Clinical trial NCT05806164: Beta-Agonist Versus OnabotulinumtoxinA Trial for Urgency Urinary Incontinence
Official title: Beta-Agonist Versus OnabotulinumtoxinA Trial for Urgency Urinary Incontinence
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Urgency Urinary Incontinence
Keywords: UUI
Urgency Incontinence
Beta Agonist
onabotulintoxinA
Botox
Community Engagement
Lead sponsor: Women and Infants Hospital of Rhode Island
Sponsor class: OTHER
Collaborators: name: University of New Mexico
name: University of Alabama at Birmingham
name: University of California, San Diego
name: Howard University
name: Brown University
name: Patient-Centered Outcomes Research Institute
First posted: 2023-04-10
Enrollment: 432
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: At 3 months, the effect of treatment with beta agonist oral medication or onabotulinumtoxinA will be evaluated within a classic RCT model. The analysis will determine the effect of treatment on the co-primary outcomes: Treatment satisfaction and urinary symptom severity.
Primary purpose: TREATMENT

Brief summary: The goal of this clinical trial is to compare treatment outcomes between an oral medication (beta agonist) versus onabotulinumtoxinA injections in women with urgency urinary incontinence (UUI).

Participants will be randomly selected to receive one of the two treatments. The primary outcome measure will be at 3 months, and women will be followed for a total of 12 months.

Based on patient expert input, there are 2 primary outcomes: Treatment satisfaction and urinary symptom severity.

Detailed description: The purpose of this study is to directly compare 2 primary outcomes (Treatment satisfaction and urinary symptom severity) between beta agonist oral medication versus onabotulinumtoxinA intradetrusor bladder injection for the treatment of UUI.

The study will also compare secondary outcomes identified as important by patients. At the end of the study, the investigators will have patient and stakeholder-derived comparative outcomes between these 2 commonly available treatment categories. A stakeholder and community engagement (CE) plan will be developed and implemented. The investigators will also develop a model to help guide patients and providers through this decision process.

SPECIFIC AIMS Specific Aim 1: Compare the efficacy of beta agonist versus onabotulinumtoxinA on patient-important treatment outcomes at 3 months in women with UUI.

This multi-center, randomized clinical trial (RCT) includes 5 sites across the U.S. Two co-primary outcomes will be measured using validated patient-reported outcomes (PROs), selected by patients: Co-primary outcome 1: Symptom severity, measured by change in Overactive Bladder Questionnaire-Symptom Bother Scale (OAB-q-SS) score.

Co-primary outcome 2: Treatment satisfaction, measured by the Functional Assessment of Chronic Illness Therapy-Treatment Satisfaction-General (FACIT-TS-G), powered based on a single item.

Specific Aim 2: Compare secondary patient-important outcomes. Direct comparisons between intervention effects on secondary outcomes chosen by patients and stakeholders, including adverse events, UUI quality of life, global improvement, and sexual function.

Specific Aim 3: Use predictive modeling to help stakeholders better determine expected outcomes after treatment with beta agonist versus onabotulinumtoxinA.

Comparators: Beta agonist oral medication (mirabegron or vibegron) versus intradetrusor onabotulinumtoxinA.

Both beta-agonists and onabotulinumtoxinA are US Food and Drug Administration (FDA) approved for the treatment of UUI, and widely available options with established efficacy.

432 women will be randomly assigned to each treatment option: 216 to beta agonist oral medication and 216 to intradetrusor onabotulintoxinA. Women will be undergo outcomes assessments at 3, 6, 9, and 12 months. The primary outcome measure will be at 3 months.

Eligibility criteria: Inclusion criteria\*:

1. 18 years or older
2. report at least "quite a bit bothered" or worse by their UUI defined by response to OAB-q-SS item #8 "How bothered are you by urine loss associated with a strong desire to urinate?"
3. are not and do not plan to become pregnant
4. have persistent UUI defined as previous unsuccessful results after conservative and anticholinergic treatment, or are unable to tolerate or have contraindications to anticholinergics
5. are currently not taking anticholinergics or are willing to stop medication for 3 weeks prior to enrollment.
6. for participants reporting mixed urinary incontinence symptoms, participant must (a) have less bother from SUI than from UUI, defined as a response of "Not at all bothered" or only "a little bit bothered" by SUI on the Urogenital Distress Inventory item "Do you experience urine leakage related to physical activity? (walking, running, laughing, sneezing, coughing), and (b) SUI symptoms be stable (\> 3 months), and (c)participant does not desire additional treatment for SUI in the upcoming 3 months.
7. Participants after unsuccessful neuromodulation trial can be eligible after a 4-week washout period.

Exclusion criteria:

1. clinical contraindication to beta-3 agonist or onabotulinumtoxinA
2. prior therapeutic trial of either study treatment
3. unevaluated hematuria, current or prior bladder malignancy
4. surgically altered detrusor muscle
5. prior pelvic radiation
6. post-void residual \>150 mL in past 3 months
7. neurogenic bladder
8. pelvic floor surgery within the past 3 months
9. anticipating pelvic surgery within primary outcome follow up period (3 months)

Primary outcomes:
measure: Change in score Overactive Bladder Questionnaire-Symptom Bother Scale (OAB-q-SS) at 3 months
timeFrame: Baseline until 3 months
measure: Functional Assessment of Chronic Illness Therapy-Treatment Satisfaction-General Questionnaire (FACIT-TS-G) at 3 months
timeFrame: 3 months
Secondary outcomes:
measure: Change in Overactive Bladder Questionnaire-Symptom Bother Scale (OAB-q-SS)
timeFrame: Baseline until 6, 9, 12 months
measure: Functional Assessment of Chronic Illness Therapy-Treatment Satisfaction-General Questionnaire (FACIT-TS-G) at 3 months
timeFrame: 6, 9, 12 months
measure: Change in Overactive Bladder Questionnaire-Health Related Quality of Life (OAB-q-HRQL)
timeFrame: Baseline to 3, 6, 9, 12 months
measure: Change in Pelvic Organ Prolapse/Urinary Incontinence Sexual Questionnaire (PISQ-IR)
timeFrame: Baseline to 3, 6, 9, 12 months
measure: Patient global impression of improvement (PGI-I)
timeFrame: 3, 6, 9, 12 months
Other outcomes:
measure: PROMIS Cognitive Function-Short Form
timeFrame: Baseline to 3, 6, 9, 12 months
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: San Diego
country: United States
facility: University of California, San Diego
state: California
city: Washington
country: United States
facility: Howard University
state: District of Columbia
city: Albuquerque
country: United States
facility: University of New Mexico
state: New Mexico
city: Providence
country: United States
facility: Women & Infants Hospital of Rhode Island
state: Rhode Island
Study officials:
affiliation: Women and Infants Hospital of Rhode Island
name: Vivian Sung, MD, MPH
role: PRINCIPAL_INVESTIGATOR
affiliation: University of New Mexico
name: Peter Jeppson, MD
role: PRINCIPAL_INVESTIGATOR
References: