Overactive BladderPhase 1PfizerOABLUTNCT01925456
Validation of Instruments for Pragmatic Clinical Trials for Overactive Bladder
A Phase 1 study testing Toviaz in people with Overactive Bladder, run by University of Pennsylvania at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older, women onlyHealthy volunteers can take part
- Conditions the study is for
- Overactive Bladder
- What is being tested?
- Toviaz
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 1 siteUnited States
- Study dates
- May 2011to Dec 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Women only
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- < 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
Show all 9Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Pennsylvania 3400 Spruce Street-1000 Courtyard
Philadelphia, Pennsylvania, 19104
About this study
Official title: Validation of Instruments for Pragmatic Clinical Trials for Overatcive Bladder
- 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.
Read the detailed description
Specific Aim 1: To determine the validity and responsiveness of pragmatic patient reported outcome instruments to measure clinical effectiveness, adherence and utility preference scores in adults undergoing treatment with anti-cholinergic medications for overactive bladder. We will perform a prospective cohort study of adults with OAB undergoing treatment with anti-cholinergic medication. We will compare improvement in urinary symptoms (clinical effectiveness), adherence, and change in utility preference scores from baseline to 3 months of treatment using new and existing 'gold standard' instruments. The results will be informative as to which, if any of these pragmatic instruments, is useful for measuring effectiveness, adherence, and utility preference scores in the 'real world' clinical setting.
Specific Aim 2: To use the newly validated pragmatic instruments to measure the impact of adherence on clinical effectiveness and health related quality of life in a real world clinical setting in adults undergoing treatment with anti-cholinergic medications for overactive bladder. The impact of adherence on clinical effectiveness and health related quality of life will be measured through comparisons in adults with high and low adherence to anti-cholinergic medication. This aim will corroborate the findings of aim 1 and establish if pragmatic instruments are useful in measuring outcomes of effectiveness, adherence and utility preference scores. These results will also provide preliminary data on the effectiveness of anti-cholinergic medication and the impact of adherence on clinical effectiveness and quality of life in the clinical setting.
- Conditions
- Overactive Bladder
- Being tested
- Toviaz
- Keywords
- urge urinary incontinence
- Medical subject headings
- Urinary Bladder, Overactive, Urinary Incontinence, Urge
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ToviazDrug
Also called Toviaz4mg and 8mg
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).
| Study group | Role | What they receive |
|---|---|---|
| Toviaz | Other | Patients willingness to take Toviaz 4mg and 8mg Receives: Drug: Toviaz |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 2.5 YEARS
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2013 (estimated)
- Study startMay 2011
- Primary completionDec 2013
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Overactive BladderStudies of the same condition, open and under way.
- More trials run by University of PennsylvaniaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Sponsor
- Investigators
- Lily A Arya, MS, MD, Principal investigator, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01925456
- Sponsor's protocol ID
- 813672
- Acronym
- PfizerOABLUT
- Status last verified
- Jun 2011
- First submitted
- 6 Aug 2013
- Last updated
- 10 Dec 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Validation of Instruments for Pragmatic Clinical Trials for Overatcive Bladder
- ClinicalTrials.gov ID
- NCT01925456
- Acronym / sponsor ID
- PfizerOABLUT / 813672
- Conditions
- Overactive Bladder
- Keywords
- urge urinary incontinence
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- May 2011
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 19 Aug 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
- 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.
Detailed description
Specific Aim 1: To determine the validity and responsiveness of pragmatic patient reported outcome instruments to measure clinical effectiveness, adherence and utility preference scores in adults undergoing treatment with anti-cholinergic medications for overactive bladder. We will perform a prospective cohort study of adults with OAB undergoing treatment with anti-cholinergic medication. We will compare improvement in urinary symptoms (clinical effectiveness), adherence, and change in utility preference scores from baseline to 3 months of treatment using new and existing 'gold standard' instruments. The results will be informative as to which, if any of these pragmatic instruments, is useful for measuring effectiveness, adherence, and utility preference scores in the 'real world' clinical setting.
Specific Aim 2: To use the newly validated pragmatic instruments to measure the impact of adherence on clinical effectiveness and health related quality of life in a real world clinical setting in adults undergoing treatment with anti-cholinergic medications for overactive bladder. The impact of adherence on clinical effectiveness and health related quality of life will be measured through comparisons in adults with high and low adherence to anti-cholinergic medication. This aim will corroborate the findings of aim 1 and establish if pragmatic instruments are useful in measuring outcomes of effectiveness, adherence and utility preference scores. These results will also provide preliminary data on the effectiveness of anti-cholinergic medication and the impact of adherence on clinical effectiveness and quality of life in the clinical setting.
Study design
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Toviaz | Other | Drug: ToviazPatients willingness to take Toviaz 4mg and 8mg |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (9 listed)
- < 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ToviazAlso: Toviaz4mg and 8mgMeans 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). | Drug | Toviaz |
Primary outcome
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
Time frame: 2.5 YEARS
QVD-Questionnaire Based Voiding Diary
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2014.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lily A Arya, MS, MD principal investigator
University of Pennsylvania
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Toviaz in people with Overactive Bladder, run by University of Pennsylvania at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: 2.5 YEARS). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Dec 2014.