UGN-103 for Bladder Cancer

(UTOPIA Trial)

Recruiting at 13 trial locations

Overseen By Renee Sumlin

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: UroGen Pharma Ltd.

Must not be taking: BCG treatment

Disqualifiers: HG bladder cancer, Neurogenic bladder, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

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 of patients with low-grade non-muscle invasive bladder cancer (LG-NMIBC).

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.

What data supports the effectiveness of the drug UGN-103 for bladder cancer?

Research on similar treatments like UGN-101 and UGN-102, which are also mitomycin-containing gels, shows promising results in treating low-grade urothelial carcinomas by increasing the time the drug stays in contact with the cancer cells, leading to better outcomes. These treatments have been effective in managing similar types of cancer, suggesting potential effectiveness for UGN-103 in bladder cancer. ^{1 2 3 4 5}

Is UGN-103 safe for humans?

Research on similar treatments like UGN-101 and UGN-102, which also use mitomycin in a gel form, shows promising safety results in clinical trials for treating certain types of bladder and upper tract cancers. These studies suggest that the gel formulation is generally safe for human use. ^{1 2 3 5 6}

What makes the drug UGN-103 unique for treating bladder cancer?

UGN-103 is unique because it uses a special gel that turns from liquid to gel at body temperature, allowing it to stick to the bladder lining and slowly release the cancer-fighting drug mitomycin, which may improve its effectiveness compared to traditional treatments that wash out quickly. ^{1 2 3 5 7}

Research Team

Sunil Raju, MBBS

Principal Investigator

UroGen Pharma

Eligibility Criteria

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.

Trial Timeline

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)

Treatment Details

Interventions

• UGN-103 (Chemotherapy)

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.

Participant Groups

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+

Findings from Research

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]

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]

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 (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]

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]

References

1. England 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. [2022]

2. United States 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. [2022]

3. United States 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. [2022]

4. United States 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. [2023]

5. England pubmed.ncbi.nlm.nih.gov

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

6. United States pubmed.ncbi.nlm.nih.gov

Sustained-release Formulation of Mitomycin C to the Upper Urinary Tract Using a Thermosensitive Polymer: A Preclinical Study. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Antegrade Instillation of UGN-101 (Mitomycin for Pyelocalyceal Solution) for Low-Grade Upper Tract Urothelial Carcinoma: Initial Clinical Experience. [2022]