The primary objective of this study is to evaluate the safety of a single treatment of hMaxi-K compared to placebo (PBS-20% sucrose) administered by direct bladder wall injections. Two dose levels (16000 µg and 24000 µg; 20 or 30 bladder wall injections, respectively) in females with moderate Overactive Bladder/Detrusor overactivity (OAB/DO) of ≥6 months duration will be evaluated. In each dose level, 6 participants will receive hMaxi-K and 3 will receive placebo.
This is a Phase 1 double-blind, placebo-controlled study assessing the safety and activity of 2 escalating doses of hMaxi-K gene transfer by direct injection into the bladder wall in female participants with OAB/DO. The safety parameters to be monitored include: adverse events, clinical laboratory tests, electrocardiograms, and physical examinations. The secondary objective is to evaluate the efficacy of multiple intramuscular injections of hMaxi-K compared to the control group. Efficacy parameters that will be evaluated are: number of micturitions per 24 hours, volume per micturition, incontinence episodes, pad weight measurement of accidental bladder leaks, uninhibited contractions during cystoscopy and other cystoscopic evaluations, and general and bladder-specific quality of life assessments (Kings Health Questionnaire, SF-12 Health Survey, and International Consultation on Incontinence Questionnaire \[ICIQ-SF\]).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
OTHER
Masking
TRIPLE
Enrollment
13
Single treatment/2 escalating dose levels (16000 µg and 24000 µg by intramuscular injection)
AccuMed Research Associates
Garden City, New York, United States
NYU
New York, New York, United States
Premier Medical Group of the Hudson Valley, PC
Poughkeepsie, New York, United States
Number of participants with any treatment-emergent adverse event during the Treatment Period
Time frame: 6 months (24 weeks) per participant
Number of participants with any treatment-emergent adverse event during the Safety Follow-up Period
Time frame: 18 months
Number of participants with any clinically significant clinical laboratory test value during the Treatment Period
Time frame: 6 months (24 weeks) per participant
Number of participants with any clinically significant clinical laboratory test value during the Safety Follow-up Period
Time frame: 18 months
Number of participants with any clinically significant electrocardiogram finding during the Treatment Period
Time frame: 6 months (24 weeks) per participant
Number of participants with any clinically significant electrocardiogram finding during the Safety Follow-up Period
Time frame: 18 months
Number of participants with any clinically significant physical examination finding during the Treatment Period
Time frame: 6 months (24 weeks) per participant
Number of participants with any clinically significant physical examination finding during the Safety Follow-up Period
Time frame: 18 months
Change from Baseline in the number of micturitions per day
Time frame: Baseline; 6 months (24 weeks) per participant
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Change from Baseline in the volume of micturitions
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in incontinence episodes
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in pad weight
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in uninhibited contractions during cystoscopy
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the participant rating of the Kings Health Questionnaire (KHQ) score
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the International Consultation on Incontinence Questionnaire-Short Form score
Time frame: Baseline; 6 months (24 weeks) per participant
Change from Baseline in the Short Form-12 (SF-12) score
Time frame: Baseline; 6 months (24 weeks) per participant