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Bladder CancerPhase 3NCT05136898

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

A Phase 3 study testing UGN-102 in people with Bladder Cancer and Urothelial Carcinoma, run by UroGen Pharma Ltd. at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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 more about this study

Who is it for?
Ages 18 years and older
What is being tested?
UGN-102
Could I receive a placebo?
No, everyone receives an active treatment
How many people
8 participants
Where
5 sitesUnited States
Study dates
30 Nov 2021to 2 Feb 2023
Phase
Phase 3
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

  • Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and the protocol.
  • Patient who has newly diagnosed or historic LG-NMIBC (Ta) histologically confirmed by cold cup biopsy at Screening or within 8 weeks before Screening.
  • 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).
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  • Negative voiding cytology for HG disease within 6 weeks before Screening.
  • 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.
  • Has no evidence of active urinary tract infection at the Screening and baseline visits.
  • Patient is willing to receive instillations of UGN-102 at home (ie, Treatment Visits 2 to 6) by an appropriately trained home health professional.
  • 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.
  • Has an anticipated life expectancy of at least the duration of the trial.

Who cannot join

  • Received Bacillus Calmette-Guérin treatment for urothelial carcinoma (UC) within previous 1 year.
  • History of HG bladder cancer (papillary or carcinoma in situ) in the past 2 years.
  • Known allergy or sensitivity to mitomycin that in the investigator's opinion cannot be readily managed.
  • Clinically significant urethral stricture that would preclude passage of a urethral catheter.
  • History of:
  • Neurogenic bladder.
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  • Active urinary retention.
  • Any other condition that would prohibit normal voiding.
  • Past or current T1 UC, muscle invasive UC (ie, T2, T3, T4), metastatic UC, or concurrent upper tract UC.
  • 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.
  • 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.
  • Has participated in a study with an investigational agent or device within 30 days of enrollment.

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites
  • Medication Management

    Greensboro, North Carolina, 27408

  • Urology Centers of Alabama (UCA)

    Homewood, Alabama, 35209

  • Indiana University School of Medicine

    Indianapolis, Indiana, 46202

  • Urology Associates, P.C.

    Nashville, Tennessee, 37209

  • Virginia Urology

    Richmond, Virginia, 23235

Other trials in United States

About this study

Official title: A Phase 3b, Open-Label, Single-Arm, Multicenter Study to Assess the Feasibility of Home Instillation of UGN-102 for Treatment of Patients With Low-Grade (LG) Non-Muscle Invasive Bladder Cancer (NMIBC) at Intermediate-Risk (IR) of Recurrence

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.

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 under chilled conditions, with subsequent conversion to a semi-solid gel depot following instillation into the bladder.

Study groups in NCT05136898
Study groupRoleWhat they receive
UGN-102ExperimentalPatients 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.
Receives: Drug: UGN-102

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 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.

    Measured: 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").

    Measured: 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.

    Measured: 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.

    Measured: 3 months

Secondary outcomes (1)
  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.

    Measured: 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Nov 2021
  2. Study start30 Nov 2021
  3. Primary completion2 Feb 2023
  4. Study completion2 Feb 2023

Resources

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 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
8 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05136898
Sponsor's protocol ID
BL010
Status last verified
Sep 2024
First submitted
23 Sep 2021
Last updated
19 Sep 2024
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing UGN-102 in people with Bladder Cancer and Urothelial Carcinoma, run by UroGen Pharma Ltd. at 5 sites in United States.

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 20 inclusion and 12 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 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 19 Sep 2024). 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?

Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest. (time frame: Up to 3 months). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
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 19 Sep 2024.

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