Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Botulinum Toxin Type A in Adult Patients with Chronic Pelvic Pain Syndrome

The primary objective of this phase 3, randomized, double-blind, placebo-controlled, multi-center study is to establish the **efficacy** of botulinumtoxin-A (BoNT/A) injections in the pelvic floor muscles for reducing pain in patients with **Chronic Pelvic Pain Syndrome (CPPS)**. This is clinically relevant as CPPS is a debilitating condition that significantly impacts the quality of life, and effective pain management is crucial for improving patient outcomes.

Secondary objectives include assessing the treatment's safety and its effects on urinary, bowel, and sexual function, as well as overall quality of life. These evaluations are important to ensure that the treatment not only alleviates pain but also does not adversely affect other critical aspects of patient health and daily functioning.

The clinical trial involves **adult patients**, both male and female, diagnosed with **chronic pelvic pain syndrome (CPPS)**. The study population includes individuals aged 18 years and older. Participants were selected based on their ability to provide informed consent and a confirmed CPPS diagnosis according to the European Association of Urology guidelines. Additionally, participants must have experienced a mean worst daily pain intensity of 4 or higher on the Pain Intensity Numerical Rating Scale (PI-NRS) for at least one week within the month preceding the first visit, and have failed at least one intervention for chronic pelvic pain according to current best clinical practice. The sponsor has not provided information regarding the total number of participants. The trial does not include a vulnerable population, and no specific lifestyle considerations such as diet or physical activity are highlighted in the selection criteria.

This clinical trial is a **phase 3**, randomized, double-blind, placebo-controlled, multi-center study designed to evaluate the efficacy of **botulinum toxin type A** injections in the pelvic floor muscles for reducing pain in patients with **chronic pelvic pain syndrome (CPPS)**. The trial will involve adult patients, both male and female, who meet specific inclusion criteria, such as being 18 years or older, having a CPPS diagnosis according to the European Association of Urology guidelines, and experiencing a mean worst daily pain intensity of 4 or higher on a Pain Intensity Numeric Rating Scale (PI-NRS) within the month preceding the screening visit. Participants must also have failed at least one prior intervention for chronic pelvic pain.

The trial will span an estimated duration from April 2025 to September 2026, with participant involvement lasting up to 24 weeks. The study will commence with a screening visit to confirm eligibility, followed by randomization into either the treatment group receiving **botulinum toxin type A** or a matching placebo. The primary endpoint is the proportion of patients achieving a 30% or greater reduction in pain intensity at week 4 compared to baseline. Secondary endpoints include assessments at various intervals (weeks 2, 6, 8, 10, 12, 14, 16, 18, 20, 22, and 24) for pain reduction, changes in rescue medication usage, and scores on various health and symptom questionnaires.

Study visits will occur at regular intervals, with key assessments at weeks 4, 8, 12, 16, 20, and 24. These visits will evaluate changes in pain intensity, medication use, and adverse events, among other health metrics. The end-of-study visit will conclude the trial for each participant, assessing the overall outcomes and any long-term effects. Participants may be withdrawn from the study early if they experience significant adverse events, fail to comply with study procedures, or withdraw consent. The trial is structured to ensure rigorous data collection and analysis, maintaining the integrity and scientific validity of the results.

The clinical trial involves the administration of **BOTOX 100 Allergan Units Powder for Solution for Injection**, which contains the active substance **botulinum toxin type A**. This investigational medicinal product is provided in the form of a powder that is reconstituted to create a solution for injection. The pharmaceutical form is specifically designed for **intramuscular injection**. The maximum daily dose is 100 international units (IU), with a total maximum dose of 100 IU over the treatment period. The treatment duration is set for a maximum of 24 weeks. The investigational product is packaged in a masked or blinded manner to ensure it appears similar to the placebo packaging, maintaining the integrity of the double-blind study design.

The study also includes a **matching placebo** that is identical in appearance to the investigational medicinal product. The placebo is used to maintain the double-blind nature of the trial, ensuring that neither the participants nor the investigators are aware of the treatment assignments. The placebo does not contain any active substance and is administered in the same manner as the investigational product, following the same dosing schedule and route of administration. This design allows for a controlled comparison to evaluate the efficacy of botulinum toxin type A in reducing pain in patients with chronic pelvic pain syndrome (CPPS).

The efficacy of **botulinum toxin type A** in the treatment of Chronic Pelvic Pain Syndrome (CPPS) will be assessed through a phase 3, randomized, double-blind, placebo-controlled, multi-center study. The primary endpoint for evaluating efficacy is the proportion of patients considered responders, defined as achieving a ≥30% reduction in pain intensity compared to baseline, at week 4. Pain intensity will be measured using the Pain Intensity Numeric Rating Scale (PI-NRS) based on a 7-day pain diary, capturing the mean of the worst daily pain intensity over the preceding week.

Secondary endpoints include the proportion of responders at additional scheduled assessments (weeks 2, 6, 8, 10, 12, 14, 16, 18, 20, 22, and 24), changes in the amount of rescue medication taken, and changes in scores from the DN4 questionnaire and Pelvic Floor Muscles Hyperalgesia (PFMH) score at each scheduled visit. Additional assessments will include the incidence and severity of adverse events, changes in the International Prostate Symptom Score (IPSS), Wexner score, Female Sexual Distress Scale (FSDS), International Index of Erectile Function (IIEF) score, and Short Form 12 Health Survey (SF-12) score at scheduled visits (weeks 4, 8, 12, 16, 20, and 24) compared to baseline.