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

NCT00495053 | Terminated Phase 1 DMC

• 1 other identifier: ION-02
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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).

Conditions

Overactive Bladder

Keywords

overactive bladder, urinary urgency

Outcome Measures

Primary Outcomes (8)

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

  6 months (24 weeks per participant)

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

  18 months

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

  6 months (24 weeks per participant)

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

  18 months

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

  6 months (24 weeks per participant)

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

  18 months

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

  6 months (24 weeks per participant)

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

  18 months

Secondary Outcomes (12)

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

  Baseline; 6 months (24 weeks) per participant

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

  Baseline; 6 months (24 weeks) per participant

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

  Baseline; 6 months (24 weeks) per participant

• Change from Baseline in the overall maximum bladder capacity

  Baseline; 6 months (24 weeks) per participant

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

  Baseline; 6 months (24 weeks) per participant

Study Arms (3)

hMaxi-K 5000 µg/mL

EXPERIMENTAL

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

Drug: hMaxi-K

hMaxi-K 10000 µg/mL

EXPERIMENTAL

10000 µg/90 mL intravesical instillation

Drug: hMaxi-K

Placebo

PLACEBO COMPARATOR

Matching placebo (PBS-20% sucrose)

Other: Placebo

Interventions

hMaxi-K DRUG

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

Also known as: URO-902

hMaxi-K 10000 µg/mL; hMaxi-K 5000 µg/mL

Placebo OTHER

Matching placebo (PBS-20% sucrose)

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: female
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Urovant Sciences GmbH: lead
• Ion Channel Innovations: collaborator

Study Sites (1)

CE3, Inc.

Branford, Connecticut, 06405, United States

Location

MeSH Terms

Conditions

Urinary Bladder, Overactive

Condition Hierarchy (Ancestors)

Urinary Bladder Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Lower Urinary Tract Symptoms; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: sequential, dose escalation

Study Record Dates

• First Submitted: June 29, 2007
• First Posted: July 2, 2007
• Study Start: April 1, 2007
• Primary Completion: July 1, 2008
• Study Completion: November 1, 2009
• Last Updated: June 28, 2019

Record last verified: 2019-06

Locations

US (1)