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).
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
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.
Urology Centers of Alabama (UCA)
Homewood, Alabama, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
Medication Management
Greensboro, North Carolina, United States
Urology Associates, P.C.
Nashville, Tennessee, United States
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
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
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
Responses to the Post-instillation Home Health Professional Questionnaire
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Virginia Urology
Richmond, Virginia, United States
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
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
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