Transvaginal Botulinum Toxin A for Interstitial Cystitis / Bladder Pain Syndrome (T3STOPBPS)
T3STOPBPS: Open Label Clinical Trial of Transvaginal Trigone Treatment (T3) With Botulinum Toxin A (BTA) for Interstitial Cystitis / Bladder Pain Syndrome (IC/BPS)
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Botulinum toxin A (BTA) injected into the bladder wall off-label for the IC/BPS indication has been effective in many women. Unfortunately most women cannot get BTA injection without general anesthesia due to pain from cystoscopy (small camera placed in the bladder). Dr. Dobberfuhl and her team created the transvaginal trigone treatment (T3) approach. Dr. Dobberfuhl was the first investigator in the world to complete a clinical trial of 31 injections of T3 BTA through the vaginal approach, for a non-painful condition, overactive bladder.
The T3STOPBPS study is an open label dose-escalation clinical study of BTA, for women with refractory IC/BPS. BTA is injected through the vaginal wall using a simple technique, the T3 approach, without the need for anesthesia or cystoscopy. The T3 BTA injection procedure has been well tolerated. The T3STOPBPS study will generate data needed for NIH R01 funding which will 1. Bring us closer to making T3 BTA available to women with IC/BPS, 2. Identify neuropeptide (pain) signaling targets for new IC/BPS therapies, and 3. Improve our understanding of fNIRS brain connectivity associated with IC/BPS treatment success.
Twelve patients will be recruited from the clinical practice of Dr. Dobberfuhl to undergo 50 unit T3 BTA injection at baseline. Psychometrically validated questionnaires (AUASS, ICIQ FLUTS, SF12, OLS ICSI / ICPI), voiding diaries, fNIRS and whole urine will be obtained at baseline (pre-treatment), 6 and 12 weeks after T3 BTA. Noninvasive fNIRS brain testing will be performed in collaboration with Dr. Hosseini, which will assess changes in cerebral connectivity from baseline. Whole urine will assess changes in neuropeptide molecular signaling using bulk RNA sequencing following T3 BTA treatment. RNA sequencing will be correlated with our single cell sequencing bladder biopsy data in IC/BPS. Following 50 unit T3 BTA injection, after the 12 week study visit, subjects will be offered dose escalation to 100 units if they do not achieve >50% improvement in PGIC. Milestone for T3 BTA efficacy will be met if >50% improvement is achieved in PGIC versus baseline. Subjects will be eligible for repeat BTA injection when they report less than 50% improvement in symptoms according to the patient global impression of change (PGIC) scale after the 12 week study visit. This will help us understand how long the effect of the BTA lasts, which is typically 3 to 12 months with the traditional cystoscopic route of delivery. A new consent form will be signed at the time of repeat injection.
Study Type
Enrollment (Actual)
Phase
- Early Phase 1
Contacts and Locations
Study Locations
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California
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Palo Alto, California, United States, 94304
- Urology Clinic (Stanford University), 1000 Welch Road, Suite 100
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Females with IC/BPS
- Females who are considering intradetrusor BTA chemodenervation for the treatment of refractory IC/BPS
Exclusion Criteria:
- Involvement in other studies with potentially overlapping indications or symptoms
- Patients who are unable to undergo a transvaginal intervention as a result of anatomic barriers or discomfort will be excluded from enrollment
- patients known to be pregnant or breastfeeding
- Known allergy to BTA injection therapy or lidocaine
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
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Experimental: Transvaginal Botulinum Toxin A (BTA) injection
Botulinum toxin A (Botox® 100 units) will be injected into the detrusor muscle of the bladder by inserting a needle through the anterior vaginal wall.
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Botulinum toxin A (Botox® 100 units) will be injected into the detrusor muscle of the bladder by inserting a needle through the anterior vaginal wall.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Change from baseline in Patient Global Impression of Change (PGIC) scale
Time Frame: Baseline through week 12
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Patients will be classified as treatment success if they achieve our primary efficacy outcome of a >50% improvement in the Patient Global Impression of Change (PGIC) scale
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Baseline through week 12
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Change from baseline in American Urological Association Symptom Score (AUASS)
Time Frame: Baseline through week 12
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American Urological Association Symptom Score
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Baseline through week 12
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Change from baseline in International Consultation on Incontinence Questionnaire Female Lower Urinary Tract Symptoms (ICIQ FLUTS)
Time Frame: Baseline through week 12
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International Consultation on Incontinence Questionnaire Female Lower Urinary Tract Symptoms
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Baseline through week 12
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Change from baseline in Short Form 12-item Survey (SF12)
Time Frame: Baseline through week 12
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Short Form 12-item Survey
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Baseline through week 12
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Change from baseline in O'Leary Sant (OLS) Symptom Score [Interstitial Cystitis Symptom Index (ICSI) / Interstitial Cystitis Problem Index (ICPI)]
Time Frame: Baseline through week 12
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O'Leary Sant Symptom Score (Interstitial Cystitis Symptom Index / Interstitial Cystitis Problem Index)
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Baseline through week 12
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Amy D. Dobberfuhl, M.D., Stanford University
Publications and helpful links
General Publications
- Syan R, Briggs MA, Olivas JC, Srivastava S, Comiter CV, Dobberfuhl AD. Transvaginal ultrasound guided trigone and bladder injection: A cadaveric feasibility study for a novel route of intradetrusor chemodenervation. Investig Clin Urol. 2019 Jan;60(1):40-45. doi: 10.4111/icu.2019.60.1.40. Epub 2018 Dec 24.
- Dobberfuhl AD, van Uem S, Versi E. Trigone as a diagnostic and therapeutic target for bladder-centric interstitial cystitis/bladder pain syndrome. Int Urogynecol J. 2021 Dec;32(12):3105-3111. doi: 10.1007/s00192-021-04878-9. Epub 2021 Jun 22.
- Dobberfuhl AD. Pathophysiology, assessment, and treatment of overactive bladder symptoms in patients with interstitial cystitis/bladder pain syndrome. Neurourol Urodyn. 2022 Nov;41(8):1958-1966. doi: 10.1002/nau.24958. Epub 2022 May 24.
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Mental Disorders
- Pathologic Processes
- Urologic Diseases
- Urinary Bladder Diseases
- Disease
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Urogenital Diseases
- Male Urogenital Diseases
- Syndrome
- Cystitis
- Somatoform Disorders
- Cystitis, Interstitial
- Physiological Effects of Drugs
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Peripheral Nervous System Agents
- Cholinergic Agents
- Membrane Transport Modulators
- Acetylcholine Release Inhibitors
- Neuromuscular Agents
- Botulinum Toxins
- Botulinum Toxins, Type A
- abobotulinumtoxinA
Other Study ID Numbers
- IRB-58532
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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