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

NCT05136898 | Completed Phase 3 Results FDA Drug

• 1 other identifier: BL010
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 5

Brief 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).

Conditions

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

Outcome Measures

Primary Outcomes (5)

• 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.

  Up to 3 months

• 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.

  Up to 3 months

• 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").

  Up to 5 weeks

• 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.

  Up to 5 weeks

• 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.

  3 months

Secondary Outcomes (1)

• Complete Response Rate (CRR)

  3 months

Study Arms (1)

UGN-102

EXPERIMENTAL

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

UGN-102 DRUG

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

UGN-102

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• UroGen Pharma Ltd.: lead

Study Sites (5)

Urology Centers of Alabama (UCA)

Homewood, Alabama, 35209, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

Medication Management

Greensboro, North Carolina, 27408, United States

Location

Urology Associates, P.C.

Nashville, Tennessee, 37209, United States

Location

Virginia Urology

Richmond, Virginia, 23235, United States

Location

MeSH Terms

Conditions

Urinary Bladder Neoplasms; Carcinoma, Transitional Cell; Non-Muscle Invasive Bladder Neoplasms

Interventions

Mitomycin

Condition Hierarchy (Ancestors)

Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type

Intervention Hierarchy (Ancestors)

Mitomycins; Indolequinones; Quinones; Organic Chemicals; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Head of Clinical Development
• Organization: UroGen Pharma

info@urogen.com

+1 855-987-6436

Study Officials

• Sunil Raju, MBBS

  UroGen Pharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2021
• First Posted: November 30, 2021
• Study Start: November 30, 2021
• Primary Completion: February 2, 2023
• Study Completion: February 2, 2023
• Last Updated: September 19, 2024
• Results First Posted: September 19, 2024

Record last verified: 2024-09

Locations

US (5)