-grade upper tract urothelial carcinoma. Kokorovic, A., Matin, SF.[2020]

MitoGel, a novel formulation of mitomycin C, was found to be safe and feasible for instillation into the upper urinary tract in a study involving 23 pigs, with no serious adverse effects such as urinary obstruction or acute kidney injury observed.

The gel remained visible in the urinary system for 4-6 hours and maintained plasma levels of mitomycin C within safe limits, suggesting its potential for effective treatment of upper tract urothelial carcinoma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sustained-release Formulation of Mitomycin C to the Upper Urinary Tract Using a Thermosensitive Polymer: A Preclinical Study. Donin, NM., Duarte, S., Lenis, AT., et al.[2022]

Citations

1. investors.urogen.com investors.urogen.com/news-releases/news-release-details/urogen-announces-completion-enrollment-phase-3-utopia-clinical

UroGen Announces Completion of Enrollment in the Phase ... All enrolled patients receive 75 mg of UGN-103 via intravesical instillation once weekly for six weeks. The primary endpoint is complete ...

2. urologytimes.com urologytimes.com/view/updated-evision-data-show-sustained-efficacy-of-mitomycin-intravesical-solution-for-lg-ir-nmibc

Updated ENVISION data show sustained efficacy of ... Treatment with mitomycin for intravesical solution led to a 24-month DOR of 72.2% among patients who achieved an initial complete response.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06331299

A Phase 3 Study of UGN-103 for Treatment of Patients With ... This Phase 3, single-arm, multicenter study will evaluate the efficacy and safety of UGN-103, a novel formulation of UGN-102, instilled in the urinary bladder ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12535501/

Mitomycin Intravesical Solution: First Approval - PMC The complete response rate (CRR; primary endpoint) at 3 months after initial treatment in the ITT population was 79.6% (191/240 patients). At 15 ...

5. investors.urogen.com investors.urogen.com/news-releases/news-release-details/zusduritm-clinical-review-published-reviews-urologytm-highlights

ZUSDURI™ Clinical Review Published in ... No Adverse Impact from Patient-Reported Outcomes: Clinical trials show that ZUSDURI did not adversely affect functionality, symptom burden, and ...

6. investors.urogen.com investors.urogen.com/news-releases/news-release-details/first-patient-dosed-phase-3-clinical-trial-ugn-103-next

First Patient Dosed in Phase 3 Clinical Trial of UGN-103, a ... UGN-103 will utilize UroGen's proprietary RTGel technology, a sustained release, hydrogel-based formulation designed to enable longer exposure of bladder ...

7. fda.gov fda.gov/drugs/resources-information-approved-drugs/fda-approves-mitomycin-intravesical-solution-recurrent-low-grade-intermediate-risk-non-muscle

FDA approves mitomycin intravesical solution for recurrent ... Serious adverse reactions occurred in 12% of patients, including urinary retention (0.8%) and urethral stenosis (0.4%). A fatal adverse ...