Interstitial Cystitis
Conditions
Keywords
women, interstitial cystitis, bladder pain syndrome, botox, onabotulinum toxin A
Brief summary
The purpose of this research study is to identify the optimal usage of Onabotulinumtoxin A in interstitial cystitis. Onabotulinumtoxin A, more commonly known by the trade name Botox®, is a medication that comes from the bacteria Clostridium botulinum. It works by blocking the release of the neurotransmitter acetylcholine(a chemical messenger that carries signals between nerve cells and other cells in the body). Blocking that neurotransmitter results in decreased muscle activity.
Detailed description
The purpose of this study is to evaluate the efficacy of intravesicular Onabotulinumtoxin A injections for the treatment of interstitial cystitis (IC). Specifically, we hypothesize that trigonal Onabotulinumtoxin A injections is an effective treatment for IC and will result in more subjective and objective symptom relief than posterior wall Onabotulinumtoxin A injections.
Interventions
100 units of botox spread out among 10 separate injections
PROCEDUREinjections upper aspect of trigone of urinary bladder
We hypothesize that injections into the trigone should be more effective in the treatment of IC than injections elsewhere in the bladder.
PROCEDUREinjections on posterior bladder wall excluding the trigone
We hypothesize that injections into the trigone should be more effective in the treatment of IC than injections elsewhere in the bladder.
Sponsors
Allergan
Wake Forest University Health Sciences
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Adult females between the ages of 18 and 80 inclusive 2. Patients being treated for IC who are refractory to conservative management and oral therapy. 3. willing and able to initiate catheterization post-treatment
Exclusion criteria
1. Any history of bladder cancer, uterine cancer, ovarian cancer, vaginal cancer, urethral diverticulum, spinal cord injury, stroke, Parkinson's disease, multiple sclerosis, spina bifida, cyclophosphamide treatment, radiation treatment to the pelvis, bladder tuberculosis, genital herpes. 2. Currently on or requiring anti-platelet/anti-coagulant concomitant therapy or having been on anti-platelet/ anti-coagulant therapy within the past 3 months 3. Pregnancy. Pregnancy is an absolute contraindication to undergoing these procedures. Thus, as part of their normal pre-operative work up, which is standard of care, pregnancy tests are administered if they are women of child-bearing age, are sexually active, and are within 10 days of the normal menstrual period. If positive, they will be excluded as they will not undergo the procedure. 4. An active urinary tract infection as shown during clean-catch urinalysis at screening visit. Subject may be re-screened if UTI is successfully treated and urinalysis is negative at rescreening. 5. A history of hypersensitivity or allergy to any botulinum toxin preparation 6. A post-void residual (PVR) urine volume \>200mL at baseline 7. Treatment with botulinum toxin during the 12 week period prior to the trial
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | 30 and 90 days post treatment | The O'Leary-Sant is one questionnaire that assesses the severity of symptoms and the how much of a problem the symptoms cause for the patient and it provides two scores. The scores ranges for the symptoms is 0-20 and for how bothersome the symptoms are, the score range is 0-16. Higher scores for both denotes worse outcomes. |
| The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | 30 and 90 days post-treatment | The PUF questionnaire evaluates symptoms of pain and how much they bother the patient. Two score are given and added together to produce a total score. The score range for symptoms is 0-28 and the range for bother is 0-16. Higher scores denotes worse outcomes. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Patient Performance in Uroflowmetry. | 30 days and 90 days post treatment | Uroflowmetry is a test that measures the volume of urine released from the body, the speed with which it is released, and how long the release takes. |
Other
| Measure | Time frame |
|---|---|
| Secondary Outcomes Will be Assessing Change in Patient Performance in Post Void Residuals. | 30 days and 90 days post treatment |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Botox Upper Aspect Trigone Subjects in the experimental cohort will receive a one time dose of 100 units of Onabotulinumtoxin A diluted in 10 mL of preservative free normal saline and injected in 1.0 mL boluses in a set pattern across the upper aspect of the trigone of the urinary bladder.
Onabotulinumtoxin A: 100 units of botox spread out among 10 separate injections
injections upper aspect of trigone of urinary bladder: We hypothesize that injections into the trigone should be more effective in the treatment of IC than injections elsewhere in the bladder. | 13 |
| Botox Periphery of Trigone Each group will receive a total of 100 units of botox spread out among 10 separate injections. Subjects in the control group will have 10 injections made about the periphery of the trigone. The control cohort will receive a one time dose of Onabotulinumtoxin A using the same dilution and number of boluses, but boluses will be administered at random sites on the posterior bladder wall (excluding the trigone).
Onabotulinumtoxin A: 100 units of botox spread out among 10 separate injections
injections on posterior bladder wall excluding the trigone: We hypothesize that injections into the trigone should be more effective in the treatment of IC than injections elsewhere in the bladder. | 14 |
| Total | 27 |
Baseline characteristics
| Characteristic | Botox Periphery of Trigone | Total | Botox Upper Aspect Trigone |
|---|---|---|---|
| Age, Continuous | 46.5 years STANDARD_DEVIATION 12.32 | 47.41 years STANDARD_DEVIATION 12.76 | 48.38 years STANDARD_DEVIATION 13.64 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 1 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 13 Participants | 26 Participants | 13 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 13 Participants | 25 Participants | 12 Participants |
| Sex: Female, Male Female | 14 Participants | 27 Participants | 13 Participants |
| Sex: Female, Male Male | 0 Participants | 0 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 13 | 0 / 14 |
| other Total, other adverse events | 4 / 13 | 7 / 14 |
| serious Total, serious adverse events | 0 / 13 | 0 / 14 |
Outcome results
Primary
The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes.
The O'Leary-Sant is one questionnaire that assesses the severity of symptoms and the how much of a problem the symptoms cause for the patient and it provides two scores. The scores ranges for the symptoms is 0-20 and for how bothersome the symptoms are, the score range is 0-16. Higher scores for both denotes worse outcomes.
Time frame: 30 and 90 days post treatment
Population: One participant was lost to follow-up in the experimental group and no data was collected at 30 days on one participant in the control group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Botox Upper Aspect Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | Symptoms @ 30 days | 10.92 units on a scale | Standard Deviation 3.15 |
| Botox Upper Aspect Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | Bother @ 30 days | 9.75 units on a scale | Standard Deviation 4 |
| Botox Upper Aspect Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | Symptoms @ 90 days | 10.75 units on a scale | Standard Deviation 3.62 |
| Botox Upper Aspect Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | Bother @ 90 days | 10.17 units on a scale | Standard Deviation 3.16 |
| Botox Periphery of Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | Bother @ 90 days | 10.29 units on a scale | Standard Deviation 4.07 |
| Botox Periphery of Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | Symptoms @ 30 days | 10.85 units on a scale | Standard Deviation 4.18 |
| Botox Periphery of Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | Symptoms @ 90 days | 12.00 units on a scale | Standard Deviation 4.54 |
| Botox Periphery of Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the O'Leary-Sant Symptom and Problem Indexes. | Bother @ 30 days | 9.92 units on a scale | Standard Deviation 4.61 |
Primary
The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire
The PUF questionnaire evaluates symptoms of pain and how much they bother the patient. Two score are given and added together to produce a total score. The score range for symptoms is 0-28 and the range for bother is 0-16. Higher scores denotes worse outcomes.
Time frame: 30 and 90 days post-treatment
Population: One participant from the experimental group was lost to follow-up. Data was not collected at 30 days for bother and symptom for one participant in the control group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Botox Upper Aspect Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | Symptoms @ 30 days | 13.83 units on a scale | Standard Deviation 4.9 |
| Botox Upper Aspect Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | Bother @ 30 days | 7.33 units on a scale | Standard Deviation 2.77 |
| Botox Upper Aspect Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | Symptoms @ 90 days | 13.33 units on a scale | Standard Deviation 4.75 |
| Botox Upper Aspect Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | Bother @ 90 days | 7.25 units on a scale | Standard Deviation 2.83 |
| Botox Periphery of Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | Bother @ 90 days | 7.5 units on a scale | Standard Deviation 2.77 |
| Botox Periphery of Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | Symptoms @ 30 days | 12.31 units on a scale | Standard Deviation 4.71 |
| Botox Periphery of Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | Symptoms @ 90 days | 13.21 units on a scale | Standard Deviation 4.42 |
| Botox Periphery of Trigone | The Primary Outcome Will be Assessing the Measurement of Subjective Patient Pain Using the Pelvic Pain and Urinary Urgency Frequency (PUF) Questionnaire | Bother @ 30 days | 7.31 units on a scale | Standard Deviation 3.17 |
Secondary
Change in Patient Performance in Uroflowmetry.
Uroflowmetry is a test that measures the volume of urine released from the body, the speed with which it is released, and how long the release takes.
Time frame: 30 days and 90 days post treatment
Population: No data was collected for two participants in the experimental group and one participant in the control group at 30 days. No data was collected for one participant in the experimental group at 90 days.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Botox Upper Aspect Trigone | Change in Patient Performance in Uroflowmetry. | @ 30 days | 11.82 ml/s | Standard Deviation 5.64 |
| Botox Upper Aspect Trigone | Change in Patient Performance in Uroflowmetry. | @ 90 days | 12.17 ml/s | Standard Deviation 7366 |
| Botox Periphery of Trigone | Change in Patient Performance in Uroflowmetry. | @ 30 days | 9.5 ml/s | Standard Deviation 7.83 |
| Botox Periphery of Trigone | Change in Patient Performance in Uroflowmetry. | @ 90 days | 15.5 ml/s | Standard Deviation 11.89 |
Other Pre-specified
Secondary Outcomes Will be Assessing Change in Patient Performance in Post Void Residuals.
Time frame: 30 days and 90 days post treatment
Population: Data was not collected on one participant in the experimental group at 30 and 90 days post treatment. Data was not collected on two individuals at 30 days post treatment in the control group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Botox Upper Aspect Trigone | Secondary Outcomes Will be Assessing Change in Patient Performance in Post Void Residuals. | @ 30 days | 27.17 mL | Standard Deviation 37.06 |
| Botox Upper Aspect Trigone | Secondary Outcomes Will be Assessing Change in Patient Performance in Post Void Residuals. | @ 90 days | 38.92 mL | Standard Deviation 48.1 |
| Botox Periphery of Trigone | Secondary Outcomes Will be Assessing Change in Patient Performance in Post Void Residuals. | @ 30 days | 35.33 mL | Standard Deviation 48 |
| Botox Periphery of Trigone | Secondary Outcomes Will be Assessing Change in Patient Performance in Post Void Residuals. | @ 90 days | 60.64 mL | Standard Deviation 71.38 |