Trial Outcomes & Findings for Feasibility of Home Instillation of UGN-102 for Treatment of Low-Grade (LG) Non-Muscle Invasive Bladder Cancer (NMIBC) (NCT NCT05136898)

NCT ID: NCT05136898

Last Updated: 2024-09-19

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

8 participants

Primary outcome timeframe

Up to 3 months

Results posted on

2024-09-19

Participant Flow

Participant milestones

Participant milestones
Measure
UGN-102
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Overall Study
STARTED
8
Overall Study
COMPLETED
6
Overall Study
NOT COMPLETED
2

Reasons for withdrawal

Reasons for withdrawal
Measure
UGN-102
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Overall Study
Adverse Event
2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
UGN-102
n=8 Participants
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
2 Participants
n=5 Participants
Age, Categorical
>=65 years
6 Participants
n=5 Participants
Age, Continuous
75 years
n=5 Participants
Sex: Female, Male
Female
3 Participants
n=5 Participants
Sex: Female, Male
Male
5 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
8 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
8 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
United States
8 Participants
n=5 Participants
Previous Low-grade (LG) Non-muscle Invasive Bladder Cancer (NMIBC) Episode(s)
Yes
8 Participants
n=5 Participants
Previous Low-grade (LG) Non-muscle Invasive Bladder Cancer (NMIBC) Episode(s)
No
0 Participants
n=5 Participants
Previous LG-NMIBC Episode(s) Within 1 Year
Yes
6 Participants
n=5 Participants
Previous LG-NMIBC Episode(s) Within 1 Year
No
2 Participants
n=5 Participants
Prior Transurethral Resection of Bladder Tumor (TURBT)
Yes
8 Participants
n=5 Participants
Prior Transurethral Resection of Bladder Tumor (TURBT)
No
0 Participants
n=5 Participants
Tumor Burden
≤ 3 cm
4 Participants
n=5 Participants
Tumor Burden
> 3 cm
4 Participants
n=5 Participants
Tumor Count
Single
3 Participants
n=5 Participants
Tumor Count
Multiple
5 Participants
n=5 Participants
Smoking History
Yes
5 Participants
n=5 Participants
Smoking History
No
3 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Up to 3 months

Population: All patients who received any dose of UGN-102.

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.

Outcome measures

Outcome measures
Measure
UGN-102
n=8 Participants
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest.
Any serious TEAEs
3 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest.
Any TEAEs leading to discontinuation
2 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest.
Any TEAEs of special interest
7 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest.
Any TEAEs
8 Participants

PRIMARY outcome

Timeframe: Up to 3 months

Population: All patients who received any dose of UGN-102 and who had a post-baseline laboratory value.

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.

Outcome measures

Outcome measures
Measure
UGN-102
n=8 Participants
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values
Hematology
0 Participants
Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values
Chemistry
0 Participants

PRIMARY outcome

Timeframe: Up to 5 weeks

Population: All patients who received any dose of UGN-102.

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").

Outcome measures

Outcome measures
Measure
UGN-102
n=8 Participants
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Composite Score for the Post-instillation Patient Questionnaire
Post 3rd instillation
32 score on a scale
Interval 30.0 to 32.0
Composite Score for the Post-instillation Patient Questionnaire
Post 5th instillation
32 score on a scale
Interval 30.0 to 32.0
Composite Score for the Post-instillation Patient Questionnaire
Post 2nd instillation
32 score on a scale
Interval 26.0 to 32.0
Composite Score for the Post-instillation Patient Questionnaire
Post 4th instillation
32 score on a scale
Interval 30.0 to 32.0
Composite Score for the Post-instillation Patient Questionnaire
Post 6th instillation
32 score on a scale
Interval 29.0 to 32.0

PRIMARY outcome

Timeframe: Up to 5 weeks

Population: All patients who received any dose of UGN-102.

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.

Outcome measures

Outcome measures
Measure
UGN-102
n=8 Participants
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Responses to the Post-instillation Home Health Professional Questionnaire
Post 5th instillation
7 Participants
Responses to the Post-instillation Home Health Professional Questionnaire
Post 6th instillation
5 Participants
Responses to the Post-instillation Home Health Professional Questionnaire
Post 2nd instillation
8 Participants
Responses to the Post-instillation Home Health Professional Questionnaire
Post 3rd instillation
7 Participants
Responses to the Post-instillation Home Health Professional Questionnaire
Post 4th instillation
7 Participants

PRIMARY outcome

Timeframe: 3 months

Population: All patients who received any dose of UGN-102 and who completed the end of study questionnaire.

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.

Outcome measures

Outcome measures
Measure
UGN-102
n=6 Participants
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Responses to End of Study Patient and Investigator Questionnaires
Recommend home instillations of UGN-102 instead of having TURBT = yes
5 Participants
Responses to End of Study Patient and Investigator Questionnaires
Having patient receive instillations at home is less difficult than in office
0 Participants
Responses to End of Study Patient and Investigator Questionnaires
Having patient receive instillations at home is more difficult than in office
1 Participants
Responses to End of Study Patient and Investigator Questionnaires
Having patient receive instillations at home is not different than in office
5 Participants
Responses to End of Study Patient and Investigator Questionnaires
Recommend home instillations of UGN-102 for other NMIBC patients = yes
5 Participants

SECONDARY outcome

Timeframe: 3 months

Population: All patients who received any dose of UGN-102.

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.

Outcome measures

Outcome measures
Measure
UGN-102
n=8 Participants
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Complete Response Rate (CRR)
75.0 percentage of participants
Interval 34.9 to 96.8

Adverse Events

UGN-102

Serious events: 3 serious events
Other events: 8 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
UGN-102
n=8 participants at risk
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Respiratory, thoracic and mediastinal disorders
Pleural effusion
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Nervous system disorders
Cerebrovascular accident
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Nervous system disorders
Embolic stroke
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Nervous system disorders
Intracranial aneurysm
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.

Other adverse events

Other adverse events
Measure
UGN-102
n=8 participants at risk
6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Renal and urinary disorders
Dysuria
25.0%
2/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
General disorders
Fatigue
25.0%
2/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Renal and urinary disorders
Hypertonic bladder
25.0%
2/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Infections and infestations
Urinary tract infection
25.0%
2/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Psychiatric disorders
Anxiety
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Cardiac disorders
Atrial flutter
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Reproductive system and breast disorders
Benign prostatic hyperplasia
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Nervous system disorders
Carotid artery stenosis
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Psychiatric disorders
Delirium
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Psychiatric disorders
Depression
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Skin and subcutaneous tissue disorders
Dermatitis contact
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Renal and urinary disorders
Haematuria
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Vascular disorders
Hypertension
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Nervous system disorders
Hypoaesthesia
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Blood and lymphatic system disorders
Leukocytosis
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Renal and urinary disorders
Micturition urgency
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Infections and infestations
Pneumonia
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Renal and urinary disorders
Pollakiuria
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Injury, poisoning and procedural complications
Procedural pneumothorax
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Skin and subcutaneous tissue disorders
Rash
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Renal cell carcinoma
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Cardiac disorders
Sinus tachycardia
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Skin and subcutaneous tissue disorders
Skin irritation
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.
Renal and urinary disorders
Urine flow decreased
12.5%
1/8 • Up to 3 months
Per protocol, sites were instructed to report AEs that occurred from the time of informed consent to the 3-month Visit.

Additional Information

Head of Clinical Development

UroGen Pharma

Phone: +1 855-987-6436

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place