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Phase 1 Study With OAB Assessing the Safety and Activity of hMaxi-K Gene Transfer (OAB)

Primary Purpose

Overactive Bladder

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Placebo (PBS-20% sucrose)

hMaxi-K

About this trial

This is an interventional other trial for Overactive Bladder focused on measuring Overactive Bladder, OAB, gene therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)FemaleDoes not accept healthy volunteers

Inclusion Criteria:

Healthy women of ≥18 years of age and non-childbearing potential
Symptoms of overactive bladder for ≥6 months including at least one of the following:
1. Frequent micturition ≥8 times per 24 hours
2. Urinary urgency or nocturia
3. Urge urinary incontinence five or more incontinence episodes per week
Detrusor overactivity with ≥1 uncontrolled phasic contraction(s) of the detrusor of at least 5 centimeters/H20 pressure documented on cystometry at Screening Visit 1A
Residual urine volume of ≤200 milliliters (ml)
Non-response or poor tolerance to previous treatment for symptoms of OAB/urinary incontinence and do not wish to continue these treatments
Have screening laboratory values and electrocardiogram that are within the normal range
Able to understand study requirements (i.e., literate in English), give written informed consent, and comply with all study procedures and requirements.

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

AccuMed Research Associates
NYU
Premier Medical Group of the Hudson Valley, PC

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo (PBS-20% sucrose)

hMaxi-K 16000 µg

hMaxi-K 24000 µg

Arm Description

PBS-20% sucrose administered during two single-treatment dose levels (16000 micrograms [µg] and 24000 µg) by direct bladder wall intramuscular injections, 20 to 30 injections depending on active dose comparator.

Single treatment (16000 µg by 20 intramuscular injections). A total of 6 participants will receive hMaxi-K, and 3 will receive placebo.

Single treatment (24000 µg by 30 intramuscular injections). A total of 6 participants will receive hMaxi-K, and 3 will receive placebo.

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 clinically significant clinical laboratory test value during the Treatment Period

Number of participants with any clinically significant clinical laboratory test value during the Safety Follow-up Period

Number of participants with any clinically significant electrocardiogram finding during the Treatment Period

Number of participants with any clinically significant electrocardiogram finding during the Safety Follow-up Period

Number of participants with any clinically significant physical examination finding during the Treatment Period

Number of participants with any clinically significant physical examination finding during the Safety Follow-up Period

Secondary Outcome Measures

Change from Baseline in the number of micturitions per day

Change from Baseline in the volume of micturitions

Change from Baseline in incontinence episodes

Change from Baseline in pad weight

Change from Baseline in uninhibited contractions during cystoscopy

Change from Baseline in the participant rating of the Kings Health Questionnaire (KHQ) score

Change from Baseline in the International Consultation on Incontinence Questionnaire-Short Form score

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

Full Information

NCT ID

NCT01870037

First Posted

June 3, 2013

Last Updated

May 7, 2019

Sponsor

Urovant Sciences GmbH

Collaborators

Ion Channel Innovations

1. Study Identification

Unique Protocol Identification Number

NCT01870037

Brief Title

Phase 1 Study With OAB Assessing the Safety and Activity of hMaxi-K Gene Transfer

Acronym

OAB

Official Title

Phase 1 Double Blind, Placebo Controlled Study Assessing Safety and Activity Of 2 Escalating Doses of hMaxi-K Gene Transfer By Direct Injection Into the Bladder Wall In Female Subjects With OAB

Study Type

Interventional

2. Study Status

Record Verification Date

May 2019

Overall Recruitment Status

Completed

Study Start Date

January 2013 (Actual)

Primary Completion Date

June 2017 (Actual)

Study Completion Date

February 17, 2018 (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 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.

Detailed Description

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]).

6. Conditions and Keywords

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

Overactive Bladder

Keywords

Overactive Bladder, OAB, gene therapy

7. Study Design

Primary Purpose

Other

Study Phase

Phase 1

Interventional Study Model

Sequential Assignment

Model Description

sequential, dose escalation

Masking

ParticipantInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

13 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo (PBS-20% sucrose)

Arm Type

Placebo Comparator

Arm Description

Arm Title

hMaxi-K 16000 µg

Arm Type

Experimental

Arm Description

Single treatment (16000 µg by 20 intramuscular injections). A total of 6 participants will receive hMaxi-K, and 3 will receive placebo.

Arm Title

hMaxi-K 24000 µg

Arm Type

Experimental

Arm Description

Single treatment (24000 µg by 30 intramuscular injections). A total of 6 participants will receive hMaxi-K, and 3 will receive placebo.

Intervention Type

Drug

Intervention Name(s)

Placebo (PBS-20% sucrose)

Other Intervention Name(s)

Placebo

Intervention Type

Drug

Intervention Name(s)

hMaxi-K

Other Intervention Name(s)

URO-902

Intervention Description

Single treatment/2 escalating dose levels (16000 µg and 24000 µg by intramuscular injection)

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 clinically significant clinical laboratory test value during the Treatment Period

Time Frame

6 months (24 weeks) per participant

Title

Number of participants with any clinically significant clinical laboratory test value during the Safety Follow-up Period

Time Frame

18 months

Title

Number of participants with any clinically significant electrocardiogram finding during the Treatment Period

Time Frame

6 months (24 weeks) per participant

Title

Number of participants with any clinically significant electrocardiogram finding during the Safety Follow-up Period

Time Frame

18 months

Title

Number of participants with any clinically significant physical examination finding during the Treatment Period

Time Frame

6 months (24 weeks) per participant

Title

Number of participants with any clinically significant physical examination finding during the Safety Follow-up Period

Time Frame

18 months

Secondary Outcome Measure Information:

Title

Change from Baseline in the number of micturitions per day

Time Frame