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NCT03558503: OPTIMA II

A Phase 2b Study of UGN-102 for Low Grade Intermediate Risk Non-Muscle-Invasive Bladder Cancer

Completed Phase 2 Results posted Last updated 16 September 2022
What this trial tests

Phase 2 trial testing UGN-102 in Bladder Cancer in 63 participants. Completed in 21 October 2020.

Timeline
15 October 2018
Primary endpoint
3 January 2020
21 October 2020

Quick facts

Lead sponsorUroGen Pharma Ltd.
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment63
Start date15 October 2018
Primary completion3 January 2020
Estimated completion21 October 2020
Sites19 locations across United States, Israel

Drugs / interventions tested

Conditions studied

Sponsor

UroGen Pharma Ltd. — full company profile →

Who can join

18 and older, any sex, with Bladder Cancer or Urothelial Carcinoma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Complete Response (CR) Rate for UGN-102 Treatment Primary · 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.

GroupValue95% CI
UGN-10265.152.0 – 76.7
Durable Complete Response (DCR) Rate Secondary · 6, 9, and 12 months after the first instillation of UGN-102

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.

6 months (3 months after CR at the 3-month Visit)
GroupValue95% CI
UGN-10295.183.5 – 99.4
9 months (6 months after CR at the 3-month Visit)
GroupValue95% CI
UGN-10273.257.1 – 85.8
12 months (9 months after CR at the 3-month Visit)
GroupValue95% CI
UGN-10261.044.5 – 75.8
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. Secondary · Up to 12 months

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).

Any TEAEs
GroupValue95% CI
UGN-10257
Any study drug or procedure related TEAEs
GroupValue95% CI
UGN-10240
Maximum severity Grade 1
GroupValue95% CI
UGN-10226
Maximum severity Grade 2
GroupValue95% CI
UGN-10224
Maximum severity Grade 3
GroupValue95% CI
UGN-1025
Maximum severity Grade 4
GroupValue95% CI
UGN-1021
Maximum severity Grade 5
GroupValue95% CI
UGN-1021
Any TEAEs leading to treatment discontinuation
GroupValue95% CI
UGN-1026
Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values Secondary · Up to 12 months

The number of patients who met PCS criteria for chemistry or hematology parameters post-baseline will be summarized

Any PCS chemistry value
GroupValue95% CI
UGN-1028
Any PCS hematology value
GroupValue95% CI
UGN-1024
Number of Participants With Post-baseline PCS Vital Signs Values Secondary · Up to 12 months

The number of patients who met PCS criteria for vital signs parameters post-baseline will be summarized

GroupValue95% CI
UGN-1028
Number of Participants With Post-baseline Clinically Significant (CS) Physical Examination Findings Secondary · Up to 12 months

The number of patients with abnormal, CS physical examination findings post-baseline will be summarized

GroupValue95% CI
UGN-1020
Number of Participants With Post-baseline CS Urology-oriented Physical Examination Findings Secondary · Up to 12 months

The number of patients with abnormal, CS urology-oriented physical examination findings post-baseline will be summarized

GroupValue95% CI
UGN-1022
Mitomycin Plasma Concentrations Secondary · 0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-102

Mitomycin plasma concentrations were assessed in a subset of 6 patients treated with UGN-102

Pre-instillation
GroupValue95% CI
UGN-1020± 0
0.5 hours post-instillation
GroupValue95% CI
UGN-1020.75± 0.57
1 hour post-instillation
GroupValue95% CI
UGN-1020.60± 0.42
2 hours post-instillation
GroupValue95% CI
UGN-1020.34± 0.17
3 hours post-instillation
GroupValue95% CI
UGN-1021.88± 3.13
4 hours post-instillation
GroupValue95% CI
UGN-1021.93± 3.49
5 hours post-instillation
GroupValue95% CI
UGN-1020.57± 0.61
6 hours post-instillation
GroupValue95% CI
UGN-1020.32± 0.32
Mitomycin Area Under the Plasma Concentration-time Curve (AUC) Secondary · 0 (pre-instillation) to 6 hours after the first instillation of UGN-102

Mitomycin AUC was assessed in a subset of 6 patients treated with UGN-102

GroupValue95% CI
UGN-1025.69± 7.30
Mitomycin Maximum Plasma Concentration (Cmax) Secondary · 0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-102

Mitomycin Cmax was assessed in a subset of 6 patients treated with UGN-102

GroupValue95% CI
UGN-1022.27± 3.33

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 12 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

UGN-102
Serious: 5/63 (8%)
Deaths: 1/63

Serious adverse events (9 terms)

ReactionSystemUGN-102
Acute myeloid leukemiaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Gastroenteropancreatic neuroendocrine tumor diseaseNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Transitional cell carcinoma metastaticNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Cardiac disorderCardiac disorders
Stress cardiomyopathyCardiac disorders
PneumoniaInfections and infestations
Pneumonia klebsiellaInfections and infestations
HematuriaRenal and urinary disorders
Chronic obstructive pulmonary diseaseRespiratory, thoracic and mediastinal disorders
Other adverse events (12 terms — click to expand)

ReactionSystemUGN-102
DysuriaRenal and urinary disorders
PollakiuriaRenal and urinary disorders
HematuriaRenal and urinary disorders
Micturition urgencyRenal and urinary disorders
Urinary tract infectionRenal and urinary disorders
FatigueGeneral disorders
Urinary incontinenceRenal and urinary disorders
NocturiaRenal and urinary disorders
Urinary retentionRenal and urinary disorders
Pruritus genitalReproductive system and breast disorders
Vulvovaginal discomfortReproductive system and breast disorders
Accidental exposure to productInjury, poisoning and procedural complications

Most-reported serious reactions: Acute myeloid leukemia, Gastroenteropancreatic neuroendocrine tumor disease, Transitional cell carcinoma metastatic, Cardiac disorder, Stress cardiomyopathy, Pneumonia, Pneumonia klebsiella, Hematuria.

Data from ClinicalTrials.gov NCT03558503 adverse events section.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Tuning the Tumor Myeloid Microenvironment to Fight Cancer.
    Jahchan NS, Mujal AM, Pollack JL, Binnewies M, et al · · 2019 · cited 101× · PMID 31402908 · DOI 10.3389/fimmu.2019.01611
  2. Non-muscle-invasive Bladder Cancer: Overview and Contemporary Treatment Landscape of Neoadjuvant Chemoablative Therapies.
    Matulewicz RS, Steinberg GD. · · 2020 · cited 67× · PMID 32760227
  3. 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, Smith AB, et al · · 2022 · cited 32× · PMID 34433303 · DOI 10.1097/ju.0000000000002186
  4. Treatment of Low-grade Intermediate-risk Nonmuscle-invasive Bladder Cancer With UGN-102 ± Transurethral Resection of Bladder Tumor Compared to Transurethral Resection of Bladder Tumor Monotherapy: A Randomized, Controlled, Phase 3 Trial (ATLAS).
    Prasad SM, Huang WC, Shore ND, Hu B, et al · · 2023 · cited 29× · PMID 37548555 · DOI 10.1097/ju.0000000000003645
  5. Hydrogels as advanced drug delivery platforms for cancer immunotherapy: promising innovations and future outlook.
    Mohammadzadeh V, Atapour-Mashhad H, Shahvali S, Salehi B, et al · · 2025 · cited 13× · PMID 40717098 · DOI 10.1186/s12951-025-03613-6
  6. UGN-101 (mitomycin gel): a novel treatment for low-grade upper tract urothelial carcinoma.
    Kokorovic A, Matin SF. · · 2020 · cited 12× · PMID 32670424 · DOI 10.1177/1758835920937950
  7. Minimal Patient-Reported Side Effects for a Chemoablative Gel (UGN-102) Used as Frontline Treatment in Adults with Nonmuscle-Invasive Bladder Cancer.
    Stover AM, Basak R, Mueller D, Lipman R, et al · · 2022 · cited 10× · PMID 35640276 · DOI 10.1097/ju.0000000000002747
  8. Safety Assessment of Ultrasound-Assisted Intravesical Chemotherapy in Normal Dogs: A Pilot Study.
    Sasaki N, Ikenaka Y, Aoshima K, Aoyagi T, et al · · 2022 · cited 9× · PMID 35370726 · DOI 10.3389/fphar.2022.837754

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03558503.

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