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Safety Study of hMaxi-K Gene Transfer to Treat Overactive Bladder and Detrusor Overactivity

Primary Purpose

Overactive Bladder

Status

Terminated

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

hMaxi-K

Placebo

About this trial

This is an interventional treatment trial for Overactive Bladder focused on measuring overactive bladder, urinary urgency

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)FemaleAccepts Healthy Volunteers

Inclusion Criteria:

Healthy women of 18 years of age or older and of non-childbearing potential
Clinical symptoms of overactive bladder for 6 months or longer including at least one of the following:
1. frequent urination (that is, having to go to the bathroom 8 or more times per day);
2. symptoms of urinary urgency, which is experiencing a sudden need to pass urine that is difficult to hold back; or the complaint of waking at night two ore more times to urinate;
3. urge urinary incontinence, which is the complaint of leakage of urine that cannot be stopped, accompanied by or immediately preceded by urgency 5 or more times per week.
Non-response or poor tolerance to previous treatment for symptoms of overactive bladder and do not wish to continue with that treatment.

Exclusion Criteria:

A woman with a positive serum (HCG) pregnancy test or who is lactating
History of three or more culture-documented recurrent urinary tract infections per year
Current history or previous diagnosis of painful bladder syndrome (interstitial cystitis) with pain in the region of the pelvis, perineum, or lower abdomen relieved by voiding
Current history of neurological bladder dysfunction
A life expectancy of less than 12 months
Current history of Grade 2 or greater cystocele
An indwelling urethral catheter or need for clean intermittent self- catheterization
Recent heart attack
Uncontrolled diabetes
Latex allergy
Stress urinary incontinence as determined by observation of the participant coughing while standing with a full bladder and/or response of 2 or 3 on the following Stress Urinary Incontinence question: Do you experience leakage when laughing, coughing, lifting heavy objects or other types of discreet, moderately intense activities? 0=NONE: No leakage. 1=MILD: Minimal leakage on rare occasions during these types of activities; easily tolerated; do not use pads for this. 2=MODERATE: Enough leakage that it requires occasional use of pads and may interfere with usual activity and tasks. 3=SEVERE: Extreme leakage and discomfort that stops all activity and tasks and requires the use of pads on all occasions

Sites / Locations

CE3, Inc.

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Arm Label

hMaxi-K 5000 µg/mL

hMaxi-K 10000 µg/mL

Placebo

Arm Description

5000 micrograms (µg)/90 milliliter (mL) intravesical instillation

10000 µg/90 mL intravesical instillation

Matching placebo (PBS-20% sucrose)

Outcomes

Primary Outcome Measures

Number of participants with any treatment-emergent adverse event during the treatment period

Number of participants with any treatment-emergent adverse event during the safety follow-up period

Number of participants with any abnormal, clinically significant clinical laboratory test value during the treatment period

Number of participants with any abnormal, clinically significant clinical laboratory test value during the safety follow-up period

Number of participants with any abnormal, clinically significant clinical electrocardiogram value during the treatment period

Number of participants with any abnormal, clinically significant clinical electrocardiogram value during the safety follow-up period

Number of participants with any abnormal, clinically significant clinical physical examination finding during the treatment period

Number of participants with any abnormal, clinically significant clinical physical examination finding during the safety follow-up period

Secondary Outcome Measures

Change from Baseline in the mean number of micturitions per 24 hours

Change from Baseline in the mean number of urge incontinence episodes per 24 hours (collected over a 3-day period)

Change from Baseline in the mean number of urgency episodes per 24 hours

Change from Baseline in the overall maximum bladder capacity

Change from Baseline in the number of uninhibited contractions during the cystometry procedure

Change from Baseline in the participant rating of urgency score

Change from Baseline in the participant rating of perceived bladder condition severity

Number of participants with the indicated response to the following question, as a measure of the participants' assessment of response to treatment: "Has the treatment been of benefit to you"?

Change from Baseline in the Kings Health Questionnaire-Quality of Life (KHQ-QOL) general health perceptions domain score and impact on life domain score

Change from Baseline in the Short Form-12 (SF-12) Health Survey

Change from Baseline in the International Consultation on Incontinence Questionnaire - Short Form (ICIQ-SF)

Change from Baseline in pad weight

Full Information

NCT ID

NCT00495053

First Posted

June 29, 2007

Last Updated

June 26, 2019

Sponsor

Urovant Sciences GmbH

Collaborators

Ion Channel Innovations

1. Study Identification

Unique Protocol Identification Number

NCT00495053

Brief Title

Safety Study of hMaxi-K Gene Transfer to Treat Overactive Bladder and Detrusor Overactivity

Official Title

A Phase 1 Multicenter Study Evaluating the Safety and Potential Activity Of Three Escalating Doses of hMaxi-K Gene Transfer In Female Participants With Overactive Bladder Syndrome and Detrusor Overactivity: Double Blind, Imbalanced Placebo Controlled Design Within 3 Sequential Active Treatment Groups

Study Type

Interventional

2. Study Status

Record Verification Date

June 2019

Overall Recruitment Status

Terminated

Why Stopped

Slow enrollment and financial issues

Study Start Date

April 2007 (undefined)

Primary Completion Date

July 2008 (Actual)

Study Completion Date

November 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Urovant Sciences GmbH

Collaborators

Ion Channel Innovations

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The purpose of this study is to evaluate the safety of a new product that uses human gene transfer (called hMaxi-K) when it is given to patients with overactive bladder. Human gene transfer is a new type of therapy that is the process of placing genetic material (DNA or RNA) into a person. The primary objective of this study is to evaluate safety parameters occurring subsequent to administration of a single intravesical instillation of study drug. Two different dose groups of hMaxi-K will be tested in this study: 5000 and 10000 micrograms. A 15000 microgram group was planned; however, the study was terminated before participants were dosed. hMaxi-K will be given as a single administration into the bladder through a catheter. Each women's participation in the study will last for up to 24 weeks (followed by an additional 18-month follow-up period).

Detailed Description

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 a single intravesical instillation of hMaxi-K compared to the control group. Efficacy parameters that will be evaluated are: mean number of micturitions per 24 hours, mean number of urge incontinence episodes per 24 hours, mean number of urgency episodes per 24 hours, overall maximum bladder capacity, number of uninhibited contractions during the cystometry procedure, participant rating of urgency score, participant rating of perceived bladder condition severity, participant assessment of response to treatment, participant rating of Quality of Life (Kings Health Questionnaire), SF-12 Health Survey, International Consultation on Incontinence Questionnaire (ICIQ-SF), and pad weight measurement of accidental bladder leaks.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Overactive Bladder

Keywords

overactive bladder, urinary urgency

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Sequential Assignment

Model Description

sequential, dose escalation

Masking

ParticipantInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

21 (Actual)

8. Arms, Groups, and Interventions

Arm Title

hMaxi-K 5000 µg/mL

Arm Type

Experimental

Arm Description

5000 micrograms (µg)/90 milliliter (mL) intravesical instillation

Arm Title

hMaxi-K 10000 µg/mL

Arm Type

Experimental

Arm Description

10000 µg/90 mL intravesical instillation

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Matching placebo (PBS-20% sucrose)

Intervention Type

Drug

Intervention Name(s)

hMaxi-K

Other Intervention Name(s)

URO-902

Intervention Description

Two dose levels (5000 µg/90 mL intravesical instillation and 10000 µg/90 mL intravesical instillation)

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

Matching placebo (PBS-20% sucrose)

Primary Outcome Measure Information:

Title

Number of participants with any treatment-emergent adverse event during the treatment period

Time Frame

6 months (24 weeks per participant)

Title

Number of participants with any treatment-emergent adverse event during the safety follow-up period

Time Frame

18 months

Title

Number of participants with any abnormal, clinically significant clinical laboratory test value during the treatment period

Time Frame

6 months (24 weeks per participant)

Title

Number of participants with any abnormal, clinically significant clinical laboratory test value during the safety follow-up period

Time Frame

18 months

Title

Number of participants with any abnormal, clinically significant clinical electrocardiogram value during the treatment period

Time Frame

6 months (24 weeks per participant)

Title

Number of participants with any abnormal, clinically significant clinical electrocardiogram value during the safety follow-up period

Time Frame

18 months

Title

Number of participants with any abnormal, clinically significant clinical physical examination finding during the treatment period

Time Frame

6 months (24 weeks per participant)

Title

Number of participants with any abnormal, clinically significant clinical physical examination finding during the safety follow-up period

Time Frame

18 months

Secondary Outcome Measure Information:

Title

Change from Baseline in the mean number of micturitions per 24 hours

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in the mean number of urge incontinence episodes per 24 hours (collected over a 3-day period)

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in the mean number of urgency episodes per 24 hours

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in the overall maximum bladder capacity

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in the number of uninhibited contractions during the cystometry procedure

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in the participant rating of urgency score

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in the participant rating of perceived bladder condition severity

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Number of participants with the indicated response to the following question, as a measure of the participants' assessment of response to treatment: "Has the treatment been of benefit to you"?

Time Frame

6 months (24 weeks) per participant

Title

Change from Baseline in the Kings Health Questionnaire-Quality of Life (KHQ-QOL) general health perceptions domain score and impact on life domain score

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in the Short Form-12 (SF-12) Health Survey

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in the International Consultation on Incontinence Questionnaire - Short Form (ICIQ-SF)

Time Frame

Baseline; 6 months (24 weeks) per participant

Title

Change from Baseline in pad weight

Time Frame

Baseline; 6 months (24 weeks) per participant

10. Eligibility

Sex

Female

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Healthy women of 18 years of age or older and of non-childbearing potential Clinical symptoms of overactive bladder for 6 months or longer including at least one of the following: frequent urination (that is, having to go to the bathroom 8 or more times per day); symptoms of urinary urgency, which is experiencing a sudden need to pass urine that is difficult to hold back; or the complaint of waking at night two ore more times to urinate; urge urinary incontinence, which is the complaint of leakage of urine that cannot be stopped, accompanied by or immediately preceded by urgency 5 or more times per week. Non-response or poor tolerance to previous treatment for symptoms of overactive bladder and do not wish to continue with that treatment. Exclusion Criteria: A woman with a positive serum (HCG) pregnancy test or who is lactating History of three or more culture-documented recurrent urinary tract infections per year Current history or previous diagnosis of painful bladder syndrome (interstitial cystitis) with pain in the region of the pelvis, perineum, or lower abdomen relieved by voiding Current history of neurological bladder dysfunction A life expectancy of less than 12 months Current history of Grade 2 or greater cystocele An indwelling urethral catheter or need for clean intermittent self- catheterization Recent heart attack Uncontrolled diabetes Latex allergy Stress urinary incontinence as determined by observation of the participant coughing while standing with a full bladder and/or response of 2 or 3 on the following Stress Urinary Incontinence question: Do you experience leakage when laughing, coughing, lifting heavy objects or other types of discreet, moderately intense activities? 0=NONE: No leakage. 1=MILD: Minimal leakage on rare occasions during these types of activities; easily tolerated; do not use pads for this. 2=MODERATE: Enough leakage that it requires occasional use of pads and may interfere with usual activity and tasks. 3=SEVERE: Extreme leakage and discomfort that stops all activity and tasks and requires the use of pads on all occasions

Facility Information:

Facility Name

CE3, Inc.

City

Branford

State/Province

Connecticut

ZIP/Postal Code

06405

Country

United States

12. IPD Sharing Statement

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Safety Study of hMaxi-K Gene Transfer to Treat Overactive Bladder and Detrusor Overactivity

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