Validation of Instruments for Pragmatic Clinical Trials for Overactive Bladder

NCT01925456 | Completed Phase 1 DMC

• 1 other identifier: 813672
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 1

Brief Summary

A. Objectives \& Hypothesis Overactive bladder (OAB) affects over 10 million adults in the United States and each year substantial costs are incurred from private and public funds to test the efficacy of new and existing drugs for OAB.14 However, effectiveness and adherence data from traditional clinical trials are not generalizable to clinical practice.3, 4 In an era of limited resources and competing demands, it is essential that cost-effectiveness data from clinical trials be generalizable to the clinical world. Pragmatic clinical trials measure the effectiveness of treatments in real clinical practice, and in the full spectrum of patients that require treatment.5 Pragmatic trials require patient reported outcomes that are valid but have low patient burden.5 The conduct of pragmatic clinical trials for OAB has been limited by a lack of instruments that have demonstrable validity and reliability in the typical clinical setting Pill counts, used to measure primary outcomes in most traditional trials, are burdensome in the clinical setting.1610 The optimal instrument for measuring utility scores, general quality of life scores for cost-effectiveness analyses for OAB, is also not known. Although poor adherence carries the potential for continued suffering for patients and wasted health care resources, there is a lack of data on the clinical and economic impact of poor adherence to treatment for OAB in real-world clinical practice. We have published preliminary data that underscores the role of adherence and utilities in the treatment of OAB. 3 Our hypothesis is that effectiveness, adherence, and utility preference scores can be measured using pragmatic patient reported instruments and that poor adherence is associated with lower effectiveness and quality of life in adults with OAB in a clinical setting. Specifically, the overriding goal of this proposal is to validate pragmatic instruments to measure effectiveness, adherence and utility preference scores and obtain preliminary data on the effect of adherence and quality of life in adults undergoing treatment for overactive bladder in a clinical setting.

Conditions

Overactive Bladder

Keywords

urge urinary incontinence

Outcome Measures

Primary Outcomes (1)

• Clinical effectiveness The primary outcome for clinical effectiveness will be patient reported improvement in urinary urgency at 3 months compared to before start of treatment. On two pragmatic instruments, QVD and OAB-q clinical effectiveness will be

  QVD-Questionnaire Based Voiding Diary

  2.5 YEARS

Study Arms (1)

Toviaz

OTHER

Patients willingness to take Toviaz 4mg and 8mg

Drug: Toviaz

Interventions

Toviaz DRUG

Means of effectiveness, adherence, and utility instruments over time will be examined to evaluate whether changes are linear from baseline to 8 weeks and 3 months. We will demonstrate the validity and responsiveness of pragmatic instruments against existing established measures (Table 3).

Also known as: Toviaz4mg and 8mg

Toviaz

Eligibility Criteria

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

You may qualify if:

• urinary urgency for at least 3 months.
• Subjects must have mean of ≥ 8 voids per 24 hours
• and ≥ 1 urgency episode per 24 hours and a rating of at least moderate bother on the single item
• Patient Perception of Bladder Condition.

You may not qualify if:

• \< 18 years
• predominant stress incontinence as measured by the validated 3-Incontinence Questionnaire
• current/recent use (≤6 m) or contra-indication to anti-cholinergic medication for OAB
• severe voiding difficulties in the judgment of the investigator
• men on unstable use of alpha blockers/5-alpha reductase inhibitors
• severe neurologic disease
• anti-incontinence or prolapse surgery
• pregnancy ≤6 months
• pelvic neuromodulation other lower urinary tract disorder such as calculus, urethral diverticulum, current or recurrent urinary tract infections.

Sponsors & Collaborators

• University of Pennsylvania: lead
• Pfizer: collaborator

Study Sites (1)

University of Pennsylvania 3400 Spruce Street-1000 Courtyard

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Urinary Bladder, Overactive; Urinary Incontinence, Urge

Interventions

fesoterodine

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; Urinary Incontinence; Urination Disorders

Study Officials

• Lily A Arya, MS, MD

  University of Pennsylvania

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2013
• First Posted: August 19, 2013
• Study Start: May 1, 2011
• Primary Completion: December 1, 2013
• Study Completion: December 1, 2013
• Last Updated: December 10, 2014

Record last verified: 2011-06

Locations

US (1)