Botulinum Toxin A for Bladder Pain Syndrome

(T3STOPBPS Trial)

Overseen By Amy D. Dobberfuhl, M.D.

Age: 18+

Sex: Female

Trial Phase: Phase < 1

Sponsor: Stanford University

Disqualifiers: Pregnancy, Breastfeeding, Others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 4

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment for women with interstitial cystitis, also known as bladder pain syndrome. This condition causes recurring pelvic pain, bladder pressure, and frequent or urgent urination. The trial explores injecting Botulinum Toxin A (commonly known as Botox) into the bladder to determine if it can relieve these symptoms. Women experiencing this painful condition and considering this treatment might be suitable candidates for the trial. As an Early Phase 1 trial, this research aims to understand how this new treatment works in people, offering participants the opportunity to be among the first to receive it.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research shows that Botulinum Toxin A, commonly known as Botox, could be a safe treatment for bladder pain syndrome. Studies have found that injecting Botox into the bladder is usually well-tolerated. Patients with similar conditions have experienced significant relief without major side effects.

A review of several studies on Botox for overactive bladder found it to be both effective and safe. This suggests that Botox might also be safe for people with bladder pain syndrome. Another study confirmed that Botox injections and bladder treatments led to noticeable improvements and were safe for patients with bladder pain.

These findings offer reassurance about the safety of Botox for bladder pain syndrome. However, since this trial is in an early stage, more research is needed to fully understand its safety for this specific condition. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for bladder pain syndrome, which often involve oral medications or invasive surgeries, transvaginal Botulinum Toxin A (BTA) injections offer a novel approach. This treatment directly targets the detrusor muscle of the bladder through a minimally invasive injection, potentially providing more localized relief with fewer systemic side effects. Researchers are excited about BTA because it utilizes Botulinum toxin A, commonly known as Botox®, to relax bladder muscles and reduce pain, offering a fresh mechanism of action compared to traditional methods. This innovative delivery method could lead to faster and more effective symptom management for patients.

What evidence suggests that Botulinum Toxin A might be an effective treatment for bladder pain syndrome?

Research has shown that botulinum toxin A injections can greatly reduce pain for people with bladder pain syndrome. One study found a 25-point drop in pain scores after injections in the pelvic area. Another study discovered that patients who received these injections needed retreatment less often within 6 to 9 months compared to other treatments. Additionally, patients with less bladder inflammation responded well to these injections. Overall, botulinum toxin A appears to be a promising treatment for reducing pain and improving the quality of life for those with bladder pain syndrome. ^{1 3 4 6 7}

Who Is on the Research Team?

Amy D. Dobberfuhl, M.D.

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

This trial is for women with Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) who are considering botulinum toxin A injections due to lack of relief from other treatments. Women who can't have transvaginal procedures, are pregnant or breastfeeding, participating in conflicting studies, or allergic to BTA or lidocaine cannot join.

Inclusion Criteria

I am considering BTA treatment for my severe bladder pain syndrome.

I am a woman with interstitial cystitis/bladder pain syndrome.

Exclusion Criteria

You are currently participating in another study that has similar goals or symptoms.

You are allergic to BTA injection therapy or lidocaine.

I am able to undergo transvaginal procedures without anatomical issues or discomfort.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a 50 unit T3 BTA injection at baseline, with follow-up assessments at 6 and 12 weeks

12 weeks

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with potential dose escalation to 100 units if needed

12 weeks

1 visit (in-person)

Open-label extension (optional)

Participants may receive repeat BTA injection if less than 50% improvement in symptoms is reported

3 to 12 months

What Are the Treatments Tested in This Trial?

Interventions

• Transvaginal Botulinum Toxin A

Trial Overview The study tests the effectiveness of transvaginal botulinum toxin A injections in alleviating symptoms of IC/BPS such as pelvic pain and frequent urge to urinate. The goal is to find a durable treatment option beyond current therapies.

How Is the Trial Designed?

Group I: Transvaginal Botulinum Toxin A (BTA) injection Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials

2,527

Recruited

17,430,000+

McCormick and Gabilan Faculty Fellowship Award

Collaborator

Trials

1

Recruited

5+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7793632/

Effectiveness of botulinum toxin for treatment ... Pelvic floor botulinum toxin injection was associated with an overall 25.0-point reduction in pain scores (95% CI 13.7 – 36.3), consistent with the observed ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05463822

Transvaginal Botulinum Toxin A Chemodenervation for ... 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 ...

3. ajog.org ajog.org/article/S0002-9378(24)00609-4/fulltext

Bladder instillations vs onabotulinumtoxinA injection for ... Patients who underwent onabotulinumtoxinA injection were significantly less likely to receive retreatment within 6 to 9 months compared to ...

4. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0002937824006094

Bladder instillations vs onabotulinumtoxinA injection for ... Our findings suggest that intradetrusor onabotulinumtoxinA injection is a more effective procedural treatment for this condition than bladder instillation ...

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36548768/

Treatment Outcomes of Intravesical Botulinum Toxin A ... IC/BPS patients with less bladder inflammation showed satisfactory outcomes with intravesical BoNT-A injections.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12037478/

Safety and efficacy of intravesical instillation of botulinum toxin ... The meta-analysis was performed to assess the efficacy and safety of a novel intravesical instillation of BTX-A for managing overactive bladder (OAB) or ...

7. clinicaltrials.stanford.edu clinicaltrials.stanford.edu/trials/t/NCT05485207.html

Transvaginal Botulinum Toxin A for Interstitial Cystitis ... Women suffer from recurring pelvic pain, bladder pressure, painful bladder, urinary frequency (needing to go often) and urgency (feeling a strong need to go).