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

NCT01870037 | Completed Phase 1 DMC

• 1 other identifier: ION 03-OAB
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 3

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.

Conditions

Overactive Bladder

Keywords

Overactive Bladder; OAB; gene therapy

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 clinically significant clinical laboratory test value during the Treatment Period

  6 months (24 weeks) per participant

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

  18 months

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

  6 months (24 weeks) per participant

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

  18 months

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

  6 months (24 weeks) per participant

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

  18 months

Secondary Outcomes (8)

• Change from Baseline in the number of micturitions per day

  Baseline; 6 months (24 weeks) per participant

• Change from Baseline in the volume of micturitions

  Baseline; 6 months (24 weeks) per participant

• Change from Baseline in incontinence episodes

  Baseline; 6 months (24 weeks) per participant

• Change from Baseline in pad weight

  Baseline; 6 months (24 weeks) per participant

• Change from Baseline in uninhibited contractions during cystoscopy

  Baseline; 6 months (24 weeks) per participant

Study Arms (3)

Placebo (PBS-20% sucrose)

PLACEBO COMPARATOR

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.

Drug: Placebo (PBS-20% sucrose)

hMaxi-K 16000 µg

EXPERIMENTAL

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

Drug: hMaxi-K

hMaxi-K 24000 µg

EXPERIMENTAL

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

Drug: hMaxi-K

Interventions

Placebo (PBS-20% sucrose) DRUG

Also known as: Placebo

Placebo (PBS-20% sucrose)

hMaxi-K DRUG

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

Also known as: URO-902

hMaxi-K 16000 µg; hMaxi-K 24000 µg

Eligibility Criteria

• Age: 18 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Frequent micturition ≥8 times per 24 hours
• Urinary urgency or nocturia
• 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.

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 (3)

AccuMed Research Associates

Garden City, New York, 11530, United States

Location

NYU

New York, New York, 10016, United States

Location

Premier Medical Group of the Hudson Valley, PC

Poughkeepsie, New York, 12601, 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: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: sequential, dose escalation

Study Record Dates

• First Submitted: June 3, 2013
• First Posted: June 5, 2013
• Study Start: January 1, 2013
• Primary Completion: June 1, 2017
• Study Completion: February 17, 2018
• Last Updated: May 8, 2019

Record last verified: 2019-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)