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CompletedNCT05136898Updated Sep 19, 2024Results posted

Feasibility of Home Instillation of UGN-102 for Treatment of Low-Grade (LG) Non-Muscle Invasive Bladder Cancer (NMIBC)

A Phase 3 interventional study of UGN-102 in Bladder Cancer, Urothelial Carcinoma and Urothelial Carcinoma Bladder, sponsored by UroGen Pharma Ltd.. Completed at 5 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-09-19.

Sponsored by UroGen Pharma Ltd. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
8
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

This study aims to demonstrate that home instillation of UGN-102 is a feasible alternative to instillation in a clinical setting, which might mitigate patient challenges (logistical, expense, and comfort) when receiving treatment for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

Read the detailed description

The primary objective of this study was to evaluate the feasibility of home instillation of UGN-102 as an alternative to instillation in a clinical setting. Feasibility was assessed by evaluation of safety and tolerability, rate of discontinuation from at home study treatment, and feedback from patients, home health professionals (HHPs), and investigators via standardized questionnaires. The secondary objective of this study was to evaluate the efficacy of UGN-102 for treatment of LG-IR-NMIBC following home instillation. Efficacy was assessed at the 3-month Visit (3 months after the first instillation of UGN-102) by the complete response rate (CRR), defined as the proportion of patients who achieved a complete response (CR) as determined by cystoscopy, for cause biopsy, and urine cytology.

Patients who provided informed consent underwent a Screening Visit to determine eligibility. Screening procedures were to provide evidence of LG-NMIBC and to rule out evidence of high-grade (HG) disease.

Eligible patients were to receive 6 once-weekly intravesical instillations of UGN-102. The first instillation was performed at the investigative site and subsequent instillations were performed at the patient's home by a trained HHP. The HHP was to call the patient 1 to 2 days after each home instillation of UGN-102 to monitor for safety.

At each home instillation visit, the patient and HHP were to complete a feasibility questionnaire. In addition, the patient and investigator were to complete a feasibility questionnaire at the 3-month Visit (end of study) or Early Termination Visit.

Patients were to return to the clinic for the 3-month Visit for determination of response to treatment. Response was determined based on visual evaluation by cystoscopy (white light) (appearance, number, and size of any remaining lesions), interpretation of urine cytology, and for cause biopsy and histopathology of any remaining lesions. Any lesions or suspect tissue were to be biopsied to evaluate for persistence of disease.

Safety was evaluated based on review of adverse events (AEs), laboratory assessments, vital signs, and physical examination.

A patient was considered to have completed the study if the patient completed the 3-month Visit. Following the 3-month Visit, all patients exited the study and continued with standard of care according to their treating physician.

02

Conditions studied

  • Bladder Cancer
  • Urothelial Carcinoma
  • Urothelial Carcinoma Bladder

Keywords

  • Non-muscle invasive bladder cancer
  • Low grade non-muscle invasive bladder cancer
  • Intermediate risk non-muscle invasive bladder cancer
  • NMIBC
  • UGN-102
  • Mitomycin
03

In context

Urinary Bladder Neoplasms

1,649 studies on the registry are indexed under Urinary Bladder Neoplasms; 427 are open to participants now.

This study's enrollment of 8 is below the median of 60 across 1,183 interventional studies indexed under Urinary Bladder Neoplasms.

Browse Urinary Bladder Neoplasms studies →

Lead sponsor

UroGen Pharma Ltd. is the lead sponsor of 19 studies on the registry; 3 are open to participants now.

Of its 6 completed or terminated interventional studies of FDA-regulated products, 5 (83%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and the protocol.
  2. Patient who has newly diagnosed or historic LG-NMIBC (Ta) histologically confirmed by cold cup biopsy at Screening or within 8 weeks before Screening.

  3. Has IR disease, 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 at the initial Screening Visit).
  4. Negative voiding cytology for HG disease within 6 weeks before Screening.

  5. Has adequate organ and bone marrow function as determined by routine laboratory tests:

    • Leukocytes ≥ 3,000/μL (≥ 3 × 10\^9/L).
    • Absolute neutrophil count ≥ 1,500/μL (≥ 1.5 × 10\^9/L).
    • Platelets ≥ 100,000/μL (≥ 100 × 10\^9/L).
    • Hemoglobin ≥ 9.0 g/dL.
    • Total bilirubin ≤ 1.5 × ULN.
    • AST and ALT ≤ 2.5 × ULN.
    • Alkaline phosphatase ≤ 2.5 × ULN.
    • Estimated glomerular filtration rate ≥ 30 mL/min.
  6. Has no evidence of active urinary tract infection at the Screening and baseline visits.
  7. Patient is willing to receive instillations of UGN-102 at home (ie, Treatment Visits 2 to 6) by an appropriately trained home health professional.
  8. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for clinical study participants. Women of childbearing potential (defined as premenopausal women who have not been sterilized), including female patients and female partners of male patients, must be willing to use 2 acceptable forms of effective contraception from first instillation through 6 months post treatment. Acceptable methods of birth control that are considered to have a low failure rate (ie, less than 1% per year) when used consistently and correctly include implants, injections, combined (estrogen/progesterone) oral contraceptives, intrauterine devices (only hormonal), condoms with spermicide, sexual abstinence, or vasectomized partner.
  9. Has an anticipated life expectancy of at least the duration of the trial.

Exclusion criteria

Exclusion Criteria:

  1. Received Bacillus Calmette-Guérin treatment for urothelial carcinoma (UC) within previous 1 year.
  2. History of HG bladder cancer (papillary or carcinoma in situ) in the past 2 years.
  3. Known allergy or sensitivity to mitomycin that in the investigator's opinion cannot be readily managed.
  4. Clinically significant urethral stricture that would preclude passage of a urethral catheter.

  5. History of:

    • Neurogenic bladder.
    • Active urinary retention.
    • Any other condition that would prohibit normal voiding.
  6. Past or current T1 UC, muscle invasive UC (ie, T2, T3, T4), metastatic UC, or concurrent upper tract UC.
  7. 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.
  8. History of prior treatment with an intravesical chemotherapeutic agent in the past 2 years except for a single dose of chemotherapy immediately after any previous TURBT.
  9. Has participated in a study with an investigational agent or device within 30 days of enrollment.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
8 participants (actual)

Study arms

  • Experimental
    UGN-102

    Patients will receive 6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin). Treatment Visit 1 will occur at the investigative site and instillation will be performed by a qualified physician. Treatment Visits 2 to 6 will occur at the patient's home and instillation will be performed by a properly trained and qualified home health professional.

    Drug: UGN-102

Interventions

  • DrugUGN-102

    Drug pages: Mitomycin · SR Mitomycin 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 under chilled conditions, with subsequent conversion to a semi-solid gel depot following instillation into the bladder.

    Also known as: UGN-102 (mitomycin) for intravesical solution

06

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest.

    The number of patients with each type of event will be summarized. TEAEs were defined as adverse events (AEs) that occurred on or after the day of the first instillation of UGN-102 or pre-treatment AEs that worsened during the study.

    Time frame: Up to 3 months

  2. Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values

    PCS laboratory criteria were defined for the following parameters: Hematology * Hemoglobin \< 0.8 × lower limit of normal and \> 20% decrease from baseline or \> 1.3 × upper limit of normal (ULN) and \> 30% increase from baseline. * Leukocytes ≤ 2.8 or ≥ 16 × 10\^3/µL. * Lymphocytes \< 0.5 or \> 20 × 10\^3/µL. * Neutrophils \< 1.0 × 10\^3/µL. * Platelets \< 75 or ≥ 700 × 10\^3/µL. Chemistry * Creatinine \> 2.2 mg/dL. * Sodium ≤ 130 or \> 150 mEq/L. * Potassium \< 3.0 or \> 5.5 mEq/L. * Total bilirubin \> 1.5 × ULN. * Alanine aminotransferase (ALT) \> 3 × ULN. * Aspartate aminotransferase (AST) \> 3 × ULN. * Gamma glutamyl transferase \> 2.5 × ULN.

    Time frame: Up to 3 months

  3. Composite Score for the Post-instillation Patient Questionnaire

    Patients were asked to rate their experience receiving UGN-102 at home after each instillation was completed. A total of 5 domains (8 questions) were assessed: * Comfort (3 questions, possible range from 3 "least favorable" to 12 "most favorable"). * Safety/concerns (1 question, possible range from 1 "least favorable" to 4 "most favorable"). * Communication (2 questions, possible range from 2 "least favorable" to 8 "most favorable"). * Preference compared to office instillation (1 question, possible range from 1 "least favorable" to 4 "most favorable"). * Overall experience/satisfaction (1 question, possible range from 1 "least favorable" to 4 "most favorable"). A composite score (sum of all 8 question scores) was also calculated (possible range from 8 "least favorable" to 32 "most favorable").

    Time frame: Up to 5 weeks

  4. Responses to the Post-instillation Home Health Professional Questionnaire

    Home health professionals (HHPs) were asked to share their experience administering UGN-102 at the patient's home after each instillation was completed. A total of 4 domains (4 questions) were assessed: * Comfort - Were you comfortable performing the instillation at this patient's home, yes (most favorable) or no (least favorable)? * Difficulty - Was it difficult to perform the instillation at this patient's home, yes (least favorable) or no (most favorable)? * Safety/concerns - Did you have any concerns performing the instillation in the home setting, yes (least favorable) or no (most favorable)? * Adequate support - Did you have sufficient support performing the instillation in the home setting, yes (most favorable) or no (least favorable)? The number of patients whose HHP had a "most favorable" response to all 4 questions is summarized for each home instillation.

    Time frame: Up to 5 weeks

  5. Responses to End of Study Patient and Investigator Questionnaires

    Patient recommendations regarding home instillation of UGN-102 were collected at the 3-month Visit or Early Termination Visit. Two questions were asked: * Will you recommend home instillations of UGN-102 for other patients with NMIBC, yes (most favorable) or no (least favorable)? * Will you recommend home instillations of UGN-102 instead of having transurethral resection of bladder tumor (TURBT) for other patients with NMIBC, yes (most favorable) or no (least favorable)? The number of patients who had a "most favorable" response is summarized by question. In addition, investigators were asked if the experience of having their patient receive instillations at home was less difficult (most favorable), more difficult (least favorable), or not different than having them receive instillations in the office. All responses are summarized.

    Time frame: 3 months

Secondary outcomes

  1. Complete Response Rate (CRR)

    CRR is defined as the percentage of patients who achieved a complete response (CR) at the 3-month Visit. A patient was considered a CR if there was no detectable disease in the bladder based on visual observation (white light cystoscopy), biopsy of remaining lesions, if applicable, and voiding urine cytology.

    Time frame: 3 months

07

Results

Posted Sep 19, 2024

Participant flow

Participant flow — Overall Study
MilestoneUGN-102
Started8
Completed6
Not completed2
Withdrew: Adverse event2

Outcome measures

PrimaryNumber of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest.

The number of patients with each type of event will be summarized. TEAEs were defined as adverse events (AEs) that occurred on or after the day of the first instillation of UGN-102 or pre-treatment AEs that worsened during the study.

Time frame:
Up to 3 months
Reported as:
Count of participants · Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest.
ParticipantsUGN-102
Any TEAEs8
Any serious TEAEs3
Any TEAEs leading to discontinuation2
Any TEAEs of special interest7
PrimaryNumber of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values

PCS laboratory criteria were defined for the following parameters: Hematology * Hemoglobin \< 0.8 × lower limit of normal and \> 20% decrease from baseline or \> 1.3 × upper limit of normal (ULN) and \> 30% increase from baseline. * Leukocytes ≤ 2.8 or ≥ 16 × 10\^3/µL. * Lymphocytes \< 0.5 or \> 20 × 10\^3/µL. * Neutrophils \< 1.0 × 10\^3/µL. * Platelets \< 75 or ≥ 700 × 10\^3/µL. Chemistry * Creatinine \> 2.2 mg/dL. * Sodium ≤ 130 or \> 150 mEq/L. * Potassium \< 3.0 or \> 5.5 mEq/L. * Total bilirubin \> 1.5 × ULN. * Alanine aminotransferase (ALT) \> 3 × ULN. * Aspartate aminotransferase (AST) \> 3 × ULN. * Gamma glutamyl transferase \> 2.5 × ULN.

Time frame:
Up to 3 months
Reported as:
Count of participants · Participants
Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values
ParticipantsUGN-102
Hematology0
Chemistry0
PrimaryComposite Score for the Post-instillation Patient Questionnaire

Patients were asked to rate their experience receiving UGN-102 at home after each instillation was completed. A total of 5 domains (8 questions) were assessed: * Comfort (3 questions, possible range from 3 "least favorable" to 12 "most favorable"). * Safety/concerns (1 question, possible range from 1 "least favorable" to 4 "most favorable"). * Communication (2 questions, possible range from 2 "least favorable" to 8 "most favorable"). * Preference compared to office instillation (1 question, possible range from 1 "least favorable" to 4 "most favorable"). * Overall experience/satisfaction (1 question, possible range from 1 "least favorable" to 4 "most favorable"). A composite score (sum of all 8 question scores) was also calculated (possible range from 8 "least favorable" to 32 "most favorable").

Time frame:
Up to 5 weeks
Reported as:
Median · score on a scale
Composite Score for the Post-instillation Patient Questionnaire
score on a scaleUGN-102
Post 2nd instillation32 (26 to 32)
Post 3rd instillation32 (30 to 32)
Post 4th instillation32 (30 to 32)
Post 5th instillation32 (30 to 32)
Post 6th instillation32 (29 to 32)
SecondaryComplete Response Rate (CRR)

CRR is defined as the percentage of patients who achieved a complete response (CR) at the 3-month Visit. A patient was considered a CR if there was no detectable disease in the bladder based on visual observation (white light cystoscopy), biopsy of remaining lesions, if applicable, and voiding urine cytology.

Time frame:
3 months
Reported as:
Number · percentage of participants
Complete Response Rate (CRR)
percentage of participantsUGN-102
Complete Response Rate (CRR)75.0 (34.9 to 96.8)
PrimaryResponses to the Post-instillation Home Health Professional Questionnaire

Home health professionals (HHPs) were asked to share their experience administering UGN-102 at the patient's home after each instillation was completed. A total of 4 domains (4 questions) were assessed: * Comfort - Were you comfortable performing the instillation at this patient's home, yes (most favorable) or no (least favorable)? * Difficulty - Was it difficult to perform the instillation at this patient's home, yes (least favorable) or no (most favorable)? * Safety/concerns - Did you have any concerns performing the instillation in the home setting, yes (least favorable) or no (most favorable)? * Adequate support - Did you have sufficient support performing the instillation in the home setting, yes (most favorable) or no (least favorable)? The number of patients whose HHP had a "most favorable" response to all 4 questions is summarized for each home instillation.

Time frame:
Up to 5 weeks
Reported as:
Count of participants · Participants
Responses to the Post-instillation Home Health Professional Questionnaire
ParticipantsUGN-102
Post 2nd instillation8
Post 3rd instillation7
Post 4th instillation7
Post 5th instillation7
Post 6th instillation5
PrimaryResponses to End of Study Patient and Investigator Questionnaires

Patient recommendations regarding home instillation of UGN-102 were collected at the 3-month Visit or Early Termination Visit. Two questions were asked: * Will you recommend home instillations of UGN-102 for other patients with NMIBC, yes (most favorable) or no (least favorable)? * Will you recommend home instillations of UGN-102 instead of having transurethral resection of bladder tumor (TURBT) for other patients with NMIBC, yes (most favorable) or no (least favorable)? The number of patients who had a "most favorable" response is summarized by question. In addition, investigators were asked if the experience of having their patient receive instillations at home was less difficult (most favorable), more difficult (least favorable), or not different than having them receive instillations in the office. All responses are summarized.

Time frame:
3 months
Reported as:
Count of participants · Participants
Responses to End of Study Patient and Investigator Questionnaires
ParticipantsUGN-102
Recommend home instillations of UGN-102 for other NMIBC patients = yes5
Recommend home instillations of UGN-102 instead of having TURBT = yes5
Having patient receive instillations at home is less difficult than in office0
Having patient receive instillations at home is more difficult than in office1
Having patient receive instillations at home is not different than in office5

Adverse events

Collected over Up to 3 months. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
UGN-1020/8 (0%)3/8 (37.5%)8/8 (100%)
Most frequent serious events
Most frequent serious events
EventUGN-102
Pleural effusionRespiratory, thoracic and mediastinal disorders1/8
Cerebrovascular accidentNervous system disorders1/8
Embolic strokeNervous system disorders1/8
Intracranial aneurysmNervous system disorders1/8
Most frequent other events
Showing 10 of 24
Most frequent other events
EventUGN-102
DysuriaRenal and urinary disorders2/8
FatigueGeneral disorders2/8
Hypertonic bladderRenal and urinary disorders2/8
Urinary tract infectionInfections and infestations2/8
AnxietyPsychiatric disorders1/8
Atrial flutterCardiac disorders1/8
Benign prostatic hyperplasiaReproductive system and breast disorders1/8
Carotid artery stenosisNervous system disorders1/8
DeliriumPsychiatric disorders1/8
DepressionPsychiatric disorders1/8

Baseline characteristics

All patients who received any dose of UGN-102.

Age, Categorical
Age, Categorical(Participants)UGN-102
<=18 years0
Between 18 and 65 years2
>=65 years6
Age, Continuous
Age, Continuous(years)UGN-102
Median75 (55 to 84)
Sex: Female, Male
Sex: Female, Male(Participants)UGN-102
Female3
Male5
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)UGN-102
Hispanic or Latino0
Not Hispanic or Latino8
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)UGN-102
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American0
White8
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(Participants)UGN-102
United States8
Previous Low-grade (LG) Non-muscle Invasive Bladder Cancer (NMIBC) Episode(s)
Previous Low-grade (LG) Non-muscle Invasive Bladder Cancer (NMIBC) Episode(s)(Participants)UGN-102
Yes8
No0
Previous LG-NMIBC Episode(s) Within 1 Year
Previous LG-NMIBC Episode(s) Within 1 Year(Participants)UGN-102
Yes6
No2

4 further baseline measures are reported on the registry.

08

Study locations

5 sites
  • Urology Centers of Alabama (UCA)
    Homewood, Alabama 35209, United States
  • Indiana University School of Medicine
    Indianapolis, Indiana 46202, United States
  • Medication Management
    Greensboro, North Carolina 27408, United States
  • Urology Associates, P.C.
    Nashville, Tennessee 37209, United States
  • Virginia Urology
    Richmond, Virginia 23235, United States
09

References and documents

Study documents

  • Study protocol · Jun 11, 2021
  • Statistical analysis plan · Jul 20, 2023

Documents are hosted by the registry — open the source record to download them.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Nov 2021 to primary completion in Feb 2023, 14 months.

Results were first posted in Sep 2024, 1 year 7 months after primary completion.

Of 72 phase 3 interventional studies under Urinary Bladder Neoplasms, half reached primary completion within 66 months of starting (the median), counting those still running; a quarter within 40 and three quarters within 92. This study's 14 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Sep 23, 2021
First posted
Nov 30, 2021
StartActual
Nov 30, 2021
Primary completionActual
Feb 2, 2023
CompletionActual
Feb 2, 2023
Results first posted
Sep 19, 2024
Last update
Sep 19, 2024
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Sep 19, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT05136898
Lead sponsor
UroGen Pharma Ltd.
Responsible party
Sponsor
First posted
Nov 30, 2021
Start date
Nov 30, 2021
Primary completion
Feb 2, 2023
Completion
Feb 2, 2023
Results posted
Sep 19, 2024
Last update
Sep 19, 2024

Study contacts

Sunil Raju, MBBS
study director · UroGen Pharma

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drugs tested here: Mitomycin · SR Mitomycin Intravesical Solution

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